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| ID | Type | Description | Link |
|---|---|---|---|
| C5341019 | Other Identifier | Alias Study Number |
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Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research, and requires further assessment.
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This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
The study will be conducted at approximately 45 sites in the United States.
This registry is an observational study to evaluate the effects of Oxbryta in individuals with SCD. Any participant who is currently taking Oxbryta, or has been prescribed and will initiate treatment with Oxbryta, is eligible to participate. Eligible participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. This study will collect data that are recorded in the participants' medical records and other secondary data sources. Study data will be collected at regular intervals and entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff. Participants will be considered to be on study for up to 5 years after their first dose of Oxbryta treatment, or until they withdraw their consent to participate, or are discontinued from the study. Treatment, including interruptions and restarting treatment, will continue at the discretion of the treating physician, and there are no pre-defined treatment requirements. Participants may receive any additional medications prescribed by their treating physician, or have any medical interventions that are deemed appropriate by the treating physician or study doctor. The participant or treating physician may discontinue Oxbryta at any time. Participants who discontinue treatment with Oxbryta earlier than 5 years will continue to be followed on study to collect clinical and quality of life (QoL) outcomes for up to 5 years after their first dose of Oxbryta treatment. Participant safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Oxbryta Product Registry |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Oxbryta® (voxelotor) 500mg Tablets | Drug | Participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. There are no pre-defined treatment requirements. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. Abbreviations used: overall number of participants analyzed=N and Number analyzed= n | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta |
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| Measure | Description | Time Frame |
|---|---|---|
| Change in Number of Outpatient Visits From Pre-Oxbryta Treatment | Outpatient visits (including infusion center, acute care, or telemedicine visit) were planned to be analyzed. | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Change in Number of Emergency Department (ED) Visits From Pre-Oxbryta Treatment |
Inclusion Criteria:
Participants who meet all the following criteria will be eligible for enrollment:
Exclusion Criteria:
Participants meeting any of the following criteria will not be eligible for study enrollment:
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All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Alabama | Mobile | Alabama | 36617 | United States | ||
| Phoenix Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41701977 | Derived | Andemariam B, Shah N, Ershler WB, Ivy ED, Darbari DS, Anderson A, Rai P, Wun T, Yu CR, Purdie DM, Valenzuela CM, Xu M. Safety and effectiveness of voxelotor in individuals with sickle cell disease in the RETRO and PROSPECT US registries. Blood Adv. 2026 May 12;10(9):3032-3043. doi: 10.1182/bloodadvances.2025018992. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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A total of 265 participants with SCD (all genotypes) aged greater than or equal to (>=) 5 years were enrolled in this study. Data was collected from participants medical records and other secondary data sources such as insurance and pharmacy claims. Participants were followed for up to 5 years after their first dose of Oxbryta treatment (irrespective of how long participants were on Oxbryta when they were enrolled in the study), or until they withdrew consent or were discontinued from study.
The study was conducted at 24 sites in the United States. The study was terminated as emerging clinical data indicated that the risk profile of Oxbryta (voxelotor) in participants with sickle cell disease (SCD) exceeded the benefits observed in previously generated global research and required further assessment. Screen failure was defined as participants who did not start or restart Oxbryta within six months of informed consent.
