NCT06609226

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 480 (estimated)
• Sponsor: Novo Nordisk A/S · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Conditions

• Sickle Cell Disease
• Thalassemia

Interventions

Type	Name	Description
DRUG	Etavopivat A	Participants will receive an oral dose of Etavopivat A.
DRUG	Etavopivat B	Participants will receive an oral dose of Etavopivat B.
DRUG	Etavopivat C	Participants will receive an oral dose of Etavopivat C.

Timeline

• Start date: 2025-01-10
• Primary completion: 2030-12-30
• Completion: 2030-12-30
• First posted: 2024-09-24
• Last updated: 2026-03-24

Locations

103 sites across 17 countries: United States, Canada, Egypt, France, Germany, Ghana, Greece, India, Italy, Kenya, Lebanon, Nigeria, Oman, Saudi Arabia, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study