Sickle Cell Disease
Conditions
Keywords
Registry Sickle Cell Disease
Brief summary
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
Detailed description
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.
Interventions
DRUGOxbryta® (voxelotor) 500mg Tablets
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.
Sponsors
Pfizer
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
4 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants who meet all the following criteria will be eligible for enrollment: 1. Willing and able to provide written informed consent (aged ≥ 18 years), parental/ guardian consent and participant assent (aged ≥ 12 to \<18 years) per local regulations, or pediatric participants (aged 4 to \<12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines 2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes) 3. Undergoing treatment with Oxbryta according to the Oxbryta USPI
Exclusion criteria
* Participants meeting any of the following criteria will not be eligible for study enrollment: 1. Current participation in an investigation clinical trial or expanded access program, in which the participant may be receiving voxelotor treatment. 2. Medical, psychological, or behavioral condition that, in the opinion of the study doctor, would confound or interfere with evaluation of safety and/or effectiveness of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Positive Pregnancy Test and Fertility Complications | 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 were reported in this outcome measure. |
| Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 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. |
| Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 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. |
| Number of Participants With Serious Adverse Events (SAEs) According to Severity | From date of first Oxbryta treatment up to end of study (up to Month 60) | 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. |
| Number of Participants With Adverse Events (AEs) According to Severity | From date of first Oxbryta treatment up to end of study (up to Month 60) | 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. |
| Number of Participants With AEs Leading to Dose Modification or Discontinuation of Oxbryta | From date of first Oxbryta treatment up to end of study (up to Month 60) | 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. |
| Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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 |
| Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Change in Iron Overload as Measured by T2-weighted Magnetic Resonance Imaging (MRI) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) up to 60 months 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. |
| Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54 months 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. |
| Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 12, 18, 24, 30 months 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. |
| Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months 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. |
| Number of Participants With SCD Complications | From date of first Oxbryta treatment through the end of study (up to Month 60) | 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. |
| Number of Participants With Treatment Initiation or Modification of SCD-related Medications | 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 is reported in this outcome measure. |
| Number of Participants With Red Blood Cell (RBC) Transfusions | From date of first Oxbryta treatment through the end of study (up to Month 60) | Number of participants with RBC transfusions is reported in this outcome measure. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change in Number of Emergency Department (ED) Visits From Pre-Oxbryta Treatment | From date of first Oxbryta treatment through the end of study (up to Month 60) | ED visits were planned to be analyzed in this outcome measure. |
| Change in Number of Hospitalizations From Pre-Oxbryta Treatment | From date of first Oxbryta treatment through the end of study (up to Month 60) | Number of hospitalizations were planned to be analyzed. |
| 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) | — |
| Change in Number of Outpatient Visits From Pre-Oxbryta Treatment | From date of first Oxbryta treatment through the end of study (up to Month 60) | Outpatient visits (including infusion center, acute care, or telemedicine visit) were planned to be analyzed. |
Countries
United States
Participant flow
Recruitment details
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.
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 265 |
| Total | 265 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 7 |
| Overall Study | Lack of Efficacy | 1 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Other | 2 |
| Overall Study | Physician Decision | 1 |
| Overall Study | Screen Failure | 8 |
| Overall Study | Study Terminated by Sponsor | 245 |
Baseline characteristics
| Characteristic | All Participants |
|---|---|
| Age, Continuous | 32.0 Years STANDARD_DEVIATION 14.78 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 21 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 240 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 246 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 11 Participants |
| Race (NIH/OMB) White | 6 Participants |
| Sex: Female, Male Female | 154 Participants |
| Sex: Female, Male Male | 111 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 6 / 260 |
| other Total, other adverse events | 185 / 260 |
| serious Total, serious adverse events | 150 / 260 |
Outcome results
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta | -15.6 10^9 cells/Liter | Standard Deviation 188.54 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta | 1.5 10^9 cells/Liter | Standard Deviation 134.05 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta | -4.1 10^9 cells/Liter | Standard Deviation 137.59 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta | 2.4 10^9 cells/Liter | Standard Deviation 130.76 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta | -17.8 10^9 cells/Liter | Standard Deviation 175.27 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta | 31.4 10^9 cells/Liter | Standard Deviation 242.25 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta | 0.1 10^9 cells/Liter | Standard Deviation 134.67 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta | -15.3 10^9 cells/Liter | Standard Deviation 228.15 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta | 2.7 10^9 cells/Liter | Standard Deviation 142.52 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta | 4.4 10^9 cells/Liter | Standard Deviation 119.59 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta | 3.3 10^9 cells/Liter | Standard Deviation 140.03 |
