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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for SICKLE CELL ANEMIA IN CHILDREN are added.
By submitting, you agree to our Terms of use
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
Locations
-
Aflac Sickle Cell Comprehensive Clinics at Children's Healthcare of Atlanta, Scottish Rite
Atlanta, Georgia, 30342, United States
-
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a common drug shield children with sickle cell from stroke?
- Could a stronger dose of an old drug protect children with sickle cell from organ damage?
- Can a 90-Minute training improve care for kids with sickle cell?
- New blood processing method could cut transfusion needs for sickle cell patients
- Water workouts may ease sickle cell struggles in kids