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This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell and progenitor stem cell (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) transplantation using lovo-cel.
Subject participation for this study will be 2 years post-transplant. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for an additional 13 years for a total of 15 years post-drug product infusion.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group A | Experimental | Participants who had rescue cells that were collected by bone marrow harvest method and had received treatment with lovo-cel which consists of autologous CD34+ hematopoietic stem cells (HSCs) and progenitor stem cells (PSCs) (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) collected from participants with sickle cell disease (SCD) by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (the original drug product manufacturing process for this study). |
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| Group B | Experimental | Group B1 participant had rescue cells and drug product cells that were collected by bone marrow harvest method and drug product was manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene. This participant's drug product was produced in 2 lots each using two different manufacturing processes (the original drug product manufacturing process and a refined drug product manufacturing process). Group B2 Plerixafor mobilization and apheresis were used for collection of rescue cells and exploratory manufacturing development. A single Group B2 participant received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (using only the refined drug product manufacturing process). Note: Groups B1 and B2 are combined as "Group B" for results reporting purposes. |
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| Group C | Experimental | Plerixafor mobilization and apheresis were used for collection of rescue cells, and drug product. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by plerixafor mobilization and apheresis transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene using a further refined manufacturing process similar to commercial manufacturing. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| lovo-cel | Genetic | lovo-cel is administered by IV infusion following myeloablative conditioning with busulfan. |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Group C Participants Who Achieved Complete Resolution of Vaso-occlusive Events (VOE-CR) | VOE-CR was defined as complete resolution of adjudicated VOEs between 6 months and 18 months post lovo-cel infusion. All reported VOEs were also adjudicated by an independent Event Adjudication Committee for purposes of endpoint analysis. VOEs were determined by adjudication committee after referring to protocol VOE. The committee was responsible for VOE assessment and determining whether an event met criteria for a VOE for all reported events. | From 6 months to 18 months post lovo-cel infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Group C Participants With Complete Resolution of Severe VOEs (sVOE-CR) | sVOE-CR was defined as the complete resolution of sVOEs, in the 6 to 18 months post lovo-cel infusion. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of endpoint analysis (referred to as Adjudicated sVOEs). sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. |
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Inclusion Criteria:
Be ≥12 and ≤50 of age at time of consent.
Diagnosis of sickle cell disease (SCD), with either βS/βS or βS/β0 or βS/β+ genotype.
Have severe SCD. i.e., in the setting of appropriate supportive care measures for SCD (e.g., pain management plan), have experienced at least 4 severe VOEs in the 24 months prior to informed consent.
For the purposes of this study, a severe VOE is defined as an event with no medically determined cause other than a vaso-occlusion, requiring a ≥ 24-hour hospital or Emergency Room (ER) observation unit visit or at least 2 visits to a day unit or ER over 72 hours with both visits requiring intravenous treatment. Exception: priapism does not require hospital admission but does require a medical facility visit; 4 priapism episodes that require a visit to a medical facility (without inpatient admission) are sufficient to meet criterion.
Severe VOEs include:
Karnofsky performance status of ≥ 60 (≥16 years of age) or a Lansky performance status of ≥60 (<16 years of age).
Have either experienced hydroxyurea (HU) failure at any point in the past or must have intolerance to HU (defined as patient being unable to continue to take HU per PI judgement).
Have been treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on SCD history.
Exclusion Criteria:
Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 and HIV-2), hepatitis B virus (HBV), or hepatitis C (HCV).
Clinically significant and active bacterial, viral, fungal, or parasitic infection.
Inadequate bone marrow function, as defined by an absolute neutrophil count of < 1000/µL (< 500/µL for subjects on HU treatment) or a platelet count < 100,000/µL.
Any history of severe cerebral vasculopathy: defined by overt or hemorrhagic stroke; abnormal transcranial Doppler [≥200 cm/sec] needing chronic transfusion; or occlusion or stenosis in the polygon of Willis; or presence of Moyamoya disease. Subjects with radiologic evidence of silent infarction in the absence of any of the above criteria would still be eligible
Baseline oxygen saturation < 90% without supplemental oxygen (excluding periods of SCD crisis, severe anemia or infection).
Baseline carbon monoxide diffusing capacity (DLCO) < 50% (corrected for Hb) in the absence of infection. If DLco cannot be assessed due to age or cognition-related restrictions, there must be a normal respiratory exam, chest radiograph without pulmonary infiltrates, and oxygen saturation by pulse oximetry ≥ 90% on room air.
Baseline left ventricular ejection fraction (LVEF) < 45% measured by cardiac echography.
Clinically significant pulmonary hypertension at baseline, as defined by the requirement for ongoing pharmacologic treatment or the consistent or intermittent use of supplemental home oxygen.
Baseline estimated glomerular filtration rate (eGFR) < 70 mL/min/1.73 m2, as determined using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (see http://www.kidney.org/professionals/kdoqi/gfr\_calculator.cfm).
Advanced liver disease, defined as:
For subjects who have history of iron overload or serum ferritin levels > 1000 ng/mL, a cardiac MRI is required. Cardiac T2* < 10 ms results in exclusion.
Contraindication to anesthesia.
Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin.
Prior receipt of an allogeneic transplant.
Immediate family member with a known or suspected Familial Cancer Syndrome.
Diagnosis of significant psychiatric disorder of the subject that, in the Investigator's judgment, could seriously impede the ability to participate in the study.
Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile subjects.
Participation in another clinical study with an investigational drug within 30 days of Screening.
Prior receipt of gene therapy.
An assessment by the Investigator that the subject or parents/caregivers (as required) will not be able to comply with the study procedures outlined in the study protocol.
Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment (patients on prophylactic doses of anticoagulants not excluded per this criteria).
Unable to receive Red Blood Cell (RBC) transfusion.
Any other condition that would render the subject ineligible for hematopoietic stem cell transplant (HSCT), as determined by the attending transplant physician.
Applicable to subjects < 18 years of age only: Availability of a willing, matched HLA-identical sibling hematopoietic cell donor.
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| Name | Affiliation | Role |
|---|---|---|
| Anjulika Chawla, MD, FAAP | bluebird bio, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42133908 | Derived | Kern KC, Inam Z, Hsieh MM, Tisdale JF, Fitzhugh CD, Limerick EM, Gebremeskel ASK, White T, Jordan LC, Lynch JK. Hematopoietic Stem Cell Transplant and Brain Volume Changes in Adults With Sickle Cell Disease. Neurology. 2026 Jun 9;106(11):e218050. doi: 10.1212/WNL.0000000000218050. Epub 2026 May 14. | |
| 36161320 | Derived |
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Bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared (if contractually or otherwise legally permitted) following study completion and and once all applicable regulatory submissions based on this study have been performed. Sharing of individual patient data will be done according to criteria established by bluebird bio and/or industry best practices to protect confidential information and maintain the privacy of study participants. For inquiries, please contact us at datasharing@bluebirdbio.com.
