Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LentiGlobin BB305 Drug Product | Experimental | LentiGlobin BB305 Drug Product (autologous CD34+ cell-enriched population that contains cells transduced with LentiGlobin BB305 lentiviral vector encoding human βA-T87Q-globin) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LentiGlobin BB305 Drug Product | Genetic | LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Have Achieved Transfusion Independence (TI) | TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion. | From 12 to 24 months post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Have Achieved Transfusion Independence (TI) at Month 24 | TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion. | At Month 24 post-transplant |
Not provided
Inclusion Criteria:
• Participants less than or equal to (<=) 50 years of age at the time of consent or assent (as applicable), and able to provide written consent (adults, or legal guardians, as applicable) or assent (adolescents or children). Provided that the data monitoring committee (DMC) has approved enrolling participants younger than 5 years of age, participants younger than 5 years of age may be enrolled if they weigh a minimum of 6 kilograms (kg) and are reasonably anticipated to be able to provide at least the minimum number of cells required to initiate the manufacturing process.
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Himal L Thakar, MD | Genetix Biotherapeutics Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | United States | ||
| Ann & Robert H. Lurie Children's Hospital of Chicago |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39527960 | Derived | Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA. Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype beta-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial. Lancet. 2024 Nov 30;404(10468):2175-2186. doi: 10.1016/S0140-6736(24)01884-1. Epub 2024 Nov 8. |
Not provided
Not provided
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 completion of this study, submission of all applicable regulatory submissions and consistent with criteria established by bluebird bio and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
Not provided
Not provided
Not provided
Not provided
A total of 19 participants were enrolled, of which 18 participants aged <=50 years were treated with LentiGlobin BB305 Drug Product.
The study was conducted at 9 study centers in France, Germany, Greece, Italy, United Kingdom, and United States, of which 8 active centers had enrolled participants from 08 June 2017 to 15 November 2022.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | LentiGlobin BB305 Drug Product | Participants <=50 years of age received a single intravenous (IV) infusion of LentiGlobin BB305 Drug Product at a dose of >=5.0 × 10^6 CD34+ cells/kilogram (kg) body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 10, 2021 | Nov 3, 2023 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Duration of Transfusion Independence (TI) | Duration of TI was calculated as the time from the start of TI (i.e. first Hb >=9 with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met using Kaplan-Meier methodology. | From start of TI up to Month 24 (actual maximum time frame of up to approximately 25 months due to visit window) |
| Time From Drug Product Infusion to Achievement of Transfusion Independence (TI) | Time from drug product infusion to achievement of TI was calculated as the time from drug product infusion to the first hemoglobin at which a participant can be declared as TI (that is to 'start of TI + >= 12 months', dependent on Hb lab schedule). | From drug product infusion to start of TI (up to Month 24 [actual maximum time frame of up to approximately 25 months due to visit window]) |
| Weighted Average Hemoglobin (Hb) During Transfusion Independence (TI) | Weighted average Hb was defined as the weighted average of Hb values without any pRBC transfusions in the proceeding 60 days for a given subject. Ratio of the time between two Hb values and the time between the first and the last Hb values was used as the weight for calculation. | Timeframe varied by subject. For a given subject, the endpoint was calculated from 60 days after the last pRBC transfusion (so transfused blood did not confound the weighted average calculation) through the Month 24 Visit for that subject. |
| Percentage of Participants Who Meet the Definition of Transfusion Reduction (TR) | TR was defined as demonstration of a 60 percent (%) reduction in the annualized volume of pRBC transfusion requirements (in milliliter per kilogram [mL/kg]) in the post-treatment time period from 12 Months post-drug product infusion through Month 24 compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to study enrollment. | From 12 to 24 months post-transplant |
| Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume | Percentage of participants with a reduction in the annualized mL/kg pRBCs transfused from 12 months post-drug product infusion through Month 24 (approximately a 12-month period) of at least 50%, 60%, 75%, 90% or 100% compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to enrollment. | 12 months post-drug product infusion through Month 24 |
| Annualized Number of pRBC Transfusions | Annualized number of pRBC transfusions from 12 months post-drug product infusion through Month 24 was reported. | From 12 months post-drug product infusion through Month 24 |
| Annualized Volume of pRBC Transfusions | Annualized volume of pRBC transfusions from 12 months post-drug product infusion through Month 24 compared to the annualized volume of transfusions during the 24 months prior to enrollment. | From 12 to 24 months post-transplant |
| Time From Drug Product Infusion to Last pRBC Transfusion | Time from drug product infusion to last pRBC transfusion was reported. | From start of drug product infusion up to Month 24 |
| Time From Last pRBC Transfusion to 24 Months | Time From Last pRBC Transfusion to the Month 24 was reported. | From last pRBC Transfusion up to Month 24 (actual maximum time frame of up to approximately 27 months due to visit window) |
| Weighted Average Nadir Hemoglobin (Hb) | The weighted average nadir Hb was defined as the most recent Hb prior to each pRBC transfusion, on the day of transfusion or within 3 days and, if there was a period of more than 60 days without transfusion, all Hb records between Day 61 and last follow-up or next transfusion (inclusive) was included. The weighted average nadir Hb during the period of 12 months post-drug product infusion to Month 24 was compared to the weighted average nadir Hb during the 24 months prior to enrollment. | 12 months post-drug product infusion through Month 24 |
| Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 | Unsupported total Hb level was defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date. | At Month 6, 9, 12, 18 and 24 |
| Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 | Number of participants with unsupported total Hb levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) meeting the thresholds were reported at Months 6, 9, 12, 18 and 24. Participants were evaluable if they had an unsupported total Hb measurement at the specific timepoint, where unsupported total Hb level is defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date. | At Months 6, 9, 12, 18 and 24 |
| Percentage of Participants Who Have Not Received Chelation Therapy for At Least 6 Months Following Drug Product Infusion | Percentage of participants who have not received chelation therapy for at least 6 months following drug product infusion were reported. | From 6 to 24 months |
| Time From Last Iron Chelation Use to Last Follow-up | Time from last iron chelation use to last follow-up to 24 months was reported. | From last Iron Chelation up to Month 24 (actual maximum time frame of up to approximately 29 months due to visit window) |
| Number of Participants Who Used Therapeutic Phlebotomy Post Drug Product Infusion | Therapeutic phlebotomy could be used in lieu of chelation in participants who had Hb consistently >= 11 g/dL and who were no longer receiving regular transfusions, at the discretion of the investigator. Number of participants who used therapeutic phlebotomy post Drug Product infusion for up to Month 24 were reported. | From drug product infusion through Month 24 |
| Annualized Phlebotomy Therapy Usage Following Drug Product Infusion | Annualized phlebotomy therapy usage (number of procedures per year, calculated from DP infusion through last follow-up) were reported. | From drug product infusion through Month 24 |
| Change From Baseline in Liver Iron Content by Magnetic Resonance Imaging (MRI) | Change From Baseline in Liver Iron Content by MRI at Months 12 and 24 were reported. Baseline is defined as value closest to but prior to conditioning. | Baseline, Month 12 and 24 |
| Change From Baseline in Cardiac T2* on MRI | Change From Baseline in Cardiac T2* on MRI from Baseline, Month 12 and 24 was reported. | Baseline, Months 12 and 24 |
| Change From Baseline in Serum Ferritin | Serum ferritin was 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. Change from baseline in serum ferritin at Months 12 and 24 was reported. | Baseline, Month 12 and 24 |
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 | PedsQL GCS designed to measure health-related quality of life in pediatric and adolescents (2-18 years). It encompassed 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), Teens (13-18 years). The questionnaire was also completed by parent/caregiver to assess parents' perceptions of their children's quality of life. The Toddler group consisted of 21 items, using a 5-point Likert scale (0 to 4); all other groups consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for young pediatric, a 5-point Likert scale for pediatric and teens groups. All reported scores were transformed on a scale from 0 to 100 for each domain where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores correspond with higher quality of life. | Baseline, Month 12 and 24 |
| Change From Baseline in EuroQol Quality of Life 5-Dimension Youth Scale (EQ-5D-Y) VAS Health Status at Months 12 and 24 | EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life questionnaire in cost effectiveness and health technologies assessment. EQ-5D-Y was a version of instrument specifically developed and validated for use by youths aged 12 through 17 years. The EQ-5D-Y visual analog scale (VAS) consisted of a 20-cm vertical VAS, with anchors of 0 (worst imaginable health state) and 100 (best imaginable health state). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Higher scores corresponded with higher quality of life. | Baseline, Months 12 and 24 |
