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Sickle cell drug voxelotor tested for Long-Term safety – but trial ends early

NCT ID NCT04188509

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looked at the long-term safety of the drug voxelotor in 162 people with sickle cell disease who had already taken it in earlier trials. Everyone received voxelotor once daily. The main goal was to track side effects. The trial was terminated early, so the full safety picture may not be 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 (GBT440)
What this could lead to
If successful, this could confirm that long-term use of voxelotor is safe and helps manage sickle cell disease symptoms.
What could go wrong
The trial was terminated early, so results may be incomplete. It only included people who had already taken voxelotor, which limits what we can learn about broader safety.

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

162 people

The number who actually took part.

Started

Nov 2019

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

6 months 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: * Male or female participant with SCD, who participated and received study drug in a GBT-sponsored voxelotor clinical study. Note: Participants 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. Note: Participants who discontinued Study GBT440-032 as the result of an abnormal transcranial Doppler (TCD) flow velocity assessment (≥ 200 cm/sec) are eligible for treatment in this study. \- Participant has provided written consent/assent (for pediatric participants, both the consent of the participant's legal representative or legal guardian and the participant's assent \[where applicable\] must be obtained). Exclusion Criteria: * Female participant who is breastfeeding or pregnant * Participant withdrew consent from a GBT-sponsored voxelotor clinical study * Known hypersensitivity to voxelotor or any other components of the study drug * Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1

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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

  • Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)

    Cairo, Egypt

  • Alexandria University Hospital - Clinical Research Center

    Alexandria, 21131, Egypt

  • American University of Beirut - Medical Center

    Beirut, Lebanon

  • Aminu Kano Teaching Hospital

    Kano, 700233, Nigeria

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Barau Dikko Teaching Hospital/Kaduna State University

    Kaduna, 800212, Nigeria

  • Barts Health NHS Trust , The Royal London Hospital

    London, E1 1BB, United Kingdom

  • Cairo University Hospital, Abu El Rish Hospital

    Cairo, 11562, Egypt

  • Children's Healthcare of Atlanta Scottish Rite

    Atlanta, Georgia, 30342, United States

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • College of Medicine, University of Ibadan

    Ibadan, Oyo State, 200212, Nigeria

  • ECU Physicians, Brody Outpatient Clinic

    Greenville, North Carolina, 27834, United States

  • Guy'S and St Thomas' Nhs Foundation Trust

    London, SE1 7EH, United Kingdom

  • Lagos University Teaching Hospital

    Lagos, 100254, Nigeria

  • Nini Hospital

    Tripoli, Lebanon

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • University College Hospital NHS Foundation Trust

    London, Greater London, NW1 2PG, United Kingdom

  • University of Calabar Teaching Hospital

    Calabar, Cross River State, 540281, Nigeria

  • Zagazig University Hospital

    Zagazig, Alsharkia, Egypt

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