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Participants were enrolled as Groups A, B (B1 and B2) and C (see Reporting Groups). Since B1 and B2 contained only 2 participants and due to other factors (eg, logistical considerations), results for B1 and B2 are reported as "Group B" throughout this summary.
A total of 54 participants-initiated stem cell collection and included in Intent-to-Treat (ITT) population, and of those 45 participants treated with lovo-cel and included in the transplant population.
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| ID | Title | Description |
|---|---|---|
| FG000 | Group A | Participants who had rescue cells that were collected by bone marrow harvest method and had received treatment with lovo-cel which consists of autologous CD34+ hematopoietic stem cells (HSCs) and progenitor stem cells (PSCs) (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) collected from patients with sickle cell disease (SCD) by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (the original drug product manufacturing process for this study). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 17, 2023 | Jul 30, 2024 |
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| From 6 months to 18 months post lovo-cel infusion |
| Percentage of Group C Participants Who Achieved Globin Response | Globin Response is defined as meeting the following criteria for a continuous period of at least 6 months post lovo-cel infusion (starting at least 60 days after last packed red blood cell [pRBC] transfusion):
| From at least 60 days after last pRBC transfusion up to Month 24 post lovo-cel infusion |
| Percentage of Group C Participants Who Meet the Definition of Globin Response at Month 24 | Globin Response at Month 24 was defined as continuously meeting the following criteria from the first date of globin response initiating to Month 24 assessment post lovo-cel infusion:
| From first date of Globin Response to Month 24 post lovo-cel infusion |
| Duration of Globin Response in Group C Participants | Duration of Globin Response is the time period from the first date of Globin Response initiating to the date of last high pressure liquid chromatography (HPLC) assessment such that the weighted average HbAT87Q (%) in non-transfused total Hb and non-transfused total Hb continuously meet Globin Response criteria.
| From first date of Globin Response to Month 24 post lovo-cel infusion |
| Change From Baseline in the Annualized Number of VOEs in Group C Participants | Change from baseline in the annualized number of VOEs was assessed from 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs were also adjudicated by an independent event adjudication committee for purposes of outcome analysis. VOEs were determined by adjudication committee after referring to protocol definition of VOE. The committee was responsible for VOE assessment and determining whether an event met criteria for a VOE for all reported events. | From Baseline up to 24 months post lovo-cel infusion |
| Change From Baseline in the Annualized Number of sVOEs in Group C Participants | Change from baseline in the annualized number of sVOEs was assessed from 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of endpoint analysis. sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | From Baseline up to 24 months post lovo-cel infusion |
| Percentage of Group C Participants With Complete Resolution of VOE Between 6 and 24 Months Post Lovo-cel Infusion (VOE-CR24) | VOE-CR24, defined as complete resolution of VOE between 6 months and 24 months post lovo-cel infusion. All reported VOEs were also adjudicated by an independent Event Adjudication Committee for purposes of outcome analysis. VOEs were determined by adjudication committee after referring to protocol definition of VOE. The committee was responsible for VOE assessment and determining whether an event met criteria for a VOE for all reported events. | From 6 months to 24 months post lovo-cel infusion |
| Percentage of Group C Participants With Complete Resolution of sVOEs Between 6 and 24 Months Post Lovo-cel Infusion (sVOE-CR24) | sVOE-CR24, defined as complete resolution of sVOEs in 6 to 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of outcome analysis. sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | From 6 months to 24 months post lovo-cel infusion |
| Percentage of Group C Participants With At Least 75% Reduction in Annualized sVOEs (sVOE-75) | sVOE-75, defined as at least a 75% reduction in annualized sVOEs in the 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of outcome analysis. sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | Up to 24 months post lovo-cel infusion |
| Change From Baseline in Annualized VOE-related Hospital Admissions in Group C Participants | Change from baseline was calculated as annualized number of VOE-related hospital admissions during post-drug product infusion discharge through the last study visit minus annualized number of VOE related hospital admissions at baseline. Baseline was annualized number of VOE-related hospital admissions in 24 months prior to the Informed Consent. | From post hospital discharge to Month 24 |
| Change From Baseline in Annualized VOE-related Total Days Hospitalized in Group C Participants | Change from baseline was calculated as annualized number of VOE-related hospital days during post-drug product infusion discharge through the last study visit minus annualized number of VOE related hospital days at baseline. Baseline was annualized number of VOE-related hospital days in 24 months prior to the Informed Consent. | From post hospital discharge to Month 24 |
| Weighted Average of Non-transfused Total Hb in Group C Participants | Non-transfused total Hb was the total g/dL of HbS + HbF + HbA2 + HbAT87Q for participants without beta + allele or HbS + HbF + HbA2 + HbAT87Q + HbA for participants with beta + allele. | At Month 6, 12, 18, and 24 post lovo-cel infusion |
| Weighted Average of HbS Percentage (%) in Non-transfused Total Hb in Group C Participants | HbS % in non-transfused total Hb = (HbS/ non-transfused total Hb)*100. | At Month 6, 12, 18, and 24 post lovo-cel infusion |
| Percentage of Group C Participants Who Achieved <= 70%, <= 60%, and <= 50% Weighted Average of HbS % in Non-transfused Total Hb | HbS % in non-transfused total Hb = (HbS/ non-transfused total Hb)*100. The denominator of the percentage is based on the total number of participants who have non-missing value at the Visit. | At Month 6, 12, 18, and 24 post lovo-cel infusion |
| Weighted Average of HbAT87Q % in Non-transfused Total Hb in Group C Participants | HbAT87Q % in non-transfused total Hb was calculated as: HbAT87Q / non-transfused total Hb *100. | At Month 6, 12, 18, and 24 post lovo-cel infusion |
| Weighted Average of Non-HbS % in Non-transfused Total Hb in Group C Participants | Non-HbS percentage in non-transfused total Hb = [(HbF + HbA2 + HbAT87Q)/ non-transfused total Hb] *100. For participants with beta + allele, HbA was also included in the calculated for "non-HbS" for samples taken >= 60 days after last pRBC transfusion. | At Month 6, 12, 18, and 24 post lovo-cel infusion |
| Non-transfused Total Hb (g/dL) Over Time | Non-transfused total Hb was the total g/dL of HbS + HbF + HbA2 + HbAT87Q for participants without beta + allele or HbS + HbF + HbA2 + HbAT87Q + HbA for participants with beta + allele. | From infusion up to Month 24 |