| Change From Baseline in EuroQol Quality of Life 5-Dimension Adult Scale (EQ-5D-3L) VAS Heath Status Score at Months 12 and 24 | EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life (HRQoL) questionnaire in cost effectiveness and health technologies assessment. Participants age >=18 at time of informed consent were eligible to complete the EQ-5D-3L visual analog scale (VAS) which consisted of a 20-cm vertical VAS, with anchors of 0 (worst imaginable health state) and 100 (best imaginable health state). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Negative change in score indicated decrease in quality of life (as measured by EQ5D VAS) from baseline. | Baseline, Months 12 and 24 |
| Change From Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Total Score | FACT-BMT is assessed bone marrow transplant related quality of life in adults. Total score was sum of sub-scale scores for 5 domains: Physical Well-Being, Social/Family Well-Being, Emotional Well-Being, Functional Well-Being, and Bone Marrow Transplantation Subscale. Each item scored on a 5-point Likert scale based on participant agreement with each statement: 0 for "not at all," 1 for "a little bit," 2 for "somewhat," 3 for "quite a bit," and 4 for "very much. Reported scores were transformed as follows: After taking into account reverse scores for questions constructed in negative form, subscale score for each domain was calculated by multiplying sum of item scores by number of items in subscale, then dividing by number of items answered. Total score was sum of subscale total added together and ranges from 0-148. Higher scores corresponded with higher quality of life. | Baseline, Months 12 and 24 |
| Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 | SF-36 was designed to measure health-related quality of life in adults. The instrument consisted of 36 items that were aggregated into 8 multi-item scales (physical functioning [1=yes, limited a lot, to 3=no, not limited at all], role-physical [1=all of time, to 5=none of time], bodily pain [1=very severe, to 6=none], general health [1=poor, to 5=excellent], vitality [1=none of time, to 5=all of time], social functioning [1=all of time, to 5=none of time], role emotional [1=all of time, to 5=none of time] and mental health [1=all of time, to 5=none of the time]). The 8 scales were summarized as Physical Component Summary (PCS) score (physical functioning, role-physical, bodily pain, general health scales) and Mental Component Summary (MCS) score (vitality, social functioning, role-emotional, mental health scales). Reported summary scores were transformed on a scale from 0-100 (higher scores corresponded with higher quality of life), with change from baseline results being presented. | Baseline, Months 12 and 24 |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Hopital d'enfants de la Timone | Marseille | 13385 | France |
| Hannover Medical School | Hanover | 30625 | Germany |
| University of Heidelberg | Heidelberg | 69120 | Germany |
| General Hospital of Thessaloniki 'G.Papanikolaou' | Thessaloniki | Greece |
| IRCCS Ospedale Pediatrico Babino Gesu | Rome | Italy |
| University College London Hospital | London | United Kingdom |
| Intent-to-Treat (ITT) Population | ITT population included all 19 participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor. |
|
| Transplant Population (TP) | TP included all participants who received beti-cel. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Data are analyzed at times based on ITT population which included all 19 participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | LentiGlobin BB305 Drug Product | Participants <=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of >=5.0 × 10^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants Who Have Achieved Transfusion Independence (TI) | TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion. | TP included all participants who received beti-cel. Participant evaluable for TI are defined as participant who have achieved TI, have not achieved TI in their parent study, or completed their parent study. | Posted | Number | 95% Confidence Interval | Percentage of participants | From 12 to 24 months post-transplant |
|
|
| |||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Have Achieved Transfusion Independence (TI) at Month 24 | TI was defined as a weighted average hemoglobin (Hb) >= 9 grams per deciliter (g/dL) without any packed red blood cell (pRBC) transfusions for a continuous period of >= 12 months at any time during the study after drug product infusion. | TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. | Posted | Number | 95% Confidence Interval | Percentage of participants | At Month 24 post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Duration of Transfusion Independence (TI) | Duration of TI was calculated as the time from the start of TI (i.e. first Hb >=9 with no transfusions in the preceding 60 days) up to the last available Hb at which the TI criteria are still met using Kaplan-Meier methodology. | TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who achieved TI. Data are presented through the Month 24 Visit based on calendar dates and including visit windows. | Posted | Median | Full Range | Months | From start of TI up to Month 24 (actual maximum time frame of up to approximately 25 months due to visit window) |
