Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD

NCT04930445 | Terminated Results FDA Drug

• 2 other identifiers: GBT440-4R2C5341019
• Study Type: observational
• Target: 265
• Locations: 1 country
• Sites: 43

Brief Summary

This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.

Conditions

Sickle Cell Disease

Keywords

Registry Sickle Cell Disease

Outcome Measures

Primary Outcomes (20)

• Change in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Percent Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Absolute Reticulocyte Count From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Bilirubin (Total, Direct and Indirect) Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used. Abbreviations used: overall number of participants analyzed=N and Number analyzed= n

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Iron Overload (Ferritin Levels) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Iron Overload as Measured by T2-weighted Magnetic Resonance Imaging (MRI) From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) up to 60 months post-Oxbryta

• Change in Serum Creatinine Level From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Albumin Creatinine Ratio (ACR) From Pre-Oxbryta Treatment (Baseline) Through Month 54 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54 months post-Oxbryta

• Number of Participants According to Hemoglobinuria Results Post-Oxbryta

  Hemoglobinuria was defined as urine dipstick results positive for blood +1 or greater and \<=2 red blood cells (RBC) by high power field. Number of participants according to hemoglobinuria results (none/negative, trace, 1+, 2+, 3+, other: large, moderate, small, small=0-3, hereditary persistence of fetal hemoglobin \[HPF\], missing) was reported in this outcome measure. Small corresponded to trace or 1+ on dipstick, medium indicated 2+, large indicated 3+ or higher on dipstick. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Change in Serum Cystatin C Results From Pre-Oxbryta Treatment (Baseline) Through Month 30 Post-Oxbryta

  The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 12, 18, 24, 30 months post-Oxbryta

• Number of Participants According to Estimated Glomerular Filtration Rate (GFR) Results Post-Oxbryta

  Estimated GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. Number of participants according to estimated GFR results (stage 1 \[G1\] - GFR \>=90 milliliter/minute \[mL/min\] per 1.73 square meters \[m\^2\], stage 2 \[G2\] - GFR 60 to 89 mL/min per 1.73 m\^2, stage 3a \[G3a\] - GFR 45 to 59 mL/min per 1.73 m\^2, stage 3b \[G3b\] - GFR 30 to 44 mL/min per 1.73 m\^2, stage 4 \[G4\] - GFR 15 to 29 mL/min per 1.73 m\^2, stage 5 \[G5\] - GFR \< 15 mL/min per 1.73 m\^2 or treatment by dialysis, missing) was reported descriptively in this outcome measure. Stages 1 and 2 indicate mild damage, while stages 3 to 5 reflected progressive severity, leading to potential renal failure. The pre-Oxbryta (baseline) value was the latest measurement on or prior to the first dose of Oxbryta. Post-Oxbryta value was the measurement for multiple assessments at the same specific visit, the average was used.

  Pre-Oxbryta (baseline) 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60 months post-Oxbryta

• Number of Participants With SCD Complications

  SCD complications included acute pain crisis, acute chest syndrome (ACS), priapism, stroke, chronic or end stage kidney disease iron overload, leg ulcers, cardiac malfunction and pulmonary hypertension (PH) and RBC transfusions.

  From date of first Oxbryta treatment through the end of study (up to Month 60)

• Number of Participants With Treatment Initiation or Modification of SCD-related Medications

  Number of participants with treatment initiation or modification of SCD related medications is reported in this outcome measure.

  From date of first Oxbryta treatment through the end of study (up to Month 60)

• Number of Participants With Red Blood Cell (RBC) Transfusions

  Number of participants with RBC transfusions is reported in this outcome measure.

  From date of first Oxbryta treatment through the end of study (up to Month 60)

• Number of Participants According to Responses to Patient Global Impression of Change (PGIC) at Month 3 Through Month 60

  PGIC was a single question that reflected a participant's or caregiver's belief about the effectiveness of treatment with Oxbryta on a 7-point scale depicting a participant's rating of overall improvement. Participants/caregivers rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse".

  Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60

• Number of Participants According to Responses to Clinical Global Impression of Change (CGIC) at Month 3 Through Month 60

  CGIC was a brief, stand-alone assessment of the clinician's view of the participant's global functioning prior to and after initiating Oxbryta. The CGI provided an overall clinician-determined summary measure that took into account all available information, including a knowledge of the participant's history, psychosocial circumstances, symptoms, behavior, and the impact of the symptoms on the participant's ability to function. Participants rated their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," "very much worse," or "not assessed".

  Month 3, 6, 9, 12, 18, 24, 30, 36, 42, 48, 54, 60

• Number of Participants With Serious Adverse Events (SAEs) According to Severity

  An SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the following criteria - resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect and other important medical events. Severity was classified using common terminology criteria for adverse events (CTCAE), version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. Mild - SAEs does not interfere with participant's usual function b) moderate - SAEs interferes to some extent with participant's usual function c) severe - SAEs interferes significantly with participant's usual function.

