NCT04218084

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 236 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 2 Years – 14 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Voxelotor	Participants are randomized 1:1 to receive voxelotor or placebo.
DRUG	Placebo	Matching placebo.

Timeline

• Start date: 2020-11-11
• Primary completion: 2024-01-18
• Completion: 2024-11-06
• First posted: 2020-01-06
• Last updated: 2026-03-10
• Results posted: 2025-06-13

Locations

35 sites across 9 countries: United States, Egypt, Ghana, Italy, Kenya, Nigeria, Oman, Saudi Arabia, United Kingdom

Regulatory

• FDA-regulated drug study