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| ID | Title | Description |
|---|---|---|
| FG000 | All Participants | Participants with SCD who were treated with Oxbryta in study C5341018 (NCT04930328) or were prescribed Oxbryta by their physician as part of their usual care were included. |
| 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 4, 2024 | Oct 7, 2025 |
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The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. |
| Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Iron Overload as Measured by T2-weighted Magnetic Resonance Imaging (MRI) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) up to 60 months post-Oxbryta |
| Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54 months post-Oxbryta |
| Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Hemoglobinuria was defined as urine dipstick results positive for blood +1 or greater and <=2 red blood cells (RBC) by high power field. Number of participants according to hemoglobinuria results (none/negative, trace, 1+, 2+, 3+, other: large, moderate, small, small=0-3, hereditary persistence of fetal hemoglobin [HPF], missing) was reported in this outcome measure. Small corresponded to trace or 1+ on dipstick, medium indicated 2+, large indicated 3+ or higher on dipstick. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 12, 18, 24, 30 months post-Oxbryta |
| Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Estimated GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. Number of participants according to estimated GFR results (stage 1 [G1] - GFR >=90 milliliter/minute [mL/min] per 1.73 square meters [m^2], stage 2 [G2] - GFR 60 to 89 mL/min per 1.73 m^2, stage 3a [G3a] - GFR 45 to 59 mL/min per 1.73 m^2, stage 3b [G3b] - GFR 30 to 44 mL/min per 1.73 m^2, stage 4 [G4] - GFR 15 to 29 mL/min per 1.73 m^2, stage 5 [G5] - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis, missing) was reported descriptively in this outcome measure. Stages 1 and 2 indicate mild damage, while stages 3 to 5 reflected progressive severity, leading to potential renal failure. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| Number of Participants With SCD Complications | SCD complications included acute pain crisis, acute chest syndrome (ACS), priapism, stroke, chronic or end stage kidney disease iron overload, leg ulcers, cardiac malfunction and pulmonary hypertension (PH) and RBC transfusions. | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Number of Participants With Treatment Initiation or Modification of SCD-related Medications | Number of participants with treatment initiation or modification of SCD related medications is reported in this outcome measure. | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Number of Participants With Red Blood Cell (RBC) Transfusions | Number of participants with RBC transfusions is reported in this outcome measure. | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | PGIC was a single question that reflected a participant's or caregiver's belief about the effectiveness of treatment with Oxbryta on a 7-point scale depicting a participant's rating of overall improvement. Participants/caregivers rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse". | Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 |
| Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | CGIC was a brief, stand-alone assessment of the clinician's view of the participant's global functioning prior to and after initiating Oxbryta. The CGI provided an overall clinician-determined summary measure that took into account all available information, including a knowledge of the participant's history, psychosocial circumstances, symptoms, behavior, and the impact of the symptoms on the participant's ability to function. Participants rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," "very much worse," or "not assessed". | Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 |
| Number of Participants With Serious Adverse Events (SAEs) According to Severity | An SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect and other important medical events. Severity was classified using common terminology criteria for adverse events (CTCAE), version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. Mild - SAEs does not interfere with participant's usual function b) moderate - SAEs interferes to some extent with participant's usual function c) severe - SAEs interferes significantly with participant's usual function. | From date of first Oxbryta treatment up to end of study (up to Month 60) |
| Number of Participants With Adverse Events (AEs) According to Severity | An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which did not necessarily have a causal relationship with this treatment. Severity was classified using CTCAE, version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. AEs are classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function. AEs included both SAEs and non-SAEs. | From date of first Oxbryta treatment up to end of study (up to Month 60) |
| Number of Participants With AEs Leading to Dose Modification or Discontinuation of Oxbryta | An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Number of participants with AEs leading to dose modification or discontinuation of Oxbryta were reported in this outcome measure. | From date of first Oxbryta treatment up to end of study (up to Month 60) |
| Number of Participants With Positive Pregnancy Test and Fertility Complications | Number of participants with positive pregnancy test and fertility complications were reported in this outcome measure. | From date of first Oxbryta treatment up to end of study (up to Month 60) |
ED visits were planned to be analyzed in this outcome measure. |
| From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Change in Number of Hospitalizations From Pre-Oxbryta Treatment | Number of hospitalizations were planned to be analyzed. | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Change in Home Oxygen Supplementation From Pre-Oxbryta Treatment | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Number of Participants With Renal Dialysis | From date of first Oxbryta treatment through the end of study (up to Month 60) |
| Phoenix |
| Arizona |
| 85016 |
| United States |
| University of California, San Diego | La Jolla | California | 92093-0987 | United States |
| Center for Inherited Blood Disorders | Orange | California | 92868 | United States |
| Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital) | Palo Alto | California | 94304 | United States |
| Department of Pediatrics, Hematology section | Palo Alto | California | 94304 | United States |
| Stanford Children's Hospital | Palo Alto | California | 94304 | United States |
| University of Connecticut Health | Farmington | Connecticut | 06030-1163 | United States |
| University of Connecticut Health | Farmington | Connecticut | 06030 | United States |
| Nemours Alfred I duPont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Nemours Children's Health, Wilmington | Wilmington | Delaware | 19803 | United States |
