Clinical Trial Goal
To find out if etavopivat is safe and works well to treat beta thalassemia major or SCD
You may be able to join this trial if you:
- Are 12 years old or older
- Have beta thalassemia major or SCD
- Have had at least 1 vaso-occlusive crises in the last 1 year
- Have not had more than 15 vaso-occlusive crises in the last 1 year
- Have not received a blood transfusion in the last 2 months
- Agree to have other standard tests done to see if you can be in the clinical trial
Trial Details
Etavopivat is an erythrocyte pyruvate kinase (PKR) activator in red blood cells that doctors think can make your red blood cells heathier and lower the number of red blood cell transfusions needed.
In this trial, you’ll be randomized to 1 of 2 groups. Once you’re randomized, you and the clinical trial doctors won’t know which group you’re in until after the trial is finished:
In this trial, you’ll be randomized to 1 of 2 groups. Once you’re randomized, you and the clinical trial doctors won’t know which group you’re in until after the trial is finished:
- Group 1 – Etavopivat
- Group 2 – Placebo
Randomized means doctors will use a computer to assign you to either group. A computer assigns you by chance, like flipping a coin or drawing a name out of a hat. You, your doctor or the clinical trial doctor won’t have any control over which group you’ll be assigned. This means you won’t be able to choose your group.
You’ll get:
- Etavopivat – Group 1 only - A pill that you take by mouth. The dose you'll get depends on when you start the trial and how safe it has been
- Placebo – Group 2 only - A pill that you take by mouth. The dose you'll get depends on when you start the trial and how safe it has been
The clinical trial doctors will check your health for 1 year.
The Food and Drug Administration (FDA) has not yet approved etavopivat.
Locations
Sponsors
lead: Novo Nordisk A/S
