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Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease — HOPE Kids 2  

Sickle Cell Disease Clinical Trial

Official title:
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease (HOPE Kids 2)

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04218084
Study type Interventional
Source Pfizer
Contact
Status Active, not recruiting
Phase Phase 3
Start date November 11, 2020
Completion date January 28, 2025
View Details
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