NCT04987489

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Forma Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin.

Detailed description

Etavopivat is a potent, selective, orally bioavailable, small-molecule activator of pyruvate kinase red blood cell (PKR) being developed by Forma Therapeutics, Inc and is intended for use as a treatment for patients with sickle cell disease (SCD) or other inherited hemoglobinopathies or refractory anemias. This study is a multicenter, Phase 2, open-label, multiple-cohort study examining the safety and efficacy of etavopivat for the treatment of patients, age 12 to 65 years, with SCD or thalassemia. Three treatment cohorts based on the patients hemoglobinopathy (SCD or thalassemia) and transfusion requirements will be evaluated.

Conditions

• Sickle Cell Disease
• Thalassemia

Interventions

Type	Name	Description
DRUG	Etavopivat tablets	Etavopivat 400 mg once daily

Timeline

• Start date: 2022-03-28
• Primary completion: 2025-09-03
• Completion: 2025-09-24
• First posted: 2021-08-03
• Last updated: 2025-12-16

Locations

32 sites across 6 countries: United States, Canada, Egypt, Italy, Lebanon, United Kingdom

Regulatory

• FDA-regulated drug study