Sickle cell drug voxelotor tested for brain benefits in kids – study cut short
NCT ID NCT05018728
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at whether voxelotor, a daily pill for sickle cell anemia, can improve blood flow and oxygen supply to the brain in children. The trial planned to enroll 22 participants aged 4 to 30 years with a severe form of sickle cell disease. However, the study was terminated early, so the results are limited and we cannot draw firm conclusions about the drug's brain benefits.
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 Oxbryta), a daily oral drug
What this could lead to
If successful, this could show that voxelotor improves brain health in children with sickle cell anemia, potentially reducing the risk of stroke or cognitive issues.
What could go wrong
The study was terminated early with only 22 participants, so we have very little data. It is unclear if the drug actually improves brain function, and it may not work for all children.
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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Aflac Sickle Cell Comprehensive Clinics at Children's Healthcare of Atlanta, Scottish Rite
Atlanta, Georgia, 30342, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States