This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
The study will be conducted at approximately 45 sites in the United States. This registry is an observational study to evaluate the effects of Oxbryta in individuals with SCD. Any participant who is currently taking Oxbryta, or has been prescribed and will initiate treatment with Oxbryta, is eligible to participate. Eligible participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. This study will collect data that are recorded in the participants' medical records and other secondary data sources. Study data will be collected at regular intervals and entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff. Participants will be considered to be on study for up to 5 years after their first dose of Oxbryta treatment, or until they withdraw their consent to participate, or are discontinued from the study. Treatment, including interruptions and restarting treatment, will continue at the discretion of the treating physician, and there are no pre-defined treatment requirements. Participants may receive any additional medications prescribed by their treating physician, or have any medical interventions that are deemed appropriate by the treating physician or study doctor. The participant or treating physician may discontinue Oxbryta at any time. Participants who discontinue treatment with Oxbryta earlier than 5 years will continue to be followed on study to collect clinical and quality of life (QoL) outcomes for up to 5 years after their first dose of Oxbryta treatment. Participant safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.
Study Type
OBSERVATIONAL
Enrollment
265
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.
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
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
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
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
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.
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University of South Alabama
Mobile, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
University of California, San Diego
La Jolla, California, United States
Center for Inherited Blood Disorders
Orange, California, United States
Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital)
Palo Alto, California, United States
Department of Pediatrics, Hematology section
Palo Alto, California, United States
Stanford Children's Hospital
Palo Alto, California, United States
University of Connecticut Health
Farmington, Connecticut, United States
University of Connecticut Health
Farmington, Connecticut, United States
Nemours Alfred I duPont Hospital for Children
Wilmington, Delaware, United States
...and 33 more locations
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Change in Iron Overload as Measured by T2-weighted Magnetic Resonance Imaging (MRI) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta
The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.
Time frame: Pre-Oxbryta (baseline) up to 60 months post-Oxbryta
Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta
The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta
The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54 months post-Oxbryta
Number of Participants According to Hemoglobinuria Results Post-Oxbryta
Hemoglobinuria was defined as urine dipstick results positive for blood +1 or greater and \<=2 red blood cells (RBC) by high power field. Number of participants according to hemoglobinuria results (none/negative, trace, 1+, 2+, 3+, other: large, moderate, small, small=0-3, hereditary persistence of fetal hemoglobin \[HPF\], missing) was reported in this outcome measure. Small corresponded to trace or 1+ on dipstick, medium indicated 2+, large indicated 3+ or higher on dipstick. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta
The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.
Time frame: Pre-Oxbryta (baseline) 3, 6, 12, 18, 24, 30 months post-Oxbryta
Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta
Estimated GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. Number of participants according to estimated GFR results (stage 1 \[G1\] - GFR \>=90 milliliter/minute \[mL/min\] per 1.73 square meters \[m\^2\], stage 2 \[G2\] - GFR 60 to 89 mL/min per 1.73 m\^2, stage 3a \[G3a\] - GFR 45 to 59 mL/min per 1.73 m\^2, stage 3b \[G3b\] - GFR 30 to 44 mL/min per 1.73 m\^2, stage 4 \[G4\] - GFR 15 to 29 mL/min per 1.73 m\^2, stage 5 \[G5\] - GFR \< 15 mL/min per 1.73 m\^2 or treatment by dialysis, missing) was reported descriptively in this outcome measure. Stages 1 and 2 indicate mild damage, while stages 3 to 5 reflected progressive severity, leading to potential renal failure. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.
Time frame: Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta
Number of Participants With SCD Complications
SCD complications included acute pain crisis, acute chest syndrome (ACS), priapism, stroke, chronic or end stage kidney disease iron overload, leg ulcers, cardiac malfunction and pulmonary hypertension (PH) and RBC transfusions.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Number of Participants With Treatment Initiation or Modification of SCD-related Medications
Number of participants with treatment initiation or modification of SCD related medications is reported in this outcome measure.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Number of Participants With Red Blood Cell (RBC) Transfusions
Number of participants with RBC transfusions is reported in this outcome measure.
Time frame: From date of first Oxbryta treatment through the end of study (up to Month 60)
Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60
PGIC was a single question that reflected a participant's or caregiver's belief about the effectiveness of treatment with Oxbryta on a 7-point scale depicting a participant's rating of overall improvement. Participants/caregivers rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse".
Time frame: Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60
Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60
CGIC was a brief, stand-alone assessment of the clinician's view of the participant's global functioning prior to and after initiating Oxbryta. The CGI provided an overall clinician-determined summary measure that took into account all available information, including a knowledge of the participant's history, psychosocial circumstances, symptoms, behavior, and the impact of the symptoms on the participant's ability to function. Participants rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," "very much worse," or "not assessed".
Time frame: Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60
Number of Participants With Serious Adverse Events (SAEs) According to Severity
An SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect and other important medical events. Severity was classified using common terminology criteria for adverse events (CTCAE), version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. Mild - SAEs does not interfere with participant's usual function b) moderate - SAEs interferes to some extent with participant's usual function c) severe - SAEs interferes significantly with participant's usual function.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Number of Participants With Adverse Events (AEs) According to Severity
An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which did not necessarily have a causal relationship with this treatment. Severity was classified using CTCAE, version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. AEs are classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function. AEs included both SAEs and non-SAEs.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Number of Participants With AEs Leading to Dose Modification or Discontinuation of Oxbryta
An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Number of participants with AEs leading to dose modification or discontinuation of Oxbryta were reported in this outcome measure.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)
Number of Participants With Positive Pregnancy Test and Fertility Complications
Number of participants with positive pregnancy test and fertility complications were reported in this outcome measure.
Time frame: From date of first Oxbryta treatment up to end of study (up to Month 60)