| Foundation for Sickle Cell Disease Research | Hollywood | Florida | 33023 | United States |
| Nemours Children's Specialty Care | Jacksonville | Florida | 32207 | United States |
| University of Miami Hospital | Miami | Florida | 33136 | United States |
| Augusta University - Clinical Trials Office (clinic) | Augusta | Georgia | 30912 | United States |
| Augusta University | Augusta | Georgia | 30912 | United States |
| University of Illinois at Chicago (UIC) Clinical Research Center | Chicago | Illinois | 60612 | United States |
| University of Illinois at Chicago (UIC) Sickle Cell Center | Chicago | Illinois | 60612 | United States |
| University of Illinois Hospital and Health Sciences System(UI Health) | Chicago | Illinois | 60612 | United States |
| University of Illinois Hospital and Health Sciences System | Chicago | Illinois | 60612 | United States |
| University of Maryland Medical Center | College Park | Maryland | 21201 | United States |
| Boston University Medical Center | Boston | Massachusetts | 02118 | United States |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | United States |
| Newark Beth Israel Medical Center | Newark | New Jersey | 07112 | United States |
| Montefiore Medical Center | The Bronx | New York | 10467 | United States |
| Levine Cancer Institute | Charlotte | North Carolina | 28204 | United States |
| Duke University Hospital | Durham | North Carolina | 27710 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| East Carolina University | Greenville | North Carolina | 27834-4300 | United States |
| ECU Health Medical Center Laboratory | Greenville | North Carolina | 27834 | United States |
| ECU Health Medical Center | Greenville | North Carolina | 27834 | United States |
| UPMC Montefiore Hospital | Pittsburgh | Pennsylvania | 14213 | United States |
| UPMC Sickle Cell Center | Pittsburgh | Pennsylvania | 15123 | United States |
| UPMC Presbyterian | Pittsburgh | Pennsylvania | 15213 | United States |
| University of Pittsburgh Medical Center (UPMC) | Pittsburgh | Pennsylvania | 15261 | United States |
| Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
| Children's Blood and Cancer Center at Dell Children's Medical Center | Austin | Texas | 78723 | United States |
| Dell Children's Medical Center | Austin | Texas | 78723 | United States |
| University of Texas Health Science Center at Houston | Houston | Texas | 77030 | United States |
| Inova Schar Cancer Institute | Fairfax | Virginia | 22031 | United States |
| INOVA Health | Falls Church | Virginia | 22042-2325 | United States |
| Treated |
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| COMPLETED |
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| NOT COMPLETED |
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Full analysis set (FAS) included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date).
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| ID | Title | Description |
|---|---|---|
| BG000 | All Participants | Participants with SCD who were treated with Oxbryta in study C5341018 (NCT04930328) or were prescribed Oxbryta by their physician as part of their usual care were included. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | 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 | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | Grams per deciliter (g/dL) | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
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| Primary | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | Percentage of reticulocytes | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
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| Primary | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | 10^9 cells/Liter | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
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| Primary | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. Abbreviations used: overall number of participants analyzed=N and Number analyzed= n | FAS included all participants who met the inclusion/exclusion criteria and signed inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of inform consent date). Here, 'N'=number of participants evaluable for this outcome measure and 'n'=participants evaluable for specified time-points. Indirect bilirubin data for month 54 and 60 was not collected as data collection was not mandatory for laboratory parameters since it is a non-interventional study. | Posted | Mean | Standard Deviation | milligrams per deciliter (mg/dL) | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
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| Primary | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | Micrograms per liter | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
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| Primary | Change in Iron Overload as Measured by T2-weighted Magnetic Resonance Imaging (MRI) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | No data was not collected for this outcome measure as the information regarding iron overload as measured by T2-weighted MRI was not available in the medical record for any of the participants. | Posted | Pre-Oxbryta (baseline) up to 60 months post-Oxbryta |
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| Primary | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | milligrams per deciliter (mg/dL) | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
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| Primary | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | Milligram per gram | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54 months post-Oxbryta |
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| Primary | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Hemoglobinuria was defined as urine dipstick results positive for blood +1 or greater and <=2 red blood cells (RBC) by high power field. Number of participants according to hemoglobinuria results (none/negative, trace, 1+, 2+, 3+, other: large, moderate, small, small=0-3, hereditary persistence of fetal hemoglobin [HPF], missing) was reported in this outcome measure. Small corresponded to trace or 1+ on dipstick, medium indicated 2+, large indicated 3+ or higher on dipstick. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). | Posted | Count of Participants | Participants | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
|
| |||||||||||||||||||||||||||||
| Primary | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Mean | Standard Deviation | milligrams per liter (mg/L) | Pre-Oxbryta (baseline) 3, 6, 12, 18, 24, 30 months post-Oxbryta |
|
| ||||||||||||||||||||||||||||
| Primary | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Estimated GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. Number of participants according to estimated GFR results (stage 1 [G1] - GFR >=90 milliliter/minute [mL/min] per 1.73 square meters [m^2], stage 2 [G2] - GFR 60 to 89 mL/min per 1.73 m^2, stage 3a [G3a] - GFR 45 to 59 mL/min per 1.73 m^2, stage 3b [G3b] - GFR 30 to 44 mL/min per 1.73 m^2, stage 4 [G4] - GFR 15 to 29 mL/min per 1.73 m^2, stage 5 [G5] - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis, missing) was reported descriptively in this outcome measure. Stages 1 and 2 indicate mild damage, while stages 3 to 5 reflected progressive severity, leading to potential renal failure. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). | Posted | Count of Participants | Participants | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta |
| ||||||||||||||||||||||||||||||
| Primary | Number of Participants With SCD Complications | SCD complications included acute pain crisis, acute chest syndrome (ACS), priapism, stroke, chronic or end stage kidney disease iron overload, leg ulcers, cardiac malfunction and pulmonary hypertension (PH) and RBC transfusions. | Safety analysis set (SAS) included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With Treatment Initiation or Modification of SCD-related Medications | Number of participants with treatment initiation or modification of SCD related medications is reported in this outcome measure. | Safety analysis set (SAS) included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With Red Blood Cell (RBC) Transfusions | Number of participants with RBC transfusions is reported in this outcome measure. | SAS included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | PGIC was a single question that reflected a participant's or caregiver's belief about the effectiveness of treatment with Oxbryta on a 7-point scale depicting a participant's rating of overall improvement. Participants/caregivers rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse". | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Count of Participants | Participants | Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | CGIC was a brief, stand-alone assessment of the clinician's view of the participant's global functioning prior to and after initiating Oxbryta. The CGI provided an overall clinician-determined summary measure that took into account all available information, including a knowledge of the participant's history, psychosocial circumstances, symptoms, behavior, and the impact of the symptoms on the participant's ability to function. Participants rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," "very much worse," or "not assessed". | FAS included all participants who met the inclusion/exclusion criteria and signed the inform consent, including screen failures (participants who did not receive Oxbryta within 6 months of the inform consent date). Here, "Overall Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies participants evaluable for specified time-points. | Posted | Count of Participants | Participants | Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 |
| ||||||||||||||||||||||||||||||
| Primary | Number of Participants With Serious Adverse Events (SAEs) According to Severity | An SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect and other important medical events. Severity was classified using common terminology criteria for adverse events (CTCAE), version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. Mild - SAEs does not interfere with participant's usual function b) moderate - SAEs interferes to some extent with participant's usual function c) severe - SAEs interferes significantly with participant's usual function. | SAS included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment up to end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With Adverse Events (AEs) According to Severity | An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which did not necessarily have a causal relationship with this treatment. Severity was classified using CTCAE, version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. AEs are classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function. AEs included both SAEs and non-SAEs. | SAS included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment up to end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With AEs Leading to Dose Modification or Discontinuation of Oxbryta | An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Number of participants with AEs leading to dose modification or discontinuation of Oxbryta were reported in this outcome measure. | SAS included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment up to end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Primary | Number of Participants With Positive Pregnancy Test and Fertility Complications | Number of participants with positive pregnancy test and fertility complications were reported in this outcome measure. | SAS included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study. | Posted | Count of Participants | Participants | From date of first Oxbryta treatment up to end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | Change in Number of Outpatient Visits From Pre-Oxbryta Treatment | Outpatient visits (including infusion center, acute care, or telemedicine visit) were planned to be analyzed. | Data for this outcome measure was not collected due to premature termination of the study. Only limited efficacy data to support the benefit-risk evaluation of Oxbryta was collected. | Posted | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Change in Number of Emergency Department (ED) Visits From Pre-Oxbryta Treatment | ED visits were planned to be analyzed in this outcome measure. | Data for this outcome measure was not collected due to premature termination of the study. Only limited efficacy data to support the benefit-risk evaluation of Oxbryta was collected. | Posted | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Change in Number of Hospitalizations From Pre-Oxbryta Treatment | Number of hospitalizations were planned to be analyzed. | Data for this outcome measure was not collected due to premature termination of the study. Only limited efficacy data to support the benefit-risk evaluation of Oxbryta was collected. | Posted | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Change in Home Oxygen Supplementation From Pre-Oxbryta Treatment | Data for this outcome measure was not collected due to premature termination of the study. Only limited efficacy data to support the benefit-risk evaluation of Oxbryta was collected. | Posted | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
| ||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Participants With Renal Dialysis | Data for this outcome measure was not collected due to premature termination of the study. Only limited efficacy data to support the benefit-risk evaluation of Oxbryta was collected. | Posted | From date of first Oxbryta treatment through the end of study (up to Month 60) |
|
|
From date of first Oxbryta treatment up to end of study (up to Month 60)
Same event may occur as both non-SAE and SAE but are distinct events. An event may be categorized as serious in 1 participant and non-serious in another, or a participant may have experienced both SAE and non-SAE. SAS included all participants in the FAS who received at least one dose of Oxbryta including participants who had received at least one dose of Oxbryta in C5341018 (NCT04930328) (RETRO) study.
