Active not recruitingPHASE3INTERVENTIONAL

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

This research study is investigating a new medication, mitapivat, for individuals living with alpha or beta thalassemia who rely on frequent blood transfusions. The main goal is to determine if mitapivat can decrease the number of blood transfusions these patients need over time. Participants will receive either mitapivat or a placebo (a dummy drug) to see if the new medicine works and if it's safe. Thalassemia is a blood condition where the body doesn't produce enough healthy red blood cells, often leading to anaemia and requiring transfusions. This study is in its advanced stages, meaning it’s already shown some promise in earlier testing. Researchers want to understand if mitapivat could be a new treatment option to improve the lives of people affected by this condition.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Agios Pharmaceuticals, Inc.

Enrolment target

258

Start

30 Nov 2021

Estimated completion

01 Jun 2029

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

What is this study about?

This study is testing a new medicine called mitapivat for people who have a specific type of blood condition called thalassemia. Thalassemia means your body can't make enough healthy red blood cells, which carry oxygen around your body. Because of this, many people with severe thalassemia, especially those with what's called 'transfusion-dependent' alpha or beta thalassemia, need regular blood transfusions. These transfusions can make life difficult and have their own risks over time.

The main idea behind this study is to see if mitapivat can help people with thalassemia reduce how often they need these blood transfusions. Some participants will receive the actual medicine, while others will get a placebo, which looks like the medicine but contains no active ingredients. This helps researchers fairly compare the new drug's effects. Knowing if a medicine can lessen the need for transfusions is very important because it could greatly improve daily life for people with thalassemia and reduce the associated medical burdens.

This is a Phase 3 study, which means it's a large, in-depth trial involving many participants. Medicines reach this stage after showing encouraging results in earlier, smaller studies. The detailed description states that 171 people were given mitapivat and 87 were given the placebo. This setup allows the researchers to gather strong evidence about the medicine's effectiveness and safety, helping them decide if it could become a widely available treatment in the future. The ultimate aim is to find better ways to manage thalassemia and improve the health and well-being of those affected.

Key takeaways

This study is testing a new medicine for severe thalassemia.
It aims to reduce the need for blood transfusions.
Participants will receive either the new medicine or a dummy treatment.
It's a large study, meaning the medicine has shown promise in earlier tests.
Careful monitoring of health and safety will be provided.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you would need to be at least 18 years old and have a confirmed diagnosis of alpha or beta thalassemia based on genetic tests. A key requirement is that your thalassemia is considered 'transfusion-dependent'. This means you've needed between 6 and 20 units of red blood cells and haven't gone longer than six weeks without a transfusion in the six months before the study starts. If you're currently taking a medicine called hydroxyurea, your dose must have been stable for at least 16 weeks before you can join.

There are also some reasons why you wouldn't be able to join. For example, if you are pregnant or breastfeeding, or if you've recently had other specific treatments like gene therapy or a bone marrow transplant. You also wouldn't be eligible if you have certain other serious health issues, like active heart or lung disease, or a recent history of certain cancers. The study also cannot include those who have recently used other specific thalassemia medications like luspatercept or other blood-stimulating agents.

Quick self-check

Are you 18 years or older?
Do you have a confirmed diagnosis of alpha or beta thalassemia?
Do you need regular blood transfusions for your thalassemia (6-20 units in the last 6 months, and no longer than 6 weeks without a transfusion)?
Are you not pregnant or breastfeeding?
Have you spoken to your doctor about this study and understand the requirements?

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

What does participation involve?

If you decide to take part in this study, you will be given either the study medicine, mitapivat, or a placebo (a dummy pill). You won't know which one you're receiving, and neither will your local study team, until the study is over. You'll have regular visits to the hospital or clinic where doctors will monitor your health, perform blood tests, and check how often you need blood transfusions. These visits are important to track the medicine's effects and ensure your safety. The study involves taking the medication for a set period, and there will be follow-up appointments even after you stop taking the study drug to see how you're doing. The total duration of your participation, including follow-up, will be explained in detail by the study team.

Potential risks and benefits

Like any medical study, there are potential benefits and risks to consider. You might benefit if mitapivat helps reduce your need for blood transfusions, which could improve your daily life. However, there's no guarantee that you will receive the active drug or that it will be effective for you. You might also experience side effects from the medicine, which will be carefully monitored by the study team. They will explain all known potential risks before you decide to join. It's very important to remember that participating is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (75)

Phoenix Children's Hospital
Phoenix, United States
San Diego Hospital, UC San Diego Health
La Jolla, United States
Children's Hospital Oakland
Oakland, United States
Stanford Medicine
Palo Alto, United States
Boston Children's Hospital
Boston, United States
Children's Hospital of Michigan
Detroit, 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
Seattle Cancer Care Alliance, University of Washington
Seattle, United States
Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, Brazil
GSH Banco de Sangue de São Paulo
São Paulo, Brazil

+63 more sites — see the official record for the full list.

Common questions

What is thalassemia?

Thalassemia is a genetic blood disorder where your body doesn't produce enough healthy red blood cells, leading to anaemia and other health issues.

What does 'transfusion-dependent' mean?

It means you regularly need blood transfusions because your body can't make enough red blood cells on its own to stay healthy.

What is a placebo?

A placebo is a substance or treatment that looks exactly like the study medicine but doesn't contain any active ingredients. It's used for comparison.

Why can't I be pregnant or breastfeeding?

For your safety and the safety of your baby, pregnant or breastfeeding individuals are typically not included in drug trials because medicines can affect a developing baby or be passed through breast milk.

Will I feel better right away?

There's no guarantee the medicine will work, or how quickly. The study aims to see effects over time, and your health will be closely monitored.