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The Effect of Voxelotor on Cerebral Hemodynamic Response in Children With Sickle Cell Anemia

Studying voxelotor's impact on brain blood flow in sickle cell kids.

Recruiting
4-30 years
All
Phase 2

Voxelotor is a medicine for people with sickle cell disease (SCD), a condition where red blood cells are shaped like a sickle, making it hard for them to carry oxygen. Voxelotor helps by improving hemoglobin, the part of blood that carries oxygen. The study aims to see if voxelotor can also make the brain's blood flow better in kids with SCD.

When kids with SCD have low hemoglobin, their brains work hard to get enough oxygen. This study will check how brain blood flow and oxygen use changes with voxelotor, using a special light test called DCS/FDNIRS. This test is safe and doesn't hurt.

  • Duration: The study lasts 12 weeks, with check-ups at the start, at 4 weeks, and at 12 weeks.
  • Participation: Participants take voxelotor every day and must come to all check-ups.
  • Eligibility: Participants must be 4-30 years old and have certain types of sickle cell disease.

Participants will take voxelotor daily, and their brain blood flow and oxygen levels will be checked three times during the study. This research will help us understand if voxelotor can make brain blood flow better for kids with SCD.

Study details
    Sickle Cell Anemia in Children

NCT05018728

Amy Tang

12 October 2024

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