What is the ENERGIZE clinical trial?
The ENERGIZE clinical trial is now enrolling adults with thalassemia who do not regularly receive transfusions. The trial’s main goal is to see whether mitapivat, the study drug, improves hemoglobin levels.
The trial is also studying whether fatigue and 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 of either
- Have received no more than five red blood cell (RBC) transfusions in the past 24 weeks
- Have not received any RBC transfusions within 8 weeks before and for approximately 6 weeks after signing the informed consent document (ICD)
- 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 a 6-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 24 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 24-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.
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.
In clinical studies, treatment groups may be “blinded,” meaning that no one is told what kind of treatment they are receiving. In ENERGIZE, 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 24-week blinded period is over and an initial analysis is done.
For eligible participants who choose to continue to take part in the open-label extension, the trial could last up to about 5¾ 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 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 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.