| HbS % in Non-transfused Total Hb Over Time | HbS (%) in non-transfused total Hb = (HbS / non-transfused total Hb)*100. | From infusion up to Month 24 |
| HbAT87Q % in Non-transfused Total Hb Over Time | HbAT87Q (%) in Non-transfused total Hb = (HbAT87Q / non-transfused total Hb)*100. | From infusion up to Month 24 |
| Non-HbS % of Non-transfused Total Hb Over Time | Non-HbS % in non-transfused total Hb = [(HbF + HbA2 + HbAT87Q)/ non-transfused total Hb] *100. | From infusion up to Month 24 |
| Change From Baseline in Absolute Reticulocyte Count in Group C Participants | Reticulocytes are immature RBCs that develop in the bone marrow and circulate in the bloodstream for about a day before developing into mature RBCs. Due to the loss of mature RBCs during hemolysis, there is usually a high demand to produce new RBCs in subjects with SCD, resulting in higher than normal absolute reticulocyte counts. For this outcome Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Percent (%) Reticulocyte/Erythrocytes in Group C Participants | Reticulocytes are immature RBCs that develop in the bone marrow and circulate in the bloodstream for about a day before developing into mature RBCs. The fraction of reticulocytes/erythrocytes in the blood is normally 0.5% to 2.5%, and is increased when there is peripheral hemolytic anemia. Due to the loss of mature RBCs during hemolysis, there is usually a high demand to produce new RBCs in subjects with SCD, resulting in higher than normal reticulocyte counts. For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Total Bilirubin in Group C Participants | Reduction of red blood cell sickling with lovo-cel has the potential to reduce or eliminate downstream complications, including hemolysis. Total Bilirubin is a marker of RBC hemolysis and so increases in Total Bilirubin levels suggests increased hemolysis. For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Haptoglobin in Group C Participants | Reduction of red blood cell sickling with lovo-cel has the potential to reduce or eliminate downstream complications, including hemolysis. Haptoglobin is a marker of RBC hemolysis and so increases in Haptoglobin levels suggests increased hemolysis. For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. It should be noted that Baseline haptoglobin levels may be impacted by a range of factors that result in variability, including (but not limited to) other therapies that a participant receives (such as blood transfusions or hydroxyurea), which limit interpretability of these data. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Lactate Dehydrogenase (LDH) in Group C Participants | Reduction of red blood cell sickling with lovo-cel has the potential to reduce or eliminate downstream complications, including hemolysis. Lactate Dehydrogenase is a marker of RBC hemolysis and so increases in LDH levels suggests increased hemolysis, For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Cardiac T2* on MRI in Group C Participants | Particulate intracellular iron causes shortening of the magnetic resonance relaxation parameter T2 due to microscopic magnetic field inhomogeneity. A myocardial T2 value of 40 msec, which equates to 0.50 mg/g of dry weight iron has widely been used as the normal mean. Myocardial T2. Values below 20 msec are considered abnormal, and values below 10 msec indicate high risk of cardiac morbidity and mortality. For this outcome, Baseline was defined as the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 24 |
| Change From Baseline in Serum Ferritin in Group C Participants | Serum ferritin is commonly used for an indirect estimation of body iron stores. Although sensitive, it is not specific for iron overload as it can be elevated in a variety of infectious and inflammatory states, and in the presence of cytolysis. For this outcome, Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Liver Iron Concentration (LIC) by Magnetic Resonance Imaging (MRI) in Group C Participants | Liver Iron Concentration of greater than 15 mg per gram dry weight (mg/g) is associated with an increased risk of hepatic disease and cardiac toxicity, whereas levels between 7 to 15 mg/g enhance the risk of hepatic fibrosis and endocrine complications. Liver Iron Concentration of greater than 1.8 mg/g dry weight is considered abnormal; however, organ damage generally does not occur with levels less than 7 mg/g. For this outcome, Baseline was defined as the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 24 |
| Change From Baseline in Annualized Volume of pRBC Transfusions in Group C Participants | Change from baseline was calculated as annualized volume of pRBC transfusions in 6 to 24 months post lovo-cel infusion - annualized volume of pRBC transfusions at baseline. Baseline was the annualized volume of pRBC transfusion in the 24 months prior to the Informed Consent. | From 6 months through 24 months post lovo-cel infusion |
| Change From Baseline in Annualized Number of Packed Red Blood Cell (pRBC) Transfusions in Group C Participants | Change from baseline was calculated as annualized number of pRBC transfusions in 6 to 24 months post lovo-cel infusion - annualized number of pRBC transfusions at baseline. Baseline was the annualized number of pRBC transfusion in the 24 months prior to the Informed Consent. | From 6 months through 24 months post lovo-cel infusion |
| Change From Baseline in Erythropoietin Levels in Group C Participants | Erythropoietin is a hormone produced mainly by the kidneys in response to hypoxia, which can be caused by anemia. Patients with SCD typically produce higher than normal levels of serum erythropoietin. Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Cardiac-pulmonary Function Via Left Ventricular Ejection Fraction (LVEF) in Group C Participants | LVEF is the central measure of left ventricular systolic function. LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). This was measured by ECHO. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 24 |
| Change From Baseline in Serum Transferrin Receptor in Group C Participants | Transferrin is a circulating iron carrier protein, delivering iron to cells via the transferrin receptor, and levels of serum transferrin receptor have been used as an indicator of excess iron in the body. Additionally, the transferrin receptor is highly expressed in erythroblasts, and increased levels of the soluble form (serum transferrin receptor) have been shown to be associated with increased levels of erythropoiesis. Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Renal Function as Measured by Estimated Glomerular Filtration Rate (eGFR) in Group C Participants | eGFR was calculated by chronic kidney disease epidemiology collaboration (CKD-EPI) formula for participants >=18 years of age and Schwartz formula was used for participants <18 years of age. Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 12 and Month 24 |
| Number of Participants With Shift From Baseline in Cardiac-pulmonary Function Via Echocardiogram (Tricuspid Regurgitant Jet Velocity [TRJV]) in Group C Participants | TRJV over time for each participant was classified into 2 categories: <2.5 m/sec versus >=2.5 m/sec. Higher value of TRJV indicates worse result. A TRJV value of >2.5 m/s was associated with a higher risk of complications, especially in participants with Sickle Cell Disease due to hemolysis and pulmonary hypertension. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 24 |
