A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) (ENERGIZE-T)

  • STATUS
    Recruiting
  • End date
    Jun 13, 2024
  • participants needed
    240
  • sponsor
    Agios Pharmaceuticals, Inc.
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Updated on 13 October 2022
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Summary

The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).

Description

The mitapivat group will include approximately 160 participants. The placebo group will include approximately 80 participants.

Details
Condition Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
Treatment Mitapivat, Placebo Matching Mitapivat
Clinical Study IdentifierNCT04770779
SponsorAgios Pharmaceuticals, Inc.
Last Modified on13 October 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

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
Considered transfusion-dependent, defined as 6 to 20 RBC units transfused and ≤6-week transfusion-free period during the 24-week period before randomization
If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization
Women of childbearing potential (WOCBP) and men with partners who are WOCBP must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle or agree to use two forms of contraception, one of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug for women and 90 days after the last dose of study drug for men. The second form of contraception can be an acceptable barrier method
Written informed consent before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study

Exclusion Criteria

Pregnant or breastfeeding
Documented history of homozygous or heterozygous sickle hemoglobin (Hb S) or hemoglobin C (Hb C)
Prior exposure to gene therapy or prior bone marrow or stem cell transplantation
Currently receiving treatment with luspatercept; the last dose must have been administered ≥36 weeks before randomization
Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥36 weeks before randomization
History of any malignancy, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ. Subjects must not have active disease or received anticancer treatment ≤5 years before providing informed consent
History of active and/or uncontrolled cardiac or pulmonary disease ≤6 months before providing informed consent
Hepatobiliary disorders
Estimated glomerular filtration rate <45 milliliters per minute (mL/min)/1.73 meter (m)^2 by Chronic Kidney Disease Epidemiology Collaboration creatinine equation
Nonfasting triglycerides >440 milligrams per deciliter (mg/dL) (5 millimoles per liter [mmol/L])
Active infection requiring systemic antimicrobial therapy at the time of providing informed consent
Positive test for hepatitis C virus antibody (HCVAb) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg)
Positive test for human immunodeficiency virus (HIV)-1 antibody (Ab) or HIV-2 Ab
History of major surgery (including splenectomy) ≤6 months before providing informed consent and/or a major surgical procedure planned during the study
Current enrollment or past participation (≤12 weeks before administration of the first dose of study drug or a timeframe equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational treatment or device
Receiving strong CYP3A4/5 inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer); or strong CYP3A4 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization
Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed. The testosterone dose and preparation must be stable for ≥12 weeks before randomization
Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate)
Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data
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