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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

Must not have

Estimated glomerular filtration rate <45 milliliters per minute (mL/min)/1.73 meter (m)^2 by Chronic Kidney Disease Epidemiology Collaboration creatinine equation

Documented history of homozygous or heterozygous sickle hemoglobin (Hb S) or hemoglobin C (Hb C)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to week 317

Awards & highlights

Pivotal 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.

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My kidney function is reduced, with an eGFR less than 45 mL/min/1.73m^2.

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I have a confirmed diagnosis of sickle cell disease or hemoglobin C disease.

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I am currently on medication for an infection.

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I haven't had blood cell boosters in the last 36 weeks.

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I haven't taken anabolic steroids in the last 4 weeks, but I may be on stable testosterone treatment for low testosterone.

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I am allergic to mitapivat or its ingredients.

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I have had gene therapy or a bone marrow/stem cell transplant before.

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I have not taken strong CYP3A4/5 inhibitors or inducers for the required time before joining.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to week 317

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to week 317

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 317 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of Participants With Transfusion Reduction Response (TRR)

Secondary study objectives

Percentage of Participants With Transfusion-Independence

Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), Graded by Severity

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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

0 / 11Eligibility criteria met