| Number of Participant With Shift From Baseline in Cardiac-pulmonary Function Via Pulmonary Function Tests (PFTs) in Group C Participants | PFTs over time of each participant were classified into one of the five categories: normal, obstructive, restrictive, mixed obstructive and restrictive and isolated low DLco (carbon monoxide diffusing capacity). Only non-zero values are reported here. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | Baseline, Month 24 |
| Change From Baseline in Meters Walked During 6-minute Walk Test in Group C Participants | The 6-minute walk test measures the distance in meters walked during 6 minutes and can be used to assess pulmonary function. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 12 and Month 24 |
| Change From Baseline in Patient-reported Quality of Life as Measured by Patient Reported Outcomes Measurement Information System-57 (PROMIS-57): Domain (T- Score) in Group C Participants | The PROMIS-57 profile was a self-reported questionnaire assessing quality of life in various 7 domains (pain interference, physical function, sleep disturbance, Ability to participate in social roles and activities, anxiety, depression, and fatigue) in participants >=18 years old.These 7 domains are scored on a 5-point Likert scale and converted into standardized T-scores with a mean of 50 and a standard deviation of 10 based on a US general population. The higher of the T-score: the better of physical function, satisfaction with participation in social roles, and ability of participate in social roles in activities; The higher of the T-score: the worse of anxiety, depression, fatigue, sleep disturbance, and pain interference. A negative value indicates improvement, and a positive value indicates no improvement in condition. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 24 |
| Change From Baseline in Patient-reported Quality of Life as Measured by PROMIS-57: Pain Intensity Score in Group C Participants | The PROMIS-57 profile was a self-reported questionnaire assessing quality of life in various domains in participants >=18 years old. Pain intensity was scored from 0 to 10, with higher scores indicating greater pain intensity. A negative value indicates improvement in pain intensity and a positive value indicates no improvement or worsening in pain intensity. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 24 |
| Change From Baseline in Patient-reported Quality of Life as Measured by Patient Reported Outcomes Measurement Information System- 49 (PROMIS-49)): Domain (T- Score) in Group C Participants | The PROMIS-49 profile was a self-reported questionnaire assessing quality of life in various 6 domains (physical function mobility, peer relationships, pain interference, anxiety, depression, and fatigue) in participants <=18 years old. These 6 domains are scored on a 5-point Likert scale and converted into standardized T-scores with a mean of 50 and a standard deviation of 10 based on a US general population. The higher of the T-score, the better of physical function mobility, and peer relationships; The higher of the T-score, the worse of anxiety, depression symptoms, fatigue, and pain interference. A negative value indicates improvement, and a positive value indicates no improvement in condition. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 24 |
| Change From Baseline in Patient-reported Quality of Life as Measured by PROMIS-49: Pain Intensity Score in Group C Participants | The PROMIS-49 profile was a self-reported questionnaire assessing quality of life in various domains in participants <=18 years old. Pain intensity was scored from 0 to 10, with higher scores indicating greater pain intensity. A negative value indicates improvement in pain intensity and a positive value indicates no improvement or worsening in pain intensity. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | Baseline, Month 24 |
| Oakland |
| California |
| United States |
| Atlanta | Georgia | 30322 | United States |
| Chicago | Illinois | United States |
| Bethesda | Maryland | United States |
| Hackensack | New Jersey | United States |
| New Hyde Park | New York | United States |
| New York | New York | United States |
| Chapel Hill | North Carolina | United States |
| Philadelphia | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Kanter J, Thompson AA, Pierciey FJ Jr, Hsieh M, Uchida N, Leboulch P, Schmidt M, Bonner M, Guo R, Miller A, Ribeil JA, Davidson D, Asmal M, Walters MC, Tisdale JF. Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study. Am J Hematol. 2023 Jan;98(1):11-22. doi: 10.1002/ajh.26741. Epub 2022 Oct 10. |
| 34922648 | Derived | Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available. |
| 34898139 | Derived | Kanter J, Walters MC, Krishnamurti L, Mapara MY, Kwiatkowski JL, Rifkin-Zenenberg S, Aygun B, Kasow KA, Pierciey FJ Jr, Bonner M, Miller A, Zhang X, Lynch J, Kim D, Ribeil JA, Asmal M, Goyal S, Thompson AA, Tisdale JF. Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. N Engl J Med. 2022 Feb 17;386(7):617-628. doi: 10.1056/NEJMoa2117175. Epub 2021 Dec 12. |
| 34115136 | Derived | Jones RJ, DeBaun MR. Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither. Blood. 2021 Sep 16;138(11):942-947. doi: 10.1182/blood.2021011488. |
| 33811823 | Derived | Del Pozo Martin Y. 47th Annual Meeting of the EBMT. Lancet Haematol. 2021 May;8(5):e317-e318. doi: 10.1016/S2352-3026(21)00104-6. Epub 2021 Mar 31. No abstract available. |
| FG001 | Group B | Group B included only 2 participants treated with drug product cells collected differently and with drug product manufactured using different processes (one participant in Group B1 and one participant in Group B2 as detailed below). Note that, due to the small size of Groups B1 and B2 and due to other factors (including logistical considerations), these intermediate groups are combined as "Group B" throughout this summary for results reporting purposes. Group B1 participant had rescue cells and drug product cells that were collected by bone marrow harvest method and drug product was manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene. This participant's drug product was produced in 2 lots each using two different manufacturing processes (the original drug product manufacturing process and a refined drug product manufacturing process). Group B2 Plerixafor mobilization and apheresis were used for collection of rescue cells and exploratory manufacturing development. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (using only the refined drug product manufacturing process). |
| FG002 | Group C | Plerixafor mobilization and apheresis were used for collection of rescue cells, and drug product. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by plerixafor mobilization and apheresis transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene using a further refined manufacturing process similar to commercial manufacturing. |
| Transplant Population (TP) | TP included participants who received lovo-cel (45 TP participants). |
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| Transplant Population for VOE (TPVOE) | TPVOE is subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent (40 TPVOE participants). |
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| COMPLETED |
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| NOT COMPLETED |
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ITT population was defined as all participants who initiate any study procedures, beginning with stem cell collection procedures (mobilization/apheresis or bone marrow harvest).