| All Participants | Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta | 21.7 10^9 cells/Liter | Standard Deviation 93.55 |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 30 months Post-Oxbryta | 80.3 Milligram per gram | Standard Deviation 164.48 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 3 months Post-Oxbryta | 67.5 Milligram per gram | Standard Deviation 451.66 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 6 months Post-Oxbryta | -42.1 Milligram per gram | Standard Deviation 137.7 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 9 months Post-Oxbryta | 59.6 Milligram per gram | Standard Deviation 129.88 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 12 months Post-Oxbryta | -32.1 Milligram per gram | Standard Deviation 158.67 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 18 months Post-Oxbryta | 125.2 Milligram per gram | Standard Deviation 330.7 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 24 months Post-Oxbryta | 7.7 Milligram per gram | Standard Deviation 178.23 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 36 months Post-Oxbryta | 14.4 Milligram per gram | Standard Deviation 130.58 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 42 months Post-Oxbryta | 38.2 Milligram per gram | Standard Deviation 96.87 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 48 months Post-Oxbryta | 273.3 Milligram per gram | Standard Deviation 599.99 |
| All Participants | Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta | 54 months Post-Oxbryta | 873.3 Milligram per gram | Standard Deviation 1488.6 |
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
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta: Direct | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 1.22 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta: Direct | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.62 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta: Direct | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 0.57 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta: Direct | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.46 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta: Direct | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 0.35 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta: Direct | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 1.57 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta: Direct | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 1.01 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta: Direct | 0.2 milligrams per deciliter (mg/dL) | Standard Deviation 0.81 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta: Direct | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.31 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta: Direct | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.37 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta: Direct | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.28 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta: Direct | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.37 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta: Indirect | -0.4 milligrams per deciliter (mg/dL) | Standard Deviation 1.19 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta: Indirect | -0.3 milligrams per deciliter (mg/dL) | Standard Deviation 1.28 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta: Indirect | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 2.23 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta: Indirect | -0.5 milligrams per deciliter (mg/dL) | Standard Deviation 1.22 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta: Indirect | 0.4 milligrams per deciliter (mg/dL) | Standard Deviation 2.64 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta: Indirect | 0.2 milligrams per deciliter (mg/dL) | Standard Deviation 1.23 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta: Indirect | 0.2 milligrams per deciliter (mg/dL) | Standard Deviation 1.4 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta: Indirect | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 0.97 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta: Indirect | -0.4 milligrams per deciliter (mg/dL) | Standard Deviation 0.78 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta: Indirect | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 1.45 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta: Total | -0.5 milligrams per deciliter (mg/dL) | Standard Deviation 1.71 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta: Total | -0.5 milligrams per deciliter (mg/dL) | Standard Deviation 1.97 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta: Total | -0.2 milligrams per deciliter (mg/dL) | Standard Deviation 2.76 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta: Total | -0.4 milligrams per deciliter (mg/dL) | Standard Deviation 2.66 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta: Total | -0.5 milligrams per deciliter (mg/dL) | Standard Deviation 2.24 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta: Total | -0.4 milligrams per deciliter (mg/dL) | Standard Deviation 2.27 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta: Total | -0.3 milligrams per deciliter (mg/dL) | Standard Deviation 2.1 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta: Total | -0.5 milligrams per deciliter (mg/dL) | Standard Deviation 2.48 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta: Total | -0.5 milligrams per deciliter (mg/dL) | Standard Deviation 2.9 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta: Total | -0.8 milligrams per deciliter (mg/dL) | Standard Deviation 2.59 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta: Total | -0.6 milligrams per deciliter (mg/dL) | Standard Deviation 2.56 |
| All Participants | Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta: Total | -0.1 milligrams per deciliter (mg/dL) | Standard Deviation 1.33 |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta | 0.5 Grams per deciliter (g/dL) | Standard Deviation 1.33 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta | 0.6 Grams per deciliter (g/dL) | Standard Deviation 1.47 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta | 0.5 Grams per deciliter (g/dL) | Standard Deviation 1.45 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta | 0.6 Grams per deciliter (g/dL) | Standard Deviation 1.36 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta | 0.7 Grams per deciliter (g/dL) | Standard Deviation 1.35 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta | 0.6 Grams per deciliter (g/dL) | Standard Deviation 1.46 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta | 0.5 Grams per deciliter (g/dL) | Standard Deviation 1.62 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta | 0.6 Grams per deciliter (g/dL) | Standard Deviation 1.62 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta | 0.4 Grams per deciliter (g/dL) | Standard Deviation 1.48 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta | 0.7 Grams per deciliter (g/dL) | Standard Deviation 1.47 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta | 0.1 Grams per deciliter (g/dL) | Standard Deviation 1.68 |
| All Participants | Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta | -0.2 Grams per deciliter (g/dL) | Standard Deviation 1.36 |
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.
