Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease Transcranial Doppler Ultrasound (TCD)
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 236
Healthy volunteers: no

Brief summary:
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

Detailed description:
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to \< 15 years old, with Sickle Cell Disease. The study will be conducted at approximately 50 international clinical sites, and will enroll approximately 224 participants. Participants will be randomized in a 1:1 ratio to receive voxelotor or placebo. All participants younger than 12 years of age and randomized to voxelotor will receive a dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg/day.

Arms:
arm 1: Voxelotor 1500mg or equivalent daily as a tablet, dispersible tablet, or as powder for oral suspension. arm 2: Matching placebo.

Interventions: intervention 1: Voxelotor intervention 2: Placebo
Intervention descriptions: intervention 1: Participants are randomized 1:1 to receive voxelotor or placebo. intervention 2: Matching placebo.

Protocol document:
Prot_000.pdf: 













































































































































































SAP_001.pdf: