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Activator of Pyruvate Kinase-R
Mitapivat for Thalassemia (ENERGIZE Trial)
Phase 3
Waitlist Available
Research Sponsored by Agios Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Non-transfusion-dependent, defined as ≤5 red blood cell (RBC) units during the 24-week period before randomization; and no RBC transfusions ≤8 weeks before providing informed consent and no RBC transfusions during the Screening Period
Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H [HbH] disease) based on Hb electrophoresis, Hb high-performance liquid chromatography (HPLC)), and/or deoxyribonucleic acid (DNA) analysis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre-dose day 1; pre-dose week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 20
Awards & highlights
ENERGIZE Trial Summary
This trial will compare the effect of the drug mitapivat versus placebo on anemia in people with either alpha- or beta-non-transfusion dependent thalassemia.
Who is the study for?
This trial is for individuals with alpha- or beta-non-transfusion dependent thalassemia (NTDT) who have stable hemoglobin levels ≤10.0 g/dL and haven't had more than 5 blood transfusions in the past 24 weeks. Participants must not be pregnant, breastfeeding, or have certain other health conditions like uncontrolled heart disease, severe kidney issues, or active infections.Check my eligibility
What is being tested?
The study is testing Mitapivat against a placebo to see if it helps improve anemia in people with NTDT. Patients will either receive the actual drug or a placebo that has no therapeutic effect to compare outcomes between the two groups.See study design
What are the potential side effects?
While specific side effects of Mitapivat are not listed here, common side effects may include reactions at the site of administration, potential liver enzyme elevations, fatigue, headache, and digestive discomfort.
ENERGIZE Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have not needed more than 5 blood transfusions in the last 6 months and none in the last 2 months.
Select...
I have been diagnosed with thalassemia based on specific blood tests or DNA analysis.
Select...
My hydroxyurea dose has been the same for at least 16 weeks.
ENERGIZE Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ pre-dose day 1; pre-dose week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 20
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~pre-dose day 1; pre-dose week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 20
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Percentage of Participants With Hemoglobin (Hb) Response
Secondary outcome measures
Area Under the Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUClast) of Mitapivat
Blood Concentration of 2,3 - diphosphoglycerate (2,3-DPG)
Blood Concentration of Adenosine Triphosphate (ATP)
+20 moreENERGIZE Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: MitapivatExperimental Treatment1 Intervention
Double-blind Period: Participants will receive mitapivat 100 milligrams (mg), orally, twice daily (BID) for 24 weeks.
Open-label Extension Period: Participants who do not discontinue study drug may choose to continue to receive mitapivat for up to an additional 5 years after the Double-blind Period.
Group II: PlaceboPlacebo Group2 Interventions
Double-blind Period: Participants will receive placebo matching mitapivat, orally, BID for 24 weeks.
Open-label Extension Period: Participants who do not discontinue study drug may choose to receive mitapivat for up to an additional 5 years after the Double-blind Period.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Mitapivat
2023
Completed Phase 1
~20
Find a Location
Who is running the clinical trial?
Agios Pharmaceuticals, Inc.Lead Sponsor
52 Previous Clinical Trials
4,012 Total Patients Enrolled
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
39 Previous Clinical Trials
8,366 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not needed more than 5 blood transfusions in the last 6 months and none in the last 2 months.I do not have any health conditions that my doctor thinks would make this study unsafe for me.I am allergic to mitapivat or its ingredients.I have had cancer within the last 5 years.I haven't had blood cell boosters in the last 18 weeks.I have not taken strong CYP3A4/5 inhibitors or inducers for the required time before joining.I haven't had serious heart or lung problems in the last 6 months.I have been diagnosed with thalassemia based on specific blood tests or DNA analysis.My hydroxyurea dose has been the same for at least 16 weeks.My kidney function is reduced, with an eGFR below 45 mL/min/1.73m^2.I have not had major surgery in the last 16 weeks and do not plan any during the study.I have been treated with luspatercept, and my last dose was over 18 weeks ago.I have a documented history of sickle cell disease.I have a liver or bile duct disorder.I have had gene therapy or a bone marrow/stem cell transplant before.My average hemoglobin level is 10 g/dL or less, based on at least 2 tests a week apart.I haven't taken anabolic steroids in the last 4 weeks, but I may be on stable testosterone treatment for low testosterone.
Research Study Groups:
This trial has the following groups:- Group 1: Mitapivat
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any other instances where Mitapivat has been used in a medical capacity?
"The first clinical trial for mitapivat was conducted in 2015 at Stanford University. In the intervening years, there have been a total of 12 completed trials. As of right now, there are 9 active studies being conducted; many of these taking place within New york City."
Answered by AI
Are we currently taking applications for this test?
"That is correct, the listing on clinicaltrials.gov does show that this trial is still recruiting patients. This particular study was posted on November 8th, 2021 and was edited on the same date one year later. They are looking for a total of 171 individuals from 12 different locations to participate."
Answered by AI
Is this trial common in American hospitals?
"12 clinical trial sites are presently recruiting patients for this study. They are based in New york, Palo Alto and Philadelphia as well as 12 other locations. To minimize travel demands, you should select the site closest to you if you participate."
Answered by AI
Has the FDA given Mitapivat their blessing?
"Mitapivat's safety is estimated to be a 3. This comes from the fact that it is a Phase 3 trial, meaning that there are data supporting efficacy and multiple rounds of data supporting safety."
Answered by AI
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