Time frame: Pre-Oxbryta (baseline) up to 60 months post-Oxbryta
Population: 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.
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta | 74.0 Micrograms per liter | Standard Deviation 1696.32 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta | 105.2 Micrograms per liter | Standard Deviation 1995.71 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta | -60.7 Micrograms per liter | Standard Deviation 2200.7 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta | 136.9 Micrograms per liter | Standard Deviation 1614.56 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta | 148.0 Micrograms per liter | Standard Deviation 1973.6 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta | 94.0 Micrograms per liter | Standard Deviation 2111.95 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta | -248.1 Micrograms per liter | Standard Deviation 1761.49 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta | -220.5 Micrograms per liter | Standard Deviation 1765.71 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta | 667.5 Micrograms per liter | Standard Deviation 2577.61 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta | 896.7 Micrograms per liter | Standard Deviation 3142.23 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta | 1672.8 Micrograms per liter | Standard Deviation 3975.5 |
| All Participants | Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta | 4512.5 Micrograms per liter | Standard Deviation 5500.9 |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta | -0.9 Percentage of reticulocytes | Standard Deviation 5.5 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta | -1.1 Percentage of reticulocytes | Standard Deviation 6.01 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta | -1.1 Percentage of reticulocytes | Standard Deviation 5.97 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta | -1.2 Percentage of reticulocytes | Standard Deviation 6.34 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta | -1.6 Percentage of reticulocytes | Standard Deviation 6.69 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta | -0.7 Percentage of reticulocytes | Standard Deviation 6.32 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta | -1.0 Percentage of reticulocytes | Standard Deviation 6.92 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta | -1.3 Percentage of reticulocytes | Standard Deviation 7.04 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta | -0.2 Percentage of reticulocytes | Standard Deviation 7.45 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta | 0.2 Percentage of reticulocytes | Standard Deviation 6.7 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta | -0.3 Percentage of reticulocytes | Standard Deviation 6.59 |
| All Participants | Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta | 1.9 Percentage of reticulocytes | Standard Deviation 3.84 |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 9 months Post-Oxbryta | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.6 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 3 months Post-Oxbryta | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 0.37 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 6 months Post-Oxbryta | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 0.46 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 12 months Post-Oxbryta | 0.0 milligrams per deciliter (mg/dL) | Standard Deviation 0.37 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 18 months Post-Oxbryta | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.43 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 24 months Post-Oxbryta | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.51 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 30 months Post-Oxbryta | 0.2 milligrams per deciliter (mg/dL) | Standard Deviation 0.6 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 36 months Post-Oxbryta | 0.2 milligrams per deciliter (mg/dL) | Standard Deviation 0.92 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 42 months Post-Oxbryta | 0.3 milligrams per deciliter (mg/dL) | Standard Deviation 1.04 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 48 months Post-Oxbryta | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.52 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 54 months Post-Oxbryta | 0.4 milligrams per deciliter (mg/dL) | Standard Deviation 1.17 |
| All Participants | Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | 60 months Post-Oxbryta | 0.1 milligrams per deciliter (mg/dL) | Standard Deviation 0.25 |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 12, 18, 24, 30 months post-Oxbryta
Population: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| All Participants | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | 30 months Post-Oxbryta | -0.1 milligrams per liter (mg/L) | Standard Deviation 0.07 |
| All Participants | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | 3 months Post-Oxbryta | 0.1 milligrams per liter (mg/L) | — |
| All Participants | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | 6 months Post-Oxbryta | -0.1 milligrams per liter (mg/L) | Standard Deviation 0.07 |
| All Participants | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | 12 months Post-Oxbryta | 0.2 milligrams per liter (mg/L) | Standard Deviation 0.07 |
| All Participants | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | 18 months Post-Oxbryta | -0.2 milligrams per liter (mg/L) | Standard Deviation 0.07 |
| All Participants | Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta | 24 months Post-Oxbryta | 0.0 milligrams per liter (mg/L) | Standard Deviation 0.11 |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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).
