NCT05228834

Voxelotor Neurocognitive Function Study

A Phase 3b, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Treatment Effect of Voxelotor on Neurocognitive Function in Pediatric Participants 8 to < 18 Years of Age With Sickle Cell Disease

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 8 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition Module of executive abilities in pediatric participants (8 to \< 18 years) with SCD.

Detailed description

Eligible participants will receive daily treatment with 1500 mg voxelotor or matching placebo for 12 weeks. During screening and at the end of 12 weeks participants will undergo a series of tests to measure the change in neurocognitive functions.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Voxelotor Only Product in Oral Dose Form	During the Randomized Treatment Period, participants will be randomized in a 1:1 ratio to receive 1500 mg of voxelotor (or the weight-adjusted equivalent dose for participants \< 12 years old), once daily (administered orally as tablets/PFOS) or matching placebo for 12 weeks in addition to ongoing standard of care (SOC) treatment.
DRUG	Placebo	During the Randomized Treatment Period, participants will be randomized in a 1:1 ratio to receive 1500 mg of voxelotor (or the weight-adjusted equivalent dose for participants \< 12 years old), once daily (administered orally as tablets/PFOS) or matching placebo for 12 weeks in addition to ongoing standard of care (SOC) treatment.

Timeline

• Start date: 2022-06-24
• Primary completion: 2022-09-30
• Completion: 2022-09-30
• First posted: 2022-02-08
• Last updated: 2024-05-02
• Results posted: 2024-05-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study