Active not recruitingPHASE3INTERVENTIONAL

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

This research study, called a Phase 3 trial, investigated a new medication called mitapivat. It was for adults (18 years and older) who have a genetic blood condition called thalassemia (either alpha or beta type) but don't need regular blood transfusions. The main goal was to find out if mitapivat was better than a placebo (a dummy pill) at improving anaemia, which is a common problem for people with thalassemia. Participants were divided into two groups: one received mitapivat, and the other received the placebo, to see how the new drug affected their red blood cells and overall health. The study also looked at how safe the new treatment was.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Agios Pharmaceuticals, Inc.

Enrolment target

194

Start

20 Dec 2021

Estimated completion

01 Dec 2028

Non-Transfusion-dependent Alpha-Thalassemia Non-Transfusion-dependent Beta-Thalassemia

What is this study about?

Imagine your blood cells are like tiny delivery vans carrying oxygen around your body. If you have thalassemia, these vans aren't made quite right, so they can't carry enough oxygen. This often leads to feeling tired and weak, a condition known as anaemia. Some people with thalassemia need regular blood transfusions, but this study focused on those who generally don't – a group called 'non-transfusion-dependent' thalassemia.

The medication being tested in this study is called mitapivat. It's a new type of drug designed to help your red blood cells work better. The researchers wanted to see if taking mitapivat could improve the anaemia in people with thalassemia, meaning they might feel less tired and have healthier blood counts without needing transfusions or other treatments as often. They compared it to a 'dummy' pill (a placebo) to make sure any improvements were actually due to mitapivat.

This study is important because it could offer a new treatment option for people living with non-transfusion-dependent alpha- or beta-thalassemia. Finding ways to manage anaemia more effectively, without the need for frequent medical procedures, could significantly improve daily life for those with this condition. The study also carefully checked the safety of mitapivat to ensure that any potential benefits outweigh the risks.

Key takeaways

This study tested a new drug, mitapivat, for a type of thalassemia.
It focused on adults (18+) who don't need regular blood transfusions.
The main goal was to see if mitapivat improved anaemia compared to a dummy pill.
Participation involved taking the study drug and regular health checks.
It could offer a new way to manage thalassemia for some patients.
Patient safety was monitored closely throughout the trial.

Who may be eligible?

This study was looking for adult volunteers, aged 18 or older, who have been diagnosed with a specific type of thalassemia (either alpha or beta) that doesn't usually require regular blood transfusions. To be considered, your haemoglobin levels (a measure of red blood cells) needed to be at a certain level or below.

You couldn't join if you were pregnant or breastfeeding, or if you had certain other serious health problems or had received particular treatments in the past, like stem cell transplants or gene therapy. Also, if you were taking certain medications for your blood condition, you might have had to stop them for a period before joining. Women who could get pregnant needed to agree to use effective contraception during and after the study.

Crucially, you couldn't have had a blood transfusion in the 8 weeks leading up to the study, nor during the screening period. If you were taking a medication called hydroxyurea, your dose needed to have been stable for at least 16 weeks.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of alpha- or beta-thalassemia?
Do you generally NOT need regular blood transfusions?
Have you NOT had a blood transfusion in the last 8 weeks?
Are you NOT pregnant or breastfeeding?
Are you able to attend regular clinic appointments?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you were eligible and chose to take part, you would have attended regular appointments at a clinic. You would have been given a study drug, either mitapivat or a placebo (a dummy pill), to take. Neither you nor your doctor would know which one you were receiving. You would have had blood tests taken regularly to monitor your red blood cell levels and to check for any side effects. You would also have given details about how you were feeling and any changes in your health. The study lasted for a significant period to properly assess the long-term effects and safety of the drug.

Potential risks and benefits

Taking part in any clinical study has potential benefits and risks. The potential benefit of this study was that mitapivat might improve your anaemia and overall well-being, potentially reducing the need for other treatments. However, like all medications, mitapivat could also have side effects, which might have varied from mild to more serious. The study carefully monitored all participants for any adverse effects. You would have been given detailed information about these before deciding to join, and you would have had the right to withdraw from the study at any time without giving a reason, and this would not affect your future medical care.

Locations (68)

San Diego Hospital, UC San Diego Health
La Jolla, United States
Stanford Medicine
Palo Alto, United States
Massachusetts General Hospital
Boston, United States
Weill Cornell Medical Center
New York, United States
Duke University Medical Center
Durham, United States
Penn Medicine - University of Pennsylvania Health System
Philadelphia, United States
Universidade de Caxias do Sul
Caxias do Sul, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, Brazil
HEMORIO Instituto Nacional de Hematologia
Rio de Janeiro, Brazil
Praxis Pesquisa Medica
Santo André, Brazil
GSH Banco de Sangue de São Paulo
São Paulo, Brazil
Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
São Paulo, Brazil

+56 more sites — see the official record for the full list.

Common questions

What is thalassemia?

Thalassemia is a blood disorder you're born with, where your body doesn't make enough healthy red blood cells, leading to tiredness and weakness (anaemia).

What does 'non-transfusion-dependent' mean?

It means you have thalassemia, but you don't typically need regular blood transfusions to manage your condition.

What is a 'placebo'?

A placebo is a dummy pill that looks just like the real drug but contains no active medicine. It helps researchers see if the real drug is truly effective.

Why were women of child-bearing potential asked to use contraception?

It's standard practice in drug studies to protect unborn babies, as the effects of new medications on pregnancy are often unknown.

Can I still join if I'm taking other medicines for thalassemia?

It depends on the medicine. If you're on hydroxyurea, your dose needed to be stable. If you were taking certain other blood-stimulating agents, you would have had to stop them some time before joining.