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 63 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 24 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 47 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 21 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 8 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | Pre-Oxbryta | Missing | 164 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 4 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 3 months Post-Oxbryta | Missing | 189 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 51 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 17 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 6 months Post-Oxbryta | Missing | 187 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 44 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 21 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 9 months Post-Oxbryta | Missing | 193 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 50 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 22 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 12 months Post-Oxbryta | Missing | 186 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 60 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 29 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 5 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 18 months Post-Oxbryta | Missing | 165 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 55 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 22 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 24 months Post-Oxbryta | Missing | 180 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 53 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 21 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 4 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 30 months Post-Oxbryta | Missing | 184 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 39 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 15 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 4 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 36 months Post-Oxbryta | Missing | 203 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 42 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 12 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 5 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 6 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 42 months Post-Oxbryta | Missing | 202 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 33 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 10 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 4 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 5 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 48 months Post-Oxbryta | Missing | 212 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 23 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 3 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 2 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 54 months Post-Oxbryta | Missing | 228 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | G1 - GFR >=90 mL/min per 1.73 m^2 | 7 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | G2 - GFR 60 to 89 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | G3a - GFR 45 to 59 mL/min per 1.73 m^2 | 1 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | G3b - GFR 30 to 44 mL/min per 1.73 m^2 | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | G4 - GFR 15 to 29 mL/min per 1.73 m^2 | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | G5 - GFR < 15 mL/min per 1.73 m^2 or treatment by dialysis | 0 Participants |
| All Participants | Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta | 60 months Post-Oxbryta | Missing | 256 Participants |
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.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Population: 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).
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | Trace | 3 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | 1+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | Missing | 254 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | Trace | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | None/Negative | 18 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | 2+ | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | Other: Moderate | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | Trace | 4 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | 1+ | 3 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | Missing | 239 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | 1+ | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | Missing | 246 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | None/Negative | 13 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | Trace | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | 1+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | Trace | 4 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | 1+ | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | Other: Small | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | None/Negative | 22 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | Trace | 7 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | 1+ | 3 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | 2+ | 6 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | Other: Large | 3 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | Other: Moderate | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | Pre-Oxbryta | Missing | 223 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | None/Negative | 7 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | Trace | 4 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | 1+ | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | Other: Small | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 3 months Post-Oxbryta | Missing | 251 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | None/Negative | 12 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | Trace | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | 1+ | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | 2+ | 3 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | Other: Moderate | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 6 months Post-Oxbryta | Missing | 247 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 9 months Post-Oxbryta | None/Negative | 8 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | None/Negative | 11 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | 1+ | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | Other: Small | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 12 months Post-Oxbryta | Missing | 250 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | Trace | 6 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | 1+ | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 18 months Post-Oxbryta | Missing | 236 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | None/Negative | 17 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 24 months Post-Oxbryta | Other: Small | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | None/Negative | 16 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | Trace | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 30 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | 3+ | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | Other: Large | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 36 months Post-Oxbryta | Missing | 247 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | None/Negative | 12 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | Other: Large | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | Other: Small | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | Other: Small- 0-3 HPF | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 42 months Post-Oxbryta | Missing | 245 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | None/Negative | 12 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | Trace | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | 1+ | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | 2+ | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | 3+ | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 48 months Post-Oxbryta | Missing | 247 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | None/Negative | 8 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | Trace | 2 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | 1+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | Other: Large | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 54 months Post-Oxbryta | Missing | 253 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | None/Negative | 1 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | Trace | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | 1+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | 2+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | 3+ | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | Other: Large | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | Other: Moderate | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | Other: Small | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | Other: Small- 0-3 HPF | 0 Participants |
| All Participants | Number of Participants According to Hemoglobinuria Results Post-Oxbryta | 60 months Post-Oxbryta | Missing | 264 Participants |
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.
