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Sickle cell drug voxelotor tested for Long-Term safety

NCT ID NCT03573882

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety of voxelotor (a daily pill) in 179 people with sickle cell disease who had already taken it in a previous trial. Participants continued taking the drug and were monitored for side effects and complications like pain crises. The trial was terminated early, so the full picture of long-term benefits and risks is not yet clear.

What this could mean

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

Active substance
Voxelotor (also called GBT440), a daily tablet
What this could lead to
If successful, this could confirm that long-term use of voxelotor is safe and helps manage sickle cell disease complications.
What could go wrong
This trial was terminated early, so long-term data is limited. It only included people who already tolerated voxelotor, so 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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

179 people

The number who actually took part.

Started

Jun 2018

Finished

Nov 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.

Ages

12 to 65 years

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: * Male or female study participants with SCD who participated and received study treatment in Study GBT440-031. Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor. * Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. * Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug. * Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained). Exclusion Criteria: * Female who is breast-feeding or pregnant. * Participant withdrew consent from Study GBT440-031. * Participant was lost to follow-up from Study GBT440-031. * Participant requiring chronic dialysis. * Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.

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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

  • Abu El Rich Hospital,Cairo University Hospital

    Cairo, 11541, Egypt

  • Academic Medical Center(AMC)

    Amsterdam, 1105 AZ, Netherlands

  • Adana Acibadem Hospital

    Adana, 01130, Turkey (Türkiye)

  • Ain Shams University Hospital

    Cairo, Egypt

  • Alexandria Clinical Research Center, Faculty of Medicine

    Alexandria, Egypt

  • Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University

    Alexandria, Egypt

  • American University of Beirut

    Beirut, Lebanon

  • Arkansas Primary Care Clinic, PA

    Little Rock, Arkansas, 72204, United States

  • Azienda Ospedaliera di Padova

    Padova, Padova Veneto, 35128, Italy

  • Barts Health NHS Trust

    London, Greater London, E1 1BB, United Kingdom

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115, United States

  • Brigham and Women's Hospital Research Pharmacy

    Boston, Massachusetts, 02215, United States

  • Centres for Disease Control and Prevention

    Siaya, Kisumu County, Kenya

  • Children's Healthcare of Atlanta - Scottish Rite

    Atlanta, Georgia, 30342, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201, United States

  • Clinical Research Services Unit- Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Clinical and Translational Research Center (CTRC)

    Chapel Hill, North Carolina, 27599, United States

  • Columbia University Medical Center - Herbert Irving Pavilion

    New York, New York, 10032, United States

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • ErasmusMC

    Rotterdam, 3015 CN, Netherlands

  • Erciyes Universitesi Tip Fakultesi

    Kayseri, 38039, Turkey (Türkiye)

  • Gertrude's Children's Hospital

    Nairobi, Kenya

  • Guys and St. Thomas Hospital NHS Foundation Trust

    Great Maze Pond, London, SE1 9RT, United Kingdom

  • Homerton University Hospital NHS Foundation Trust

    London, Greater London, E9 6SR, United Kingdom

  • Hôpital Européen Georges Pompidou - Medecine Interne

    Paris, 75015, France

  • Imperial College Healthcare NHS Trust, Hammersmith Hospital

    London, W12 0HS, United Kingdom

  • Indiana Hemophilia and Thrombosis Center

    Indianapolis, Indiana, 46260, United States

  • Investigational Drug Service, Duke University Hospital

    Durham, North Carolina, 27710, United States

  • Jackson Memorial Hospital

    Miami, Florida, 33136, United States

  • Jackson Memorial Hospital (Investigational Drug Services)

    Miami, Florida, 33136, United States

  • Johns Hopkins University/Sickle Cell Infusion Center

    Baltimore, Maryland, 21287, United States

  • KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research

    Nairobi, 00101, Kenya

  • King's College Hospital

    London, SE5 9RS, United Kingdom

  • Lynn Health Science Institute

    Oklahoma City, Oklahoma, 73112, United States

  • McMillan Cancer Centre

    London, WC1E 6AG, United Kingdom

  • Medical University of South Carolina - Comprehensive Sickle Cell Clinic

    Charleston, South Carolina, 29425, United States

  • Medical University of South Carolina: Investigational Drug Services Pharmacy

    Charleston, South Carolina, 29425, United States

  • Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi

    Mersin, 33343, Turkey (Türkiye)

  • Methodist Comprehensive Sickle Cell Clinic

    Memphis, Tennessee, 38104, United States

  • Montefiore - Einstein Center for Cancer Care

    The Bronx, New York, 10461, United States

  • Newark Beth Israel Medical Center

    Newark, New Jersey, 07112, United States

  • Nini Hospital

    Tripoli, Lebanon

  • Our Lady of the Lake Physician Group-Medical Oncology

    Baton Rouge, Louisiana, 70809, United States

  • St. Jude Affiliate Clinic Baton Rouge

    Baton Rouge, Louisiana, 70808, United States

  • Sultan Qaboos University Hospital

    Muscat, 123, Oman

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • Texas Children's Hospital - Investigational Pharmacy

    Houston, Texas, 77030, United States

  • The Egyptian Thalassemia Association ( E.T.A)

    Cairo, Egypt

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107, United States

  • Toronto General Hospital-University Health Network

    Toronto, Ontario, M5G 2C4, Canada

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • UNC Hospitals

    Chapel Hill, North Carolina, 27514, United States

  • University Medical Center New Orleans

    New Orleans, Louisiana, 70112, United States

  • University of Illinois Hospital and Health Science System

    Chicago, Illinois, 60612, United States

  • University of Illinois at Chicago Clinical Research Center

    Chicago, Illinois, 60612, United States

  • University of Miami

    Miami, Florida, 33136, United States

  • University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)

    Miami, Florida, 33136, United States

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania, 15232, United States

  • Zagazig University Hospital

    Alsharkia, Egypt

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