NCT05725902

Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

A Pilot Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Novo Nordisk A/S · Industry
• Sex: All
• Age: 12 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.

Detailed description

This study is a pilot, open-label, single-arm study to evaluate the effect of etavopivat on cerebral hemodynamics, as measured by frequency domain near-infrared spectroscopy/diffuse correlation spectroscopy (FDNIRS/DCS) in participants 12 to 21 years of age with sickle cell disease (SCD). Cerebral blood flow (CBF), oxygen ejection fraction (OEF), and cerebral metabolic rate of oxygen (CMRO2) will be assessed FDNIRS/DCS in participants prior to, periodically throughout, and after 24 weeks of treatment with etavopivat. Approximately 12 participants will be enrolled. The duration of study treatment will be 24 weeks. The study duration for individual participants may last up to 36 to 38 weeks and includes the Screening Period (up to 4 weeks before study treatment), the 24-week treatment period, a Safety Follow-up Visit at 4 weeks (+ 7 days) after the last dose of study drug, and an End of Study (EOS) visit approximately 8 weeks (± 7 days) after the last dose of study drug. A participant is considered to have completed the study if he or she has completed all phases of the study including the last visit or the last scheduled procedure shown in the Schedule of Events.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Etavopivat	The study intervention is etavopivat (400 mg), administered orally and once daily (QD)

Timeline

• Start date: 2024-03-09
• Primary completion: 2026-03-09
• Completion: 2026-03-09
• First posted: 2023-02-13
• Last updated: 2026-04-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study