|
| ||||||||||||||||||||||||||
| Secondary | Time From Drug Product Infusion to Achievement of Transfusion Independence (TI) | Time from drug product infusion to achievement of TI was calculated as the time from drug product infusion to the first hemoglobin at which a participant can be declared as TI (that is to 'start of TI + >= 12 months', dependent on Hb lab schedule). | TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who achieved TI. Data are presented through the Month 24 Visit based on calendar dates and including visit windows. | Posted | Median | Full Range | Months | From drug product infusion to start of TI (up to Month 24 [actual maximum time frame of up to approximately 25 months due to visit window]) |
|
| ||||||||||||||||||||||||||
| Secondary | Weighted Average Hemoglobin (Hb) During Transfusion Independence (TI) | Weighted average Hb was defined as the weighted average of Hb values without any pRBC transfusions in the proceeding 60 days for a given subject. Ratio of the time between two Hb values and the time between the first and the last Hb values was used as the weight for calculation. | TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who achieved TI. | Posted | Mean | Standard Deviation | gram per deciliter (g/dL) | Timeframe varied by subject. For a given subject, the endpoint was calculated from 60 days after the last pRBC transfusion (so transfused blood did not confound the weighted average calculation) through the Month 24 Visit for that subject. |
|
| ||||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Meet the Definition of Transfusion Reduction (TR) | TR was defined as demonstration of a 60 percent (%) reduction in the annualized volume of pRBC transfusion requirements (in milliliter per kilogram [mL/kg]) in the post-treatment time period from 12 Months post-drug product infusion through Month 24 compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to study enrollment. | TP included all participants who received beti-cel. Participants evaluable for TI are defined as participants who have achieved TI, have not achieved TI in their parent study, or completed their parent study. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome. | Posted | Number | 95% Confidence Interval | Percentage of participants | From 12 to 24 months post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Had a Reduction of At Least 50%, 60%, 75%, 90% or 100% in the Annualized pRBCs Transfusion Volume | Percentage of participants with a reduction in the annualized mL/kg pRBCs transfused from 12 months post-drug product infusion through Month 24 (approximately a 12-month period) of at least 50%, 60%, 75%, 90% or 100% compared to the annualized mL/kg pRBC transfusion requirement during the 24 months prior to enrollment. | TP included all participants who received beti-cel. | Posted | Number | 95% Confidence Interval | Percentage of participants | 12 months post-drug product infusion through Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Annualized Number of pRBC Transfusions | Annualized number of pRBC transfusions from 12 months post-drug product infusion through Month 24 was reported. | TP included all participants who received beti-cel. | Posted | Mean | Standard Deviation | pRBC transfusions per year | From 12 months post-drug product infusion through Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Annualized Volume of pRBC Transfusions | Annualized volume of pRBC transfusions from 12 months post-drug product infusion through Month 24 compared to the annualized volume of transfusions during the 24 months prior to enrollment. | TP included all participants who received beti-cel. | Posted | Mean | Standard Deviation | milliliter/kilogram/year (mL/kg/year) | From 12 to 24 months post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Time From Drug Product Infusion to Last pRBC Transfusion | Time from drug product infusion to last pRBC transfusion was reported. | TP included all participants who received beti-cel. | Posted | Median | Full Range | months | From start of drug product infusion up to Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Time From Last pRBC Transfusion to 24 Months | Time From Last pRBC Transfusion to the Month 24 was reported. | TP included all participants who received beti-cel. Data are presented through the Month 24 Visit based on calendar dates and including visit windows. | Posted | Median | Full Range | Months | From last pRBC Transfusion up to Month 24 (actual maximum time frame of up to approximately 27 months due to visit window) |
|
| ||||||||||||||||||||||||||
