NCT06198712

A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Forma Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Etavopivat	Participants will receive oral tablets of etavopivat once daily.

Timeline

• Start date: 2023-01-12
• Primary completion: 2028-02-23
• Completion: 2029-08-08
• First posted: 2024-01-10
• Last updated: 2026-03-24

Locations

18 sites across 7 countries: Canada, France, Kenya, Lebanon, Nigeria, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study