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Group A | Participants who had rescue cells that were collected by bone marrow harvest method and had received treatment with lovo-cel which consists of autologous CD34+ hematopoietic stem cells (HSCs) and progenitor stem cells (PSCs) (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) collected from patients with sickle cell disease (SCD) by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (the original drug product manufacturing process for this study). |
| BG001 | Group B | Group B included only 2 participants treated with drug product cells collected differently and with drug product manufactured using different processes (one participant in Group B1 and one participant in Group B2 as detailed below). Note that, due to the small size of Groups B1 and B2 and due to other factors (including logistical considerations), these intermediate groups are combined as "Group B" throughout this summary for results reporting purposes. Group B1 participant had rescue cells and drug product cells that were collected by bone marrow harvest method and drug product was manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene. This participant's drug product was produced in 2 lots each using two different manufacturing processes (the original drug product manufacturing process and a refined drug product manufacturing process). Group B2 Plerixafor mobilization and apheresis were used for collection of rescue cells and exploratory manufacturing development. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (using only the refined drug product manufacturing process). |
| BG002 | Group C | Plerixafor mobilization and apheresis were used for collection of rescue cells, and drug product. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by plerixafor mobilization and apheresis transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene using a further refined manufacturing process similar to commercial manufacturing. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Median | Full Range | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
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| Primary | Percentage of Group C Participants Who Achieved Complete Resolution of Vaso-occlusive Events (VOE-CR) | VOE-CR was defined as complete resolution of adjudicated VOEs between 6 months and 18 months post lovo-cel infusion. All reported VOEs were also adjudicated by an independent Event Adjudication Committee for purposes of endpoint analysis. VOEs were determined by adjudication committee after referring to protocol VOE. The committee was responsible for VOE assessment and determining whether an event met criteria for a VOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis in SAP, data was analyzed and reported for Group C participants. Analysis of efficacy endpoint for Group C was planned to be reported based on the adjudicated vaso-occlusive events (VOEs). | Posted | Number | 95% Confidence Interval | percentage of participants | From 6 months to 18 months post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants With Complete Resolution of Severe VOEs (sVOE-CR) | sVOE-CR was defined as the complete resolution of sVOEs, in the 6 to 18 months post lovo-cel infusion. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of endpoint analysis (referred to as Adjudicated sVOEs). sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis in SAP, data was analyzed and reported for Group C participants. | Posted | Number | 95% Confidence Interval | percentage of participants | From 6 months to 18 months post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants Who Achieved Globin Response | Globin Response is defined as meeting the following criteria for a continuous period of at least 6 months post lovo-cel infusion (starting at least 60 days after last packed red blood cell [pRBC] transfusion):
| TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported only for TP Group C participants. | Posted | Number | 95% Confidence Interval | percentage of participants | From at least 60 days after last pRBC transfusion up to Month 24 post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants Who Meet the Definition of Globin Response at Month 24 | Globin Response at Month 24 was defined as continuously meeting the following criteria from the first date of globin response initiating to Month 24 assessment post lovo-cel infusion:
| TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported only for TP Group C participants. Here, "Overall number of participants analyzed" signifies TP Group C participants who achieved Globin Response. | Posted | Number | 95% Confidence Interval | percentage of participants | From first date of Globin Response to Month 24 post lovo-cel infusion |
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| Secondary | Duration of Globin Response in Group C Participants | Duration of Globin Response is the time period from the first date of Globin Response initiating to the date of last high pressure liquid chromatography (HPLC) assessment such that the weighted average HbAT87Q (%) in non-transfused total Hb and non-transfused total Hb continuously meet Globin Response criteria.
| TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported only for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who achieved Globin Response. | Posted | Median | Full Range | months | From first date of Globin Response to Month 24 post lovo-cel infusion |
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| Secondary | Change From Baseline in the Annualized Number of VOEs in Group C Participants | Change from baseline in the annualized number of VOEs was assessed from 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs were also adjudicated by an independent event adjudication committee for purposes of outcome analysis. VOEs were determined by adjudication committee after referring to protocol definition of VOE. The committee was responsible for VOE assessment and determining whether an event met criteria for a VOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported for TPVOE Group C participants. | Posted | Median | Full Range | Number of VOEs/year | From Baseline up to 24 months post lovo-cel infusion |
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| Secondary | Change From Baseline in the Annualized Number of sVOEs in Group C Participants | Change from baseline in the annualized number of sVOEs was assessed from 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of endpoint analysis. sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported for TPVOE Group C participants. | Posted | Median | Full Range | Number of sVOEs/year | From Baseline up to 24 months post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants With Complete Resolution of VOE Between 6 and 24 Months Post Lovo-cel Infusion (VOE-CR24) | VOE-CR24, defined as complete resolution of VOE between 6 months and 24 months post lovo-cel infusion. All reported VOEs were also adjudicated by an independent Event Adjudication Committee for purposes of outcome analysis. VOEs were determined by adjudication committee after referring to protocol definition of VOE. The committee was responsible for VOE assessment and determining whether an event met criteria for a VOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported for TPVOE Group C participants. | Posted | Number | 95% Confidence Interval | percentage of participants | From 6 months to 24 months post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants With Complete Resolution of sVOEs Between 6 and 24 Months Post Lovo-cel Infusion (sVOE-CR24) | sVOE-CR24, defined as complete resolution of sVOEs in 6 to 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of outcome analysis. sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported for TPVOE Group C participants. | Posted | Number | 95% Confidence Interval | percentage of participants | From 6 months to 24 months post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants With At Least 75% Reduction in Annualized sVOEs (sVOE-75) | sVOE-75, defined as at least a 75% reduction in annualized sVOEs in the 24 months post lovo-cel infusion compared to the 24 months prior to Informed Consent. All reported VOEs (including sVOEs) were adjudicated by an independent Event Adjudication Committee for purposes of outcome analysis. sVOE were determined by adjudication committee after referring to protocol definition of sVOE. The committee was responsible for VOE assessment and determining whether an event met criteria for an sVOE for all reported events. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis, data was analyzed and reported for TPVOE Group C participants. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 24 months post lovo-cel infusion |
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| Secondary | Change From Baseline in Annualized VOE-related Hospital Admissions in Group C Participants | Change from baseline was calculated as annualized number of VOE-related hospital admissions during post-drug product infusion discharge through the last study visit minus annualized number of VOE related hospital admissions at baseline. Baseline was annualized number of VOE-related hospital admissions in 24 months prior to the Informed Consent. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis in SAP, data was analyzed and reported for Group C participants. | Posted | Median | Full Range | hospital admissions per year | From post hospital discharge to Month 24 |