| Secondary | Weighted Average Nadir Hemoglobin (Hb) | The weighted average nadir Hb was defined as the most recent Hb prior to each pRBC transfusion, on the day of transfusion or within 3 days and, if there was a period of more than 60 days without transfusion, all Hb records between Day 61 and last follow-up or next transfusion (inclusive) was included. The weighted average nadir Hb during the period of 12 months post-drug product infusion to Month 24 was compared to the weighted average nadir Hb during the 24 months prior to enrollment. | TP included all participant who received beti-cel. | Posted | Mean | Standard Deviation | g/dL | 12 months post-drug product infusion through Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Unsupported Total Hb Levels at Month 6, 9, 12, 18 and 24 | Unsupported total Hb level was defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date. | TP included all participant who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who had a least 1 unsupported total Hb measurement during Study HGB-212 and "number analyzed" signifies those participants who had an unsupported total Hb measurement at the specific timepoint. | Posted | Mean | Standard Deviation | g/dL | At Month 6, 9, 12, 18 and 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants With Unsupported Total Hb Levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) at Months 6, 9, 12, 18 and 24 | Number of participants with unsupported total Hb levels (>=10 g/dL, >=11 g/dL, >=12 g/dL, >=13 g/dL, and >=14 g/dL) meeting the thresholds were reported at Months 6, 9, 12, 18 and 24. Participants were evaluable if they had an unsupported total Hb measurement at the specific timepoint, where unsupported total Hb level is defined as the total Hb measurement level without any acute or chronic pRBC transfusions within 60 days prior to the measurement date. | TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who had a least 1 unsupported total Hb measurement during Study HGB-212 and "number analyzed" signifies those participants who had an unsupported total Hb measurement at the specific timepoint. | Posted | Count of Participants | Participants | At Months 6, 9, 12, 18 and 24 |
|
| |||||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Have Not Received Chelation Therapy for At Least 6 Months Following Drug Product Infusion | Percentage of participants who have not received chelation therapy for at least 6 months following drug product infusion were reported. | TP included all participant who received beti-cel. | Posted | Number | Percentage of participants | From 6 to 24 months |
|
| |||||||||||||||||||||||||||
| Secondary | Time From Last Iron Chelation Use to Last Follow-up | Time from last iron chelation use to last follow-up to 24 months was reported. | TP included all participants who received beti-cel. Participants were evaluable for this endpoint if they had not received iron chelation therapy for at least 6 months following drug product infusion. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome. Data are presented through the Month 24 Visit based on calendar dates and including visit windows. | Posted | Median | Full Range | Months | From last Iron Chelation up to Month 24 (actual maximum time frame of up to approximately 29 months due to visit window) |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants Who Used Therapeutic Phlebotomy Post Drug Product Infusion | Therapeutic phlebotomy could be used in lieu of chelation in participants who had Hb consistently >= 11 g/dL and who were no longer receiving regular transfusions, at the discretion of the investigator. Number of participants who used therapeutic phlebotomy post Drug Product infusion for up to Month 24 were reported. | TP included all participants who received beti-cel. | Posted | Count of Participants | Participants | From drug product infusion through Month 24 |
|
| |||||||||||||||||||||||||||
| Secondary | Annualized Phlebotomy Therapy Usage Following Drug Product Infusion | Annualized phlebotomy therapy usage (number of procedures per year, calculated from DP infusion through last follow-up) were reported. | TP included all participants who received beti-cel. Here, "Overall number of participants analyzed" signifies those participants who received therapeutic phlebotomy. | Posted | Mean | Standard Deviation | Number of procedures per year | From drug product infusion through Month 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Liver Iron Content by Magnetic Resonance Imaging (MRI) | Change From Baseline in Liver Iron Content by MRI at Months 12 and 24 were reported. Baseline is defined as value closest to but prior to conditioning. | TP included all participants who received beti-cel. Participants were evaluable for TI if they had completed the study (i.e., completed the Month 24 Visit), achieved TI, or did not achieve TI during the study. Here, "number analyzed" signifies those participants who had LIC data available at Baseline, Months 12 and 24. | Posted | Mean | Standard Deviation | milligram per gram (mg/g) | Baseline, Month 12 and 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Cardiac T2* on MRI | Change From Baseline in Cardiac T2* on MRI from Baseline, Month 12 and 24 was reported. | TP included all participants who received beti-cel. Participants were evaluable for TI if they had