NCT05228821

Voxelotor Brain Oxygenation and Neurocognitive Study

An Open Label, Single Arm, Multicenter Study to Evaluate the Effect of Voxelotor on Cerebral Blood Flow and Neurocognitive Function in Adolescents and Adults With Sickle Cell Disease

• Status: Withdrawn
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 12 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).

Detailed description

Eligible participants will receive daily treatment with 1500 mg voxelotor for 24 weeks. During screening, at 12 and 24 weeks, participants will undergo an MRI for evaluation of cerebral blood flow and oxygen extraction fraction as well as NIH toolbox testing for evaluation of executive function, processing speed, and nonexecutive function.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Voxelotor Oral Tablet	During the Treatment Period, participants will receive 1500 mg of voxelotor once daily (administered as tablets) for 24 weeks in addition to ongoing current standard of care (SOC) treatment

Timeline

• Start date: 2023-08-09
• Primary completion: 2023-08-23
• Completion: 2023-08-23
• First posted: 2022-02-08
• Last updated: 2024-08-09

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study