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| Secondary | Change From Baseline in Annualized VOE-related Total Days Hospitalized in Group C Participants | Change from baseline was calculated as annualized number of VOE-related hospital days during post-drug product infusion discharge through the last study visit minus annualized number of VOE related hospital days at baseline. Baseline was annualized number of VOE-related hospital days in 24 months prior to the Informed Consent. | TPVOE was subset of TP participants who had at least 4 protocol VOEs in the 24 months prior to Informed Consent.TP included participants who received lovo-cel. As per planned analysis in SAP, data was analyzed and reported for Group C participants. | Posted | Median | Full Range | hospitalized days per year | From post hospital discharge to Month 24 |
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| Secondary | Weighted Average of Non-transfused Total Hb in Group C Participants | Non-transfused total Hb was the total g/dL of HbS + HbF + HbA2 + HbAT87Q for participants without beta + allele or HbS + HbF + HbA2 + HbAT87Q + HbA for participants with beta + allele. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refer to the participants who were evaluable at specified time points. Evaluable participants are defined as participants who have weighted average of non-transfused total Hb at Month 6, 12, 18 and 24. | Posted | Median | Full Range | gram per deciliter (g/dL) | At Month 6, 12, 18, and 24 post lovo-cel infusion |
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| Secondary | Weighted Average of HbS Percentage (%) in Non-transfused Total Hb in Group C Participants | HbS % in non-transfused total Hb = (HbS/ non-transfused total Hb)*100. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refer to the participants who were evaluable at specified time points. Evaluable participants are defined as participants who have weighted average of HbS % in Non-transfused Total Hb at Month 6, 12, 18 and 24. | Posted | Median | Full Range | percentage of non-transfused total HbS | At Month 6, 12, 18, and 24 post lovo-cel infusion |
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| Secondary | Percentage of Group C Participants Who Achieved <= 70%, <= 60%, and <= 50% Weighted Average of HbS % in Non-transfused Total Hb | HbS % in non-transfused total Hb = (HbS/ non-transfused total Hb)*100. The denominator of the percentage is based on the total number of participants who have non-missing value at the Visit. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refer to the participants who were evaluable at specified time points. Evaluable participants are defined as participants who achieved <= 70%, <= 60%, and <= 50% HbS at Month 6, 12, 18 and 24. | Posted | Number | Percentage of participants | At Month 6, 12, 18, and 24 post lovo-cel infusion |
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| Secondary | Weighted Average of HbAT87Q % in Non-transfused Total Hb in Group C Participants | HbAT87Q % in non-transfused total Hb was calculated as: HbAT87Q / non-transfused total Hb *100. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refer to the participants who were evaluable at specified time points. Evaluable participants are defined as participants who have weighted average of HbAT87Q % in Non-transfused Total Hb at Month 6, 12, 18 and 24. | Posted | Median | Full Range | percentage of non-transfused total Hb | At Month 6, 12, 18, and 24 post lovo-cel infusion |
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| Secondary | Weighted Average of Non-HbS % in Non-transfused Total Hb in Group C Participants | Non-HbS percentage in non-transfused total Hb = [(HbF + HbA2 + HbAT87Q)/ non-transfused total Hb] *100. For participants with beta + allele, HbA was also included in the calculated for "non-HbS" for samples taken >= 60 days after last pRBC transfusion. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refer to the participants who were evaluable at specified time points. Evaluable participants are defined as participants who have weighted average of Non-HbS % in Non-transfused Total Hb at Month 6, 12, 18 and 24. | Posted | Median | Full Range | percentage of non-HbS in total Hb | At Month 6, 12, 18, and 24 post lovo-cel infusion |
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| Secondary | Non-transfused Total Hb (g/dL) Over Time | Non-transfused total Hb was the total g/dL of HbS + HbF + HbA2 + HbAT87Q for participants without beta + allele or HbS + HbF + HbA2 + HbAT87Q + HbA for participants with beta + allele. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. | Posted | Median | Full Range | g/dL | From infusion up to Month 24 |
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| Secondary | HbS % in Non-transfused Total Hb Over Time | HbS (%) in non-transfused total Hb = (HbS / non-transfused total Hb)*100. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. | Posted | Median | Full Range | Percent | From infusion up to Month 24 |
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| Secondary | HbAT87Q % in Non-transfused Total Hb Over Time | HbAT87Q (%) in Non-transfused total Hb = (HbAT87Q / non-transfused total Hb)*100. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. | Posted | Median | Full Range | Percent | From infusion up to Month 24 |
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| Secondary | Non-HbS % of Non-transfused Total Hb Over Time | Non-HbS % in non-transfused total Hb = [(HbF + HbA2 + HbAT87Q)/ non-transfused total Hb] *100. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. | Posted | Median | Full Range | Percent | From infusion up to Month 24 |
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| Secondary | Change From Baseline in Absolute Reticulocyte Count in Group C Participants | Reticulocytes are immature RBCs that develop in the bone marrow and circulate in the bloodstream for about a day before developing into mature RBCs. Due to the loss of mature RBCs during hemolysis, there is usually a high demand to produce new RBCs in subjects with SCD, resulting in higher than normal absolute reticulocyte counts. For this outcome Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who have absolute reticulocyte count at Baseline and at Months 12 and 24. | Posted | Median | Full Range | cells*10^9 per liter (cells*10^9/L) | Baseline, Month 12 and Month 24 |
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| Secondary | Change From Baseline in Percent (%) Reticulocyte/Erythrocytes in Group C Participants | Reticulocytes are immature RBCs that develop in the bone marrow and circulate in the bloodstream for about a day before developing into mature RBCs. The fraction of reticulocytes/erythrocytes in the blood is normally 0.5% to 2.5%, and is increased when there is peripheral hemolytic anemia. Due to the loss of mature RBCs during hemolysis, there is usually a high demand to produce new RBCs in subjects with SCD, resulting in higher than normal reticulocyte counts. For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who achieved % Reticulocyte/Erythrocytes at Baseline and at Months 12 and 24. | Posted | Median | Full Range | Percent | Baseline, Month 12 and Month 24 |
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| Secondary | Change From Baseline in Total Bilirubin in Group C Participants | Reduction of red blood cell sickling with lovo-cel has the potential to reduce or eliminate downstream complications, including hemolysis. Total Bilirubin is a marker of RBC hemolysis and so increases in Total Bilirubin levels suggests increased hemolysis. For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who achieved total bilirubin count at Baseline and at Months 12 and 24. | Posted | Median | Full Range | milligram per deciliter (mg/dL) | Baseline, Month 12 and Month 24 |
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| Secondary | Change From Baseline in Haptoglobin in Group C Participants | Reduction of red blood cell sickling with lovo-cel has the potential to reduce or eliminate downstream complications, including hemolysis. Haptoglobin is a marker of RBC hemolysis and so increases in Haptoglobin levels suggests increased hemolysis. For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. It should be noted that Baseline haptoglobin levels may be impacted by a range of factors that result in variability, including (but not limited to) other therapies that a participant receives (such as blood transfusions or hydroxyurea), which limit interpretability of these data. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who achieved haptoglobin count at Baseline and at Months 12 and 24. | Posted | Median | Full Range | mg/dL | Baseline, Month 12 and Month 24 |
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| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) in Group C Participants | Reduction of red blood cell sickling with lovo-cel has the potential to reduce or eliminate downstream complications, including hemolysis. Lactate Dehydrogenase is a marker of RBC hemolysis and so increases in LDH levels suggests increased hemolysis, For this outcome, Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who achieved lactate dehydrogenase count at Baseline and at Months 12 and 24. | Posted | Median | Full Range | units per liter (U/L) | Baseline, Month 12 and Month 24 |
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| Secondary | Change From Baseline in Cardiac T2* on MRI in Group C Participants | Particulate intracellular iron causes shortening of the magnetic resonance relaxation parameter T2 due to microscopic magnetic field inhomogeneity. A myocardial T2 value of 40 msec, which equates to 0.50 mg/g of dry weight iron has widely been used as the normal mean. Myocardial T2. Values below 20 msec are considered abnormal, and values below 10 msec indicate high risk of cardiac morbidity and mortality. For this outcome, Baseline was defined as the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who had Cardiac T2 at Month 24. | Posted | Median | Full Range | milliseconds (msec) | Baseline, Month 24 |
