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Information for Healthcare Professionals

The ENERGIZE-T Clinical Trial

What is the ENERGIZE-T clinical trial?

The ENERGIZE-T clinical trial is now enrolling adults with thalassemia who regularly receive transfusions. The trial’s main goal is to see whether mitapivat, the study drug, decreases the need for transfusions, and by how much.

The study is also investigating whether certain blood values change over the course of the study and by how much.

Who is eligible to participate?

To enroll in the trial, you must:

  • Be 18 years of age or older at the time your participation begins
  • Have a confirmed diagnosis via DNA analysis of either
    • Alpha-thalassemia
    • Beta-thalassemia
  • Require regular transfusions. This means that, during the 24 weeks before you start receiving study drug, you must:
    • Need a transfusion at least every 6 weeks
    • Have received 6 to 20 red blood cell (RBC) units
    • Not be pregnant or breastfeeding

If you are able to have children, or if you are male and have a partner who can have children, you must agree to use two forms of contraception throughout the trial or abstain from having sex.

There are other health criteria that must be met. You may not be able to participate if you have

  • Taken certain medications or have had specific types of treatment
  • Other medical conditions

Talk to your doctor to find out more about eligibility.

What does participation look like?

If you’re found eligible to participate and choose to enroll in the trial, you’ll undergo an 8-week screening period. Then you’ll be randomly assigned (by chance) to one of two study groups. For every two people assigned to Group A, one will be assigned to Group B.

For the next 48 weeks, one group will receive placebo, and the other group will receive the study drug. No one will know whether they received placebo or the study drug until the 48-week “blinded” period is over and an initial analysis has been performed.

This will be followed by an up to 5-year period during which everyone who chooses to continue their participation will receive the study drug.

What is a placebo?

A placebo is a substance that has no treatment effect. Comparing the experience of a group taking the placebo to a group taking the study drug helps make it clear whether any differences are the result of chance. When a study is set up this way, it’s sometimes referred to as a “placebo-controlled” clinical study.

What does “blinded” mean?

In clinical studies, treatment groups may be “blinded,” meaning that they are not told what kind of treatment they are receiving. In ENERGIZE-T, this period can also be referred to as “double-blind” because neither participants nor study staff will know who is receiving the study drug and who is receiving placebo until after the 48-week blinded period is over and an initial analysis is done.


~8-week screening period

During this time, doctors will confirm you are eligible and learn how your thalassemia affects you

Randomization: For every 2
people assigned to Group A, 1 will be assigned to Group B

48-week blinded period

GROUP
A

100 mg of mitapivat,
two times a day, as a tablet

GROUP
B

Placebo,
two times a day, as a tablet

Up to 5-year open-label extension

All participants (both Groups A and B) would receive mitapivat

~8-week
screening period

During this time, doctors will confirm you are eligible and
learn how your thalassemia affects you

Randomization: For every 2 people assigned to Group A, 1 will be assigned to Group B

48-week
blinded period

GROUP
A

100 mg of mitapivat,
two times a day, as a tablet

GROUP
B

Placebo,
two times a day, as a tablet

Up to 5-year
open-label extension

All participants (both Groups A and B) would receive mitapivat

For eligible participants who choose to continue to take part in the open-label extension, the trial could last more than 6 years. The length of your participation will also depend on whether:

  • Your anemia gets worse
  • You experience side effects and how badly they affect you
  • You decide to stop participating in the trial for any reason

Is there a study site near me?

ENERGIZE-T study locations, as well as additional details about the trial, are listed on the US government site ClinicalTrials.gov.

How can I learn more?

Read our FAQ (frequently asked questions), or visit some of the resources listed below.

About thalassemia
The Cooley’s Anemia Foundation
Thalassemia International Federation

About ENERGIZE-T
ClinicalTrials.gov (NCT04770779)

About clinical trials
Clinical trial information from the U.S. National Library of Medicine

Mitapivat is not approved by health authorities for the treatment of thalassemia.