Sickle cell drug voxelotor tested for Long-Term safety
NCT ID NCT03573882
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Abu El Rich Hospital,Cairo University Hospital
Cairo, 11541, Egypt
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Academic Medical Center(AMC)
Amsterdam, 1105 AZ, Netherlands
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Adana Acibadem Hospital
Adana, 01130, Turkey (Türkiye)
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Ain Shams University Hospital
Cairo, Egypt
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Alexandria Clinical Research Center, Faculty of Medicine
Alexandria, Egypt
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Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
Alexandria, Egypt
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American University of Beirut
Beirut, Lebanon
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Arkansas Primary Care Clinic, PA
Little Rock, Arkansas, 72204, United States
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Azienda Ospedaliera di Padova
Padova, Padova Veneto, 35128, Italy
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Barts Health NHS Trust
London, Greater London, E1 1BB, United Kingdom
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Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
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Brigham and Women's Hospital Research Pharmacy
Boston, Massachusetts, 02215, United States
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Centres for Disease Control and Prevention
Siaya, Kisumu County, Kenya
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Children's Healthcare of Atlanta - Scottish Rite
Atlanta, Georgia, 30342, United States
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Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
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Clinical Research Services Unit- Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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Clinical and Translational Research Center (CTRC)
Chapel Hill, North Carolina, 27599, United States
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Columbia University Medical Center - Herbert Irving Pavilion
New York, New York, 10032, United States
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Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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ErasmusMC
Rotterdam, 3015 CN, Netherlands
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Erciyes Universitesi Tip Fakultesi
Kayseri, 38039, Turkey (Türkiye)
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Gertrude's Children's Hospital
Nairobi, Kenya
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Guys and St. Thomas Hospital NHS Foundation Trust
Great Maze Pond, London, SE1 9RT, United Kingdom
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Homerton University Hospital NHS Foundation Trust
London, Greater London, E9 6SR, United Kingdom
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Hôpital Européen Georges Pompidou - Medecine Interne
Paris, 75015, France
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Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, W12 0HS, United Kingdom
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Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260, United States
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Investigational Drug Service, Duke University Hospital
Durham, North Carolina, 27710, United States
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Jackson Memorial Hospital
Miami, Florida, 33136, United States
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Jackson Memorial Hospital (Investigational Drug Services)
Miami, Florida, 33136, United States
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Johns Hopkins University/Sickle Cell Infusion Center
Baltimore, Maryland, 21287, United States
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KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
Nairobi, 00101, Kenya
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King's College Hospital
London, SE5 9RS, United Kingdom
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Lynn Health Science Institute
Oklahoma City, Oklahoma, 73112, United States
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McMillan Cancer Centre
London, WC1E 6AG, United Kingdom
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Medical University of South Carolina - Comprehensive Sickle Cell Clinic
Charleston, South Carolina, 29425, United States
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Medical University of South Carolina: Investigational Drug Services Pharmacy
Charleston, South Carolina, 29425, United States
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Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
Mersin, 33343, Turkey (Türkiye)
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Methodist Comprehensive Sickle Cell Clinic
Memphis, Tennessee, 38104, United States
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Montefiore - Einstein Center for Cancer Care
The Bronx, New York, 10461, United States
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Newark Beth Israel Medical Center
Newark, New Jersey, 07112, United States
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Nini Hospital
Tripoli, Lebanon
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Our Lady of the Lake Physician Group-Medical Oncology
Baton Rouge, Louisiana, 70809, United States
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St. Jude Affiliate Clinic Baton Rouge
Baton Rouge, Louisiana, 70808, United States
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Sultan Qaboos University Hospital
Muscat, 123, Oman
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Texas Children's Hospital
Houston, Texas, 77030, United States
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Texas Children's Hospital - Investigational Pharmacy
Houston, Texas, 77030, United States
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The Egyptian Thalassemia Association ( E.T.A)
Cairo, Egypt
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Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
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Toronto General Hospital-University Health Network
Toronto, Ontario, M5G 2C4, Canada
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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UNC Hospitals
Chapel Hill, North Carolina, 27514, United States
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University Medical Center New Orleans
New Orleans, Louisiana, 70112, United States
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University of Illinois Hospital and Health Science System
Chicago, Illinois, 60612, United States
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University of Illinois at Chicago Clinical Research Center
Chicago, Illinois, 60612, United States
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University of Miami
Miami, Florida, 33136, United States
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University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
Miami, Florida, 33136, United States
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University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15232, United States
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Zagazig University Hospital
Alsharkia, Egypt