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 | All Participants | Participants with SCD who were treated with Oxbryta in study C5341018 (NCT04930328) or were prescribed Oxbryta by their physician as part of their usual care were included. | 6 | 260 | 150 | 260 | 185 | 260 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Hypersplenism | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Acute coronary syndrome | Cardiac disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pulmonary valve incompetence | Cardiac disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia | Congenital, familial and genetic disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Goitre | Endocrine disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Tooth impacted | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Sudden death | General disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Systemic inflammatory response syndrome | General disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Liver disorder | Hepatobiliary disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Abdominal wall abscess | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Corynebacterium bacteraemia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Mycobacterial infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Delayed haemolytic transfusion reaction | Injury, poisoning and procedural complications | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA 27.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Epstein-Barr virus test positive | Investigations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA 27.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Joint effusion | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Middle cerebral artery stroke | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Psychogenic seizure | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Brachiocephalic artery occlusion | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Jugular vein occlusion | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Peripheral vascular disorder | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Subclavian vein occlusion | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
| |
| Vena cava thrombosis | Vascular disorders | MedDRA 27.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Atrial thrombosis | Cardiac disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Mitral valve incompetence | Cardiac disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Sickle cell anaemia | Congenital, familial and genetic disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Ophthalmic artery aneurysm | Eye disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Retinopathy | Eye disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Pancreatitis chronic | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Drug ineffective | General disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA27.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA27.0 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA27.0 | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA27.0 | Non-systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA27.0 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA27.0 | Non-systematic Assessment |
| |
| Blood bilirubin unconjugated increased | Investigations | MedDRA27.0 | Non-systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA27.0 | Non-systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Intracranial aneurysm | Nervous system disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| End stage renal disease | Renal and urinary disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Priapism | Reproductive system and breast disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA27.0 | Non-systematic Assessment |
| |
| Superficial vein thrombosis | Vascular disorders | MedDRA27.0 | Non-systematic Assessment |
|
For outcome measure 9, the terminology used for other: small, medium, and large were entered by sites to describe the severity or amount but is not clinically precise and should be interpreted cautiously.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov.Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 31, 2025 | Oct 7, 2025 | 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 |
|---|---|
| C000628792 | voxelotor |
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 |
|
|
| 9 months Post-Oxbryta |
|
|
| 12 months Post-Oxbryta |
|
|
| 18 months Post-Oxbryta |
|
|
| 24 months Post-Oxbryta |
|
|
| 30 months Post-Oxbryta |
|
|
| 36 months Post-Oxbryta |
|
|
| 42 months Post-Oxbryta |
|
|
| 48 months Post-Oxbryta |
|
|
| 54 months Post-Oxbryta |
|
|
| 60 months Post-Oxbryta |
|
|
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
|
|
|
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
|
| 3+ |
|
| Other: Large |
|
| Other: Moderate |
|
| Other: Small |
|
| Other: Small- 0-3 HPF |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| G3b - GFR 30 to 44 mL/min per 1.73 m^2 |
|
| G4 - GFR 15 to 29 mL/min per 1.73 m^2 |
|
| G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis |
|
| Missing |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|
| No change |
|
| Minimally worse |
|
| Much worse |
|
| Very much worse |
|
| Not assessed |
|