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| Secondary | Change From Baseline in Serum Ferritin in Group C Participants | Serum ferritin is commonly used for an indirect estimation of body iron stores. Although sensitive, it is not specific for iron overload as it can be elevated in a variety of infectious and inflammatory states, and in the presence of cytolysis. For this outcome, Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who achieved serum ferritin count at Baseline and at Months 12 and 24. | Posted | Median | Full Range | nanogram per milliliter (ng/mL) | Baseline, Month 12 and Month 24 |
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| Secondary | Change From Baseline in Liver Iron Concentration (LIC) by Magnetic Resonance Imaging (MRI) in Group C Participants | Liver Iron Concentration of greater than 15 mg per gram dry weight (mg/g) is associated with an increased risk of hepatic disease and cardiac toxicity, whereas levels between 7 to 15 mg/g enhance the risk of hepatic fibrosis and endocrine complications. Liver Iron Concentration of greater than 1.8 mg/g dry weight is considered abnormal; however, organ damage generally does not occur with levels less than 7 mg/g. For this outcome, Baseline was defined as the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who achieved liver iron concentration at Month 24. | Posted | Median | Full Range | milligram per gram (mg/g) | Baseline, Month 24 |
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| Secondary | Change From Baseline in Annualized Volume of pRBC Transfusions in Group C Participants | Change from baseline was calculated as annualized volume of pRBC transfusions in 6 to 24 months post lovo-cel infusion - annualized volume of pRBC transfusions at baseline. Baseline was the annualized volume of pRBC transfusion in the 24 months prior to the Informed Consent. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants with >=1 pRBC Transfusion. | Posted | Median | Full Range | mL/kg/year | From 6 months through 24 months post lovo-cel infusion |
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| Secondary | Change From Baseline in Annualized Number of Packed Red Blood Cell (pRBC) Transfusions in Group C Participants | Change from baseline was calculated as annualized number of pRBC transfusions in 6 to 24 months post lovo-cel infusion - annualized number of pRBC transfusions at baseline. Baseline was the annualized number of pRBC transfusion in the 24 months prior to the Informed Consent. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants with >=1 pRBC Transfusion. | Posted | Median | Full Range | number of pRBC transfusions/year | From 6 months through 24 months post lovo-cel infusion |
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| Secondary | Change From Baseline in Erythropoietin Levels in Group C Participants | Erythropoietin is a hormone produced mainly by the kidneys in response to hypoxia, which can be caused by anemia. Patients with SCD typically produce higher than normal levels of serum erythropoietin. Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who have erythropoietin levels at Baseline and at Months 12 and 24 | Posted | Median | Full Range | units per liter (U/L) | Baseline, Month 12 and Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Cardiac-pulmonary Function Via Left Ventricular Ejection Fraction (LVEF) in Group C Participants | LVEF is the central measure of left ventricular systolic function. LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). This was measured by ECHO. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" refers to the participants who had LVEF at Month 24 | Posted | Median | Full Range | percent | Baseline, Month 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Serum Transferrin Receptor in Group C Participants | Transferrin is a circulating iron carrier protein, delivering iron to cells via the transferrin receptor, and levels of serum transferrin receptor have been used as an indicator of excess iron in the body. Additionally, the transferrin receptor is highly expressed in erythroblasts, and increased levels of the soluble form (serum transferrin receptor) have been shown to be associated with increased levels of erythropoiesis. Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who had serum transferrin receptor count at Baseline and at Months 12 and 24. | Posted | Median | Full Range | milligrams per deciliter (mg/dL) | Baseline, Month 12 and Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Renal Function as Measured by Estimated Glomerular Filtration Rate (eGFR) in Group C Participants | eGFR was calculated by chronic kidney disease epidemiology collaboration (CKD-EPI) formula for participants >=18 years of age and Schwartz formula was used for participants <18 years of age. Baseline is the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "number analyzed" refers to the participants who had renal function at Baseline and at Months 12 and 24. | Posted | Median | Full Range | milliliter/minute/1.73 square meter | Baseline, Month 12 and Month 24 |
| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Shift From Baseline in Cardiac-pulmonary Function Via Echocardiogram (Tricuspid Regurgitant Jet Velocity [TRJV]) in Group C Participants | TRJV over time for each participant was classified into 2 categories: <2.5 m/sec versus >=2.5 m/sec. Higher value of TRJV indicates worse result. A TRJV value of >2.5 m/s was associated with a higher risk of complications, especially in participants with Sickle Cell Disease due to hemolysis and pulmonary hypertension. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" refers to the participants who had shift at baseline for TRJV at Month 24 | Posted | Count of Participants | Participants | Baseline, Month 24 |
| ||||||||||||||||||||||||||||
| Secondary | Number of Participant With Shift From Baseline in Cardiac-pulmonary Function Via Pulmonary Function Tests (PFTs) in Group C Participants | PFTs over time of each participant were classified into one of the five categories: normal, obstructive, restrictive, mixed obstructive and restrictive and isolated low DLco (carbon monoxide diffusing capacity). Only non-zero values are reported here. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. If no records at screening, the last assessment prior to Informed Consent was used. Note: while data are provided for this outcome measure, the results for this outcome are not anticipated to be fully mature until extended participant long-term follow-up in Study LTF-307 has been achieved. The results presented below should be interpreted in that context. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" refers to the participants who had shift from baseline in PFTs at Month 24 | Posted | Count of Participants | Participants | Baseline, Month 24 |
| ||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Meters Walked During 6-minute Walk Test in Group C Participants | The 6-minute walk test measures the distance in meters walked during 6 minutes and can be used to assess pulmonary function. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who were evaluable for this outcome and "number analyzed" refer to the participants walked during 6-minute walk test at Baseline and at Months 12 and 24 | Posted | Median | Full Range | meters | Baseline, Month 12 and Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient-reported Quality of Life as Measured by Patient Reported Outcomes Measurement Information System-57 (PROMIS-57): Domain (T- Score) in Group C Participants | The PROMIS-57 profile was a self-reported questionnaire assessing quality of life in various 7 domains (pain interference, physical function, sleep disturbance, Ability to participate in social roles and activities, anxiety, depression, and fatigue) in participants >=18 years old.These 7 domains are scored on a 5-point Likert scale and converted into standardized T-scores with a mean of 50 and a standard deviation of 10 based on a US general population. The higher of the T-score: the better of physical function, satisfaction with participation in social roles, and ability of participate in social roles in activities; The higher of the T-score: the worse of anxiety, depression, fatigue, sleep disturbance, and pain interference. A negative value indicates improvement, and a positive value indicates no improvement in condition. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who were evaluable for this outcome and "number analyzed" refer to the participants who had domain scores at Month 24. | Posted | Median | Full Range | T-score | Baseline, Month 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient-reported Quality of Life as Measured by PROMIS-57: Pain Intensity Score in Group C Participants | The PROMIS-57 profile was a self-reported questionnaire assessing quality of life in various domains in participants >=18 years old. Pain intensity was scored from 0 to 10, with higher scores indicating greater pain intensity. A negative value indicates improvement in pain intensity and a positive value indicates no improvement or worsening in pain intensity. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who were evaluable for this outcome and "number analyzed" refer to the participants who had pain intensity score at Month 24. | Posted | Median | Full Range | Score on a scale | Baseline, Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient-reported Quality of Life as Measured by Patient Reported Outcomes Measurement Information System- 49 (PROMIS-49)): Domain (T- Score) in Group C Participants | The PROMIS-49 profile was a self-reported questionnaire assessing quality of life in various 6 domains (physical function mobility, peer relationships, pain interference, anxiety, depression, and fatigue) in participants <=18 years old. These 6 domains are scored on a 5-point Likert scale and converted into standardized T-scores with a mean of 50 and a standard deviation of 10 based on a US general population. The higher of the T-score, the better of physical function mobility, and peer relationships; The higher of the T-score, the worse of anxiety, depression symptoms, fatigue, and pain interference. A negative value indicates improvement, and a positive value indicates no improvement in condition. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who had domain scores at Month 24. | Posted | Median | Full Range | T-score | Baseline, Month 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Patient-reported Quality of Life as Measured by PROMIS-49: Pain Intensity Score in Group C Participants | The PROMIS-49 profile was a self-reported questionnaire assessing quality of life in various domains in participants <=18 years old. Pain intensity was scored from 0 to 10, with higher scores indicating greater pain intensity. A negative value indicates improvement in pain intensity and a positive value indicates no improvement or worsening in pain intensity. Baseline was the first assessment on or after Informed Consent but before initiation of stem cell collection. | TP included participants who received lovo-cel. As per planned analysis data was analyzed and reported for TP Group C participants. Here, "Overall number of participants analyzed" signifies participants who had pain intensity scores at Month 24. | Posted | Median | Full Range | Score on a Scale | Baseline, Month 24 |
