Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Sickle cell drug oxbryta tracked in real life: what happened?

NCT ID NCT04930445

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 265 people with sickle cell disease who were taking Oxbryta (voxelotor) as part of their normal care. Researchers wanted to see how the drug affected hemoglobin levels, sickle cell complications, and overall health over up to 5 years. The study was observational, meaning patients were not assigned to a specific treatment plan—they just took Oxbryta as their doctor prescribed. Unfortunately, the study was terminated early, so the full planned data was not collected.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Oxbryta (voxelotor) tablets
What this could lead to
If successful, this registry could confirm that Oxbryta helps manage sickle cell disease in everyday settings, improving hemoglobin levels and reducing complications.
What could go wrong
The study was terminated early, so long-term data is limited. As an observational registry, it cannot prove cause and effect, and results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

265 people

The number who actually took part.

Started

Feb 2022

Finished

Oct 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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

Ages

4 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Sickle cell disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

Contacts and locations

Locations

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Augusta University - Clinical Trials Office (clinic)

    Augusta, Georgia, 30912, United States

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

    Palo Alto, California, 94304, United States

  • Boston University Medical Center

    Boston, Massachusetts, 02118, United States

  • Center for Inherited Blood Disorders

    Orange, California, 92868, United States

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

    Austin, Texas, 78723, United States

  • Dell Children's Medical Center

    Austin, Texas, 78723, United States

  • Department of Pediatrics, Hematology section

    Palo Alto, California, 94304, United States

  • Duke University Hospital

    Durham, North Carolina, 27710, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • ECU Health Medical Center

    Greenville, North Carolina, 27834, United States

  • ECU Health Medical Center Laboratory

    Greenville, North Carolina, 27834, United States

  • East Carolina University

    Greenville, North Carolina, 27834-4300, United States

  • Foundation for Sickle Cell Disease Research

    Hollywood, Florida, 33023, United States

  • INOVA Health

    Falls Church, Virginia, 22042-2325, United States

  • Inova Schar Cancer Institute

    Fairfax, Virginia, 22031, United States

  • Levine Cancer Institute

    Charlotte, North Carolina, 28204, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

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

    Charleston, South Carolina, 29425, United States

  • Mississippi Center for Advanced Medicine

    Madison, Mississippi, 39110, United States

  • Montefiore Medical Center

    The Bronx, New York, 10467, United States

  • Nemours Alfred I duPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Nemours Children's Health, Wilmington

    Wilmington, Delaware, 19803, United States

  • Nemours Children's Specialty Care

    Jacksonville, Florida, 32207, United States

  • Newark Beth Israel Medical Center

    Newark, New Jersey, 07112, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • Stanford Children's Hospital

    Palo Alto, California, 94304, United States

  • UPMC Montefiore Hospital

    Pittsburgh, Pennsylvania, 14213, United States

  • UPMC Presbyterian

    Pittsburgh, Pennsylvania, 15213, United States

  • UPMC Sickle Cell Center

    Pittsburgh, Pennsylvania, 15123, United States

  • University of California, San Diego

    La Jolla, California, 92093-0987, United States

  • University of Connecticut Health

    Farmington, Connecticut, 06030-1163, United States

  • University of Connecticut Health

    Farmington, Connecticut, 06030, United States

  • University of Illinois Hospital and Health Sciences System

    Chicago, Illinois, 60612, United States

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

    Chicago, Illinois, 60612, United States

  • University of Illinois at Chicago (UIC) Clinical Research Center

    Chicago, Illinois, 60612, United States

  • University of Illinois at Chicago (UIC) Sickle Cell Center

    Chicago, Illinois, 60612, United States

  • University of Maryland Medical Center

    College Park, Maryland, 21201, United States

  • University of Miami Hospital

    Miami, Florida, 33136, United States

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania, 15261, United States

  • University of South Alabama

    Mobile, Alabama, 36617, United States

  • University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.