completed the study (i.e., completed the Month 24 Visit), achieved TI, or did not achieve TI during the study. Here, "number analyzed" signifies those participants who were evaluable at specific timepoint. | Posted | Median | Full Range | milliseconds | Baseline, Months 12 and 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Serum Ferritin | Serum ferritin was 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. Change from baseline in serum ferritin at Months 12 and 24 was reported. | TP included all participants who received beti-cel. Participants are evaluable for TI if they had completed the study (i.e., completed the Month 24 Visit), achieved TI, or did not achieve TI during the study. Here, "number analyzed" signifies those participants who were evaluable at specific timepoint. | Posted | Mean | Standard Deviation | picomole per liter (pmol/L) | Baseline, Month 12 and 24 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Scores at Months 12 and 24 | PedsQL GCS designed to measure health-related quality of life in pediatric and adolescents (2-18 years). It encompassed 4 dimensions of functioning (physical [8 items], emotional [5 items], social [5 items], school [3 items]). Age groups: Toddler (2-4 years), Young pediatric (5-7 years), Pediatric (8-12 years), Teens (13-18 years). The questionnaire was also completed by parent/caregiver to assess parents' perceptions of their children's quality of life. The Toddler group consisted of 21 items, using a 5-point Likert scale (0 to 4); all other groups consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for young pediatric, a 5-point Likert scale for pediatric and teens groups. All reported scores were transformed on a scale from 0 to 100 for each domain where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores correspond with higher quality of life. | TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome and "number analyzed" signifies those participants who were evaluable at specific timepoint. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Month 12 and 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in EuroQol Quality of Life 5-Dimension Youth Scale (EQ-5D-Y) VAS Health Status at Months 12 and 24 | EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life questionnaire in cost effectiveness and health technologies assessment. EQ-5D-Y was a version of instrument specifically developed and validated for use by youths aged 12 through 17 years. The EQ-5D-Y visual analog scale (VAS) consisted of a 20-cm vertical VAS, with anchors of 0 (worst imaginable health state) and 100 (best imaginable health state). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Higher scores corresponded with higher quality of life. | TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Months 12 and 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in EuroQol Quality of Life 5-Dimension Adult Scale (EQ-5D-3L) VAS Heath Status Score at Months 12 and 24 | EQ-5D is a validated, standardized, generic instrument that was most widely used preference based health related quality of life (HRQoL) questionnaire in cost effectiveness and health technologies assessment. Participants age >=18 at time of informed consent were eligible to complete the EQ-5D-3L visual analog scale (VAS) which consisted of a 20-cm vertical VAS, with anchors of 0 (worst imaginable health state) and 100 (best imaginable health state). Respondents were asked to rate their own health state today by drawing a line from a box containing these words to the point on the scale that they felt most accurately reflected their current health state. The VAS was reported (raw data) on a scale of 0-100 where 0= death and 100= perfect health. Negative change in score indicated decrease in quality of life (as measured by EQ5D VAS) from baseline. | TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Months 12 and 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Questionnaire Total Score | FACT-BMT is assessed bone marrow transplant related quality of life in adults. Total score was sum of sub-scale scores for 5 domains: Physical Well-Being, Social/Family Well-Being, Emotional Well-Being, Functional Well-Being, and Bone Marrow Transplantation Subscale. Each item scored on a 5-point Likert scale based on participant agreement with each statement: 0 for "not at all," 1 for "a little bit," 2 for "somewhat," 3 for "quite a bit," and 4 for "very much. Reported scores were transformed as follows: After taking into account reverse scores for questions constructed in negative form, subscale score for each domain was calculated by multiplying sum of item scores by number of items in subscale, then dividing by number of items answered. Total score was sum of subscale total added together and ranges from 0-148. Higher scores corresponded with higher quality of life. | TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Months 12 and 24 |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Short Form-36 Health Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores) at Months 12 and 24 | SF-36 was designed to measure health-related quality of life in adults. The instrument consisted of 36 items that were aggregated into 8 multi-item scales (physical functioning [1=yes, limited a lot, to 3=no, not limited at all], role-physical [1=all of time, to 5=none of time], bodily pain [1=very severe, to 6=none], general health [1=poor, to 5=excellent], vitality [1=none of time, to 5=all of time], social functioning [1=all of time, to 5=none of time], role emotional [1=all of time, to 5=none of time] and mental health [1=all of time, to 5=none of the time]). The 8 scales were summarized as Physical Component Summary (PCS) score (physical functioning, role-physical, bodily pain, general health scales) and Mental Component Summary (MCS) score (vitality, social functioning, role-emotional, mental health scales). Reported summary scores were transformed on a scale from 0-100 (higher scores corresponded with higher quality of life), with change from baseline results being presented. | TP included all participants who received beti-cel. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Months 12 and 24 |
|
From date of informed consent to the date of last followup (an average of 32.5 months of followup for ITT subjects).
ITT population included all participants who initiated any study procedures, beginning with mobilization by G-CSF and/or plerixafor.
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 | LentiGlobin BB305 Drug Product | Participants <=50 years of age received a single IV infusion of LentiGlobin BB305 Drug Product at a dose of >=5.0 × 10^6 CD34+ cells/kg body weight on Day 1 after myeloablative conditioning with busulfan (4 days of conditioning followed by at least 48 hours of washout) (termed the Transplant population). | 0 | 19 | 6 | 19 | 19 | 19 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac failure congestive | Cardiac disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Gastric ulcer perforation | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Bartholin's cyst | Reproductive system and breast disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Soft tissue infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Focal nodular hyperplasia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Adverse drug reaction | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Application site pain | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Application site swelling | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Feeling cold | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Mucosal inflammation | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Puncture site pain | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Laryngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pharyngeal inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Blood testosterone decreased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Coombs direct test positive | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
| |
| Incision site pain | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
| |
| Post procedural discomfort | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA (23.0) | Non-systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Ageusia | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Neutrophilia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Chalazion | Eye disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Anal inflammation | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Lip dry | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Palatal ulcer | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hepatic mass | Hepatobiliary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hidradenitis | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Intertrigo | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Skin hypopigmentation | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Delayed puberty | Endocrine disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypogonadism | Endocrine disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Secondary hypogonadism | Endocrine disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Lice infestation | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Mucosal infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Systemic infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Varicella zoster virus infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Vulvovaginal candidiasis | Infections and infestations | MedDRA (23.0) | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hyperchloraemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
| |
| Vitamin K deficiency | Metabolism and nutrition disorders | MedDRA (23.0) | Non-systematic Assessment |
|
Limitations of the trial such as small numbers of participants analysed or technical problems leading to unreliable data.
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Medical Director | bluebird bio, Inc | +1-833-999-6378 | clinicaltrials@bluebirdbio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 28, 2022 | Nov 3, 2023 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| 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 |
|---|---|
| C000721148 | betibeglogene autotemcel |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Participants |
|
|
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|