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Sickle cell drug trial in kids halted early – but data may still help

NCT ID NCT04218084

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested a daily pill called voxelotor in children aged 2 to 14 with sickle cell disease who had slightly abnormal blood flow in the brain. The goal was to see if the drug could improve that blood flow and reduce stroke risk. The trial was stopped early, but the results may still provide useful information for managing the condition.

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

236 people

The number who actually took part.

Started

Nov 2020

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

2 to 14 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: 1. Male or female participants with Sickle Cell Anemia (SCA) HbSS, HbSβ0 thalassemia genotype 2. TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥ 170 to \< 200cm/sec during the Screening Period 3. Hb ≥ 5.5 and ≤ 10.5 g/dL during screening 4. For participants taking HU, the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the informed consent form (ICF) and/or assent form, and with no anticipated need for dose adjustments (other than weight based) or for initiation of HU for non-chronic use during the study, in the opinion of the Investigator 5. Written informed parental/guardian consent and participant assent (where applicable) has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines. Exclusion Criteria: 1. Body weight \< 10kg at the screening visit 2. Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent 3. More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit 4. Stroke resulting in focal neurological deficit; previous silent infarcts are permitted. 5. Known history or findings suggestive of significant cerebral vasculopathy 6. History of seizure disorder 7. Has been treated with erythropoietin or other hematopoietic growth factors within 28 days of signing informed consent/assent or if, in the opinion of the Investigator, there is an anticipated need for such agents during the study 8. RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion) or has received an RBC transfusion or exchange transfusion for any reason within 90 days of signing the informed consent/assent

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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, 11562, Egypt

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

    Cairo, Abassia, 11566, Egypt

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

    Alexandria, 21131, Egypt

  • Aminu Kano Teaching Hospital

    Kano, 700233, Nigeria

  • Azienda Ospedaliera Universita' (AOU ) Padova

    Padova, 35128, Italy

  • Azienda Ospedaliera Universita' (AOU) Padova

    Padova, 35128, Italy

  • Azienda Ospedaliera Universitaria (A.O.U.) "Luigi Vanvitelli"

    Naples, 80138, Italy

  • Azienda Ospedaliera Universitaria Meyer "A.O.U. Meyer" - SOC "Oncologia, Ematologia e TCSE"

    Florence, 50139, Italy

  • Barau Dikko Teaching/Kaduna State University

    Kaduna, 800212, Nigeria

  • Barts Health NHS Trust

    London, E1 1BB, United Kingdom

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Boston Children's Hospital - Clinical Research Pharmacy

    Boston, Massachusetts, 02115, United States

  • Children's Healthcare of Atlanta: Hughes Spalding

    Atlanta, Georgia, 30303, 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

  • Department of Child Health, University of Ghana Medical School, College of Health Sciences, Korle-Bu

    Accra, Greater Accra Region, GA-221-1570, Ghana

  • Gertrude's Children Hospital

    Nairobi, 100, Kenya

  • KEMRI CRDR Clinical Research Annex

    Nairobi, 00100, Kenya

  • Kemri/Crdr,Siaya,Kemri Clinical Research Annex

    Kisumu, Siaya County, 40600, Kenya

  • King Abdullah International Medical Research Center (KAIMRC), Ministry of National Guard - Health

    Riyadh, 11481, Saudi Arabia

  • Komfo Anokye Teaching Hospital

    Kumasi, Ashanti Region, 00233, Ghana

  • Lagos University Teaching Hospital

    Surulere, Lagos, 100254, Nigeria

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medical University of South Carolina: Investigational Drug Services

    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

  • Strathmore University Medical Centre

    Nairobi, 00200, Kenya

  • Sultan َQaboos University Hospital

    Muscat, Oman

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • Texas Children's Hospital - Investigational Pharmacy

    Houston, Texas, 77030, United States

  • Texas Children's Hospital- Wallace Tower

    Houston, Texas, 77030, United States

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • University of Miami

    Miami, Florida, 33136, United States

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216, United States

  • University of Nigeria Teaching Hospital

    Enugu, 460000, Nigeria

  • Zagazig University Hospital

    Zagazig, Alsharkia, Egypt

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