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

What this Trial Is About

Researchers are studying etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein that carries oxygen in the blood. This phase 3 open-label study aims to assess the long-term safety and effectiveness of etavopivat in adults, adolescents, and children who have completed treatment in an earlier etavopivat study. The study is sponsored by Novo Nordisk AS and may last up to 264 weeks unless etavopivat is approved earlier in the participants country. Participants will receive an oral dose of etavopivat, with dosing varying by age and condition. Those aged 12 years or older will receive either Etavopivat A or C, while children under 12 years old will receive Etavopivat B. The study includes groups with sickle cell disease or thalassaemia, some of whom may be transfusion-dependent. The treatment will be continuous throughout the study period, aiming to observe long-term effects and safety. During the study, participants will be closely monitored for treatment-emergent adverse events and adverse reactions. Researchers will track clinical outcomes such as vaso-occlusive crisis rates, hemoglobin levels, hospitalizations, and red blood cell transfusions, both at baseline and throughout treatment. Data will be collected regularly to assess safety and treatment impact across different age groups and disease types, with the total study duration potentially extending to over six years.

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