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Activator of Pyruvate Kinase-R

Mitapivat for Thalassemia (ENERGIZE-T 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
Considered transfusion-dependent, defined as 6 to 20 RBC units transfused and ≤6-week transfusion-free period during the 24-week period before randomization
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 deoxyribonucleic acid (DNA) analysis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre-dose day 1; pre-dose week 12; pre-dose week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 36
Awards & highlights

ENERGIZE-T Trial Summary

This trial will compare the effect of the drug mitapivat versus a placebo on transfusion burden in people with transfusion-dependent alpha- or beta-thalassemia.

Who is the study for?
This trial is for people with transfusion-dependent alpha- or beta-thalassemia who need regular blood transfusions. They must have a confirmed diagnosis, stable treatment if using hydroxyurea, and agree to use effective contraception if applicable. Excluded are those with certain medical conditions, recent treatments that could interfere, or risks that might affect study results.Check my eligibility
What is being tested?
The trial is testing the effectiveness of Mitapivat versus a placebo in reducing the need for blood transfusions in patients with thalassemia. Participants will be randomly assigned to receive either Mitapivat or a placebo to compare outcomes between the two groups.See study design
What are the potential side effects?
While specific side effects of Mitapivat aren't listed here, common ones may include digestive issues, headaches, fatigue, and potential allergic reactions. The severity can vary from person to person.

ENERGIZE-T Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have needed 6 to 20 blood transfusions in the last 6 months without going more than 6 weeks without one.
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I have been diagnosed with thalassemia based on DNA analysis.
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My hydroxyurea dose has been the same for at least 16 weeks.

ENERGIZE-T Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~pre-dose day 1; pre-dose week 12; pre-dose week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 36
This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-dose day 1; pre-dose week 12; pre-dose week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 36 for reporting.

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 Transfusion Reduction Response (TRR)
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)
+16 more

ENERGIZE-T 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 48 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 48 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
3,958 Total Patients Enrolled
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
39 Previous Clinical Trials
8,302 Total Patients Enrolled

Media Library

Mitapivat (Activator of Pyruvate Kinase-R) Clinical Trial Eligibility Overview. Trial Name: NCT04770779 — Phase 3
Beta Thalassemia Research Study Groups: Placebo, Mitapivat
Beta Thalassemia Clinical Trial 2023: Mitapivat Highlights & Side Effects. Trial Name: NCT04770779 — Phase 3
Mitapivat (Activator of Pyruvate Kinase-R) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04770779 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many different staff members are managing this research project?

"There are 17 sites across the country where this trial is taking place. If you enroll in the study, pick a location that is close to minimize travel requirements."

Answered by AI

Are there any opportunities for patients to enroll in this research project?

"From what is published on clinicaltrials.gov, it appears that this experiment is still looking for individuals to take part in it. The trial was first advertised on 11/30/2021 and the most recent update was 11/8/2022."

Answered by AI

Could you please provide a list of other clinical trials that have utilized Mitapivat?

"The first study investigating mitapivat was completed in 2015 at Stanford University. As of now, 12 studies have been wrapped up with 9 more currently underway. The majority of these ongoing investigations are taking place out of Philadelphia, Pennsylvania."

Answered by AI

Could you share how many test subjects are involved in this experiment?

"The information available on clinicaltrials.gov supports that this study is looking for volunteers. The trial was originally posted on November 30th, 2021 and the most recent update occurred on November 8th, 2022. A total of 240 patients will be accepted at 17 different sites."

Answered by AI

Has this investigation been done before?

"Agios Pharmaceuticals, Inc. first sponsored a clinical trial for Mitapivat in 2015. Since the initial study with 52 patients, there have been 9 more trials conducted in 54 cities across 28 countries."

Answered by AI

Are there any severe side effects associated with taking Mitapivat?

"Mitapivat's safety was given a 3 because there is clinical evidence affirming its efficacy and multiple rounds of data to support its safety."

Answered by AI
~0 spots leftby Apr 2024