Time frame: Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60
Population: 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.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Much improved | 12 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Not assessed | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Very much improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Much improved | 7 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Minimally improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | No change | 9 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 18 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Very much improved | 3 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Much improved | 13 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Minimally improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | No change | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Minimally worse | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 24 | Not assessed | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Very much improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Much improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Minimally improved | 9 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | No change | 5 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 30 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Much improved | 8 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Minimally improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | No change | 4 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Very much worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 36 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Very much improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Minimally improved | 10 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | No change | 8 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 42 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Very much improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Minimally improved | 10 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | No change | 6 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 48 | Not assessed | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Very much improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Much improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Minimally improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | No change | 4 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 54 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Minimally improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | No change | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 60 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Minimally improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | No change | 2 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 3 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Minimally improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | No change | 5 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 6 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Very much improved | 3 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Much improved | 4 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Minimally improved | 7 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | No change | 5 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 9 | Not assessed | 0 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Much improved | 4 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | Minimally improved | 7 Participants |
| All Participants | Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60 | Month 12 | No change | 7 Participants |
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.
Time frame: Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60
Population: 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.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | Much worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | Much improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | Minimally improved | 8 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | Very much improved | 4 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | Much improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | Minimally improved | 3 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | No change | 4 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 3 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | Very much improved | 4 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | Much improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | Minimally improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | No change | 5 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 6 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | Very much improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | No change | 10 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 9 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | Very much improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | Much improved | 12 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | Minimally improved | 8 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | No change | 15 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 12 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | Very much improved | 4 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | Much improved | 17 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | Minimally improved | 11 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | No change | 7 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | Minimally worse | 2 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 18 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | Very much improved | 3 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | Much improved | 14 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | Minimally improved | 9 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | No change | 13 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | Much worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 24 | Very much worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | Very much improved | 6 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | Much improved | 12 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | Minimally improved | 11 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | No change | 11 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | Much worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 30 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | Much improved | 12 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | Minimally improved | 16 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | No change | 5 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | Minimally worse | 2 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 36 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | Very much improved | 8 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | Much improved | 11 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | Minimally improved | 17 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | No change | 11 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 42 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | Very much improved | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | Much improved | 17 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | Minimally improved | 9 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | No change | 7 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | Minimally worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 48 | Very much worse | 1 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | Very much improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | Much improved | 5 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | Minimally improved | 3 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | No change | 6 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 54 | Very much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | Very much improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | Much improved | 2 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | Minimally improved | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | No change | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | Minimally worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | Much worse | 0 Participants |
| All Participants | Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60 | Month 60 | Very much worse | 0 Participants |
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.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Population: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Participants | Number of Participants With Adverse Events (AEs) According to Severity | Mild | 28 Participants |
| All Participants | Number of Participants With Adverse Events (AEs) According to Severity | Moderate | 83 Participants |
| All Participants | Number of Participants With Adverse Events (AEs) According to Severity | Severe | 99 Participants |
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.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Population: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| All Participants | Number of Participants With AEs Leading to Dose Modification or Discontinuation of Oxbryta | 7 Participants |
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.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Population: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| All Participants | Number of Participants With Positive Pregnancy Test and Fertility Complications | 7 Participants |
Primary
Number of Participants With Red Blood Cell (RBC) Transfusions
Number of participants with RBC transfusions is reported in this outcome measure.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| All Participants | Number of Participants With Red Blood Cell (RBC) Transfusions | 172 Participants |
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.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| All Participants | Number of Participants With SCD Complications | 205 Participants |
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.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Population: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| All Participants | Number of Participants With Serious Adverse Events (SAEs) According to Severity | Severe | 87 Participants |
| All Participants | Number of Participants With Serious Adverse Events (SAEs) According to Severity | Mild | 8 Participants |
| All Participants | Number of Participants With Serious Adverse Events (SAEs) According to Severity | Moderate | 55 Participants |
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.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| All Participants | Number of Participants With Treatment Initiation or Modification of SCD-related Medications | 259 Participants |
Other Pre-specified
Change in Home Oxygen Supplementation From Pre-Oxbryta Treatment
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
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.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
Other Pre-specified
Change in Number of Hospitalizations From Pre-Oxbryta Treatment
Number of hospitalizations were planned to be analyzed.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
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.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.
Other Pre-specified
Number of Participants With Renal Dialysis
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Population: 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.