|
|
From date of Informed Consent signing up to Month 24
ITT population was defined as all participants who initiate any study procedures, beginning with stem cell collection procedures (mobilization/apheresis or bone marrow harvest).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Group A | Participants who had rescue cells that were collected by bone marrow harvest method and had received treatment with lovo-cel which consists of autologous CD34+ HSCs and PSCs (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) collected from participants with SCD by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (the original drug product manufacturing process for this study). | 0 | 9 | 9 | 9 | 9 | 9 |
| EG001 | Group B | Group B included only 2 participants treated with drug product cells collected differently and with drug product manufactured using different processes (one participant in Group B1 and one participant in Group B2 as detailed below). Note that, due to the small size of Groups B1 and B2 and due to other factors (including logistical considerations), these intermediate groups are combined as "Group B" throughout this summary for results reporting purposes. Group B1 participant had rescue cells and drug product cells that were collected by bone marrow harvest method and drug product was manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene. This participant's drug product was produced in 2 lots each using two different manufacturing processes (the original drug product manufacturing process and a refined drug product manufacturing process). Group B2 Plerixafor mobilization and apheresis were used for collection of rescue cells and exploratory manufacturing development. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (using only the refined drug product manufacturing process). | 0 | 2 | 2 | 2 | 2 | 2 |
| EG002 | Group C | Plerixafor mobilization and apheresis were used for collection of rescue cells, and drug product. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by plerixafor mobilization and apheresis transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene using a further refined manufacturing process similar to commercial manufacturing. | 1 | 43 | 33 | 43 | 43 | 43 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Long QT syndrome | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Lung abscess | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Drug dependence | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Face oedema | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Splenic haematoma | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Atrial thrombosis | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Device related thrombosis | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Drug withdrawal syndrome | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Sudden death | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Liver injury | Hepatobiliary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Epiglottitis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Gastroenteritis norovirus | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Infective thrombosis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Sedation complication | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hemiparesis | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperaesthesia | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Psychogenic seizure | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Subarachnoid haemorrhage | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Affective disorder | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Bipolar I disorder | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Substance-induced psychotic disorder | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Catheter bacteraemia | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Costochondritis | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Lymphocytosis | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Neutrophilia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Atrial flutter | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Diplopia | Eye disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Vitreous floaters | Eye disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Vitreous haemorrhage | Eye disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Anal inflammation | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Gastrointestinal inflammation | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Tongue discolouration | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Device related thrombosis | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Puncture site oedema | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Staphylococcal skin infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Anaemia postoperative | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Back injury | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Post procedural fever | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Wrist fracture | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Antiphospholipid antibodies positive | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood magnesium decreased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood oestrogen decreased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Cytomegalovirus test positive | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Troponin T increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Weight increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Synovial cyst | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Device issue | Product Issues | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Nocturia | Renal and urinary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hydrosalpinx | Reproductive system and breast disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Priapism | Reproductive system and breast disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Vaginal discharge | Reproductive system and breast disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Nasal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pharyngeal inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Respiratory acidosis | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dermatitis exfoliative | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pruritus allergic | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Rash vesicular | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Skin hypopigmentation | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Post procedural drainage | Surgical and medical procedures | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Superficial vein prominence | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Atrial thrombosis | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperthyroidism | Endocrine disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Lower respiratory tract infection fungal | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Orchitis | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Skin candida | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypervolaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Carotid artery aneurysm | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pharyngeal haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Embolism | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Tricuspid valve incompetence | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Blood follicle stimulating hormone increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypermagnesaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Premature menopause | Reproductive system and breast disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA version 23.0 | Non-systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Medical Director | bluebird bio, Inc | 339-499-9300 | medinfo@bluebirdbio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 4, 2024 | Jul 24, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C000729928 | lyfgenia |
| D005079 | Excipients |
| ID | Term |
|---|---|
| D014677 | Pharmaceutical Vehicles |
| D010592 | Pharmaceutic Aids |
| D004364 | Pharmaceutical Preparations |
| D020313 | Specialty Uses of Chemicals |
| D020164 | Chemical Actions and Uses |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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