Sickle cell drug voxelotor tested for Long-Term safety – but trial ends early
NCT ID NCT04188509
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.
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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Ain Shams University Hospital - Clinical Research Center (MASRI-CRC)
Cairo, Egypt
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Alexandria University Hospital - Clinical Research Center
Alexandria, 21131, Egypt
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American University of Beirut - Medical Center
Beirut, Lebanon
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Aminu Kano Teaching Hospital
Kano, 700233, Nigeria
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Barau Dikko Teaching Hospital/Kaduna State University
Kaduna, 800212, Nigeria
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Barts Health NHS Trust , The Royal London Hospital
London, E1 1BB, United Kingdom
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Cairo University Hospital, Abu El Rish Hospital
Cairo, 11562, Egypt
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Children's Healthcare of Atlanta Scottish Rite
Atlanta, Georgia, 30342, United States
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Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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College of Medicine, University of Ibadan
Ibadan, Oyo State, 200212, Nigeria
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ECU Physicians, Brody Outpatient Clinic
Greenville, North Carolina, 27834, United States
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Guy'S and St Thomas' Nhs Foundation Trust
London, SE1 7EH, United Kingdom
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Lagos University Teaching Hospital
Lagos, 100254, Nigeria
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Nini Hospital
Tripoli, Lebanon
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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University College Hospital NHS Foundation Trust
London, Greater London, NW1 2PG, United Kingdom
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University of Calabar Teaching Hospital
Calabar, Cross River State, 540281, Nigeria
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Zagazig University Hospital
Zagazig, Alsharkia, Egypt