  From date of first Oxbryta treatment up to end of study (up to Month 60)

• Number of Participants With Adverse Events (AEs) According to Severity

  An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which did not necessarily have a causal relationship with this treatment. Severity was classified using CTCAE, version 5.0, where Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe. AEs are classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function. AEs included both SAEs and non-SAEs.

  From date of first Oxbryta treatment up to end of study (up to Month 60)

• Number of Participants With AEs Leading to Dose Modification or Discontinuation of Oxbryta

  An AE was any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Number of participants with AEs leading to dose modification or discontinuation of Oxbryta were reported in this outcome measure.

  From date of first Oxbryta treatment up to end of study (up to Month 60)

• Number of Participants With Positive Pregnancy Test and Fertility Complications

  Number of participants with positive pregnancy test and fertility complications were reported in this outcome measure.

  From date of first Oxbryta treatment up to end of study (up to Month 60)

Other Outcomes (5)

• 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)

• 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)

• Change in Number of Hospitalizations From Pre-Oxbryta Treatment

  From date of first Oxbryta treatment through the end of study (up to Month 60)

Study Arms (1)

Oxbryta Product Registry

Drug: Oxbryta® (voxelotor) 500mg Tablets

Interventions

Oxbryta® (voxelotor) 500mg Tablets DRUG

Participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. There are no pre-defined treatment requirements.

Also known as: Voxelotor, Oxbryta®

Oxbryta Product Registry

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.

You may qualify if:

• Participants who meet all the following criteria will be eligible for enrollment:
• 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
• Male or female participants with documented diagnosis of sickle cell disease (all genotypes)
• Undergoing treatment with Oxbryta according to the Oxbryta USPI

You may not qualify if:

• Participants meeting any of the following criteria will not be eligible for study enrollment:
• Current participation in an investigation clinical trial or expanded access program, in which the participant may be receiving voxelotor treatment.
• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (43)

University of South Alabama

Mobile, Alabama, 36617, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

University of California, San Diego

La Jolla, California, 92093-0987, United States

Location

Center for Inherited Blood Disorders

Orange, California, 92868, United States

Location

Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital)

Palo Alto, California, 94304, United States

Location

Department of Pediatrics, Hematology section

Palo Alto, California, 94304, United States

Location

Stanford Children's Hospital

Palo Alto, California, 94304, United States

Location

University of Connecticut Health

Farmington, Connecticut, 06030-1163, United States

Location

University of Connecticut Health

Farmington, Connecticut, 06030, United States

Location

Nemours Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Nemours Children's Health, Wilmington

Wilmington, Delaware, 19803, United States

Location

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33023, United States

Location

Nemours Children's Specialty Care

Jacksonville, Florida, 32207, United States

Location

University of Miami Hospital

Miami, Florida, 33136, United States

Location

Augusta University - Clinical Trials Office (clinic)

Augusta, Georgia, 30912, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

University of Illinois at Chicago (UIC) Clinical Research Center

Chicago, Illinois, 60612, United States

Location

University of Illinois at Chicago (UIC) Sickle Cell Center

Chicago, Illinois, 60612, United States

Location

University of Illinois Hospital and Health Sciences System(UI Health)

Chicago, Illinois, 60612, United States

Location

University of Illinois Hospital and Health Sciences System

Chicago, Illinois, 60612, United States

Location

University of Maryland Medical Center

College Park, Maryland, 21201, United States

Location

Boston University Medical Center

Boston, Massachusetts, 02118, United States

Location

Mississippi Center for Advanced Medicine

Madison, Mississippi, 39110, United States

Location

Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

Levine Cancer Institute

Charlotte, North Carolina, 28204, United States

Location

Duke University Hospital

Durham, North Carolina, 27710, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

East Carolina University

Greenville, North Carolina, 27834-4300, United States

Location

ECU Health Medical Center Laboratory

Greenville, North Carolina, 27834, United States

Location

ECU Health Medical Center

Greenville, North Carolina, 27834, United States

Location

UPMC Montefiore Hospital

Pittsburgh, Pennsylvania, 14213, United States

Location

UPMC Sickle Cell Center

Pittsburgh, Pennsylvania, 15123, United States

Location

UPMC Presbyterian

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, 15261, United States

Location

Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital

Charleston, South Carolina, 29425, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Children's Blood and Cancer Center at Dell Children's Medical Center

Austin, Texas, 78723, United States

Location

Dell Children's Medical Center

Austin, Texas, 78723, United States

Location

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Inova Schar Cancer Institute

Fairfax, Virginia, 22031, United States

Location

INOVA Health

Falls Church, Virginia, 22042-2325, United States

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

For outcome measure 9, the terminology used for other: small, medium, and large were entered by sites to describe the severity or amount but is not clinically precise and should be interpreted cautiously.

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov.Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 4, 2021
• First Posted: June 18, 2021
• Study Start: February 4, 2022
• Primary Completion: October 10, 2024
• Study Completion: October 10, 2024
• Last Updated: December 31, 2025
• Results First Posted: December 31, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (43)