Clinical Trials Logo

Clinical Trial Summary

Voxelotor is a new drug for adolescents and adults with sickle cell disease that improves hemoglobin levels and reduces the incidence of worsening anemia. However, it is unclear whether this increase in hemoglobin is associated with a reduction in cerebral metabolic stress. This study will measure the effects of voxelotor on cerebral hemodynamics.


Clinical Trial Description

Sickle cell disease (SCD) is a genetic blood disorder that has profound effects on the brain. In the presence of chronic anemia, the brain microvasculature dilates in order to maintain adequate oxygen delivery to the tissue. As cerebral blood flow increases and the vasculature becomes maximally dilated, oxygen extraction fraction (OEF) increases to keep up with metabolic demand. Unfortunately, this state of maximized response leaves the brain vulnerable and ill-equipped to adapt to increased metabolic demand; consequently, the tissue is susceptible to infarction when blood flow/volume are insufficient to maintain sufficient oxygen metabolism. Voxelotor is a first-in-class drug approved by the FDA for treatment of adults and children (aged four years and older) with SCD. This study will examine the effects of voxelotor on cerebral hemodynamics. The researchers hypothesize that as hemoglobin increases due to voxelotor, cerebral blood flow and oxygen extraction fraction will decrease. To test this hypothesis, measurements of brain blood flow, oxygen extraction, and oxygen metabolism will be made using a customized diffuse correlation spectroscopy and frequency domain near-infrared spectroscopy system (DCS/FDNIRS). This non-invasive, optical technique uses light to estimate an average oxygen saturation of the microvasculature, i.e., capillaries, arterioles, and venules. All participants will receive voxelotor, administered orally once daily as tablets, dispersible tablets or as powder for oral suspension at a dose based on their body weight, for 12 weeks. Measurements of cerebral blood flow, oxygen extraction, and oxygen metabolism will be made at baseline (before starting drug), and at 4, and 12 weeks after the start of voxelotor. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05018728
Study type Interventional
Source Emory University
Contact Amy Tang, MD
Phone 404-785-1441
Email amy.tang@choa.org
Status Recruiting
Phase Phase 2
Start date March 28, 2022
Completion date December 2024

See also
  Status Clinical Trial Phase
Recruiting NCT06387758 - Low Systemic/High Local Exercise Load in Peds SCD N/A
Completed NCT04579926 - PINPOINT: Gaming Technology for SCD Pain N/A
Completed NCT03632876 - Nutritional Outcomes After Vitamin A Supplementation in Subjects With SCD N/A
Recruiting NCT05285917 - Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa Phase 3
Completed NCT04301336 - Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia Phase 2/Phase 3
Completed NCT03176849 - A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT Phase 4
Active, not recruiting NCT04750707 - Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II ) Phase 3
Recruiting NCT04191213 - Gum Arabic as Anti-oxidant, Anti-inflammatory and Fetal Hemoglobin Inducing Agent in Sickle Cell Anemia Patients Phase 2/Phase 3
Completed NCT04844099 - Dihydroartemisinin-Piperaquine or Sulphadoxine-Pyrimethamine for the Chemoprevention of Malaria in Sickle Cell Anaemia Phase 3
Enrolling by invitation NCT03948867 - Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE) Phase 2
Completed NCT04800809 - The Afolabi Stroke Registry for Children and Young Adults With SCD in Northern Nigeria