A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia

In brief

The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

Key facts

Study ID

NCT04770753

Run by

Agios Pharmaceuticals, Inc.

People needed

194

Starts

2021-12-20

Expected to finish

2028-12-01

Last updated by the study team

2026-07-31

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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;
• Hb concentration ≤10.0 grams per deciliter (g/dL) (100.0 grams per liter [g/L]), based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period;
• 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;
• If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization;
• Women of child-bearing potential (WOCBP) must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 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. 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.

You may not qualify if…

• Pregnant, breastfeeding, or parturient
• Documented history of homozygous or heterozygous sickle hemoglobin (HbS) or hemoglobin C (HbC);
• 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 ≥18 weeks before randomization;
• Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization;
• History of malignancy, (active or treated) ≤5 years before providing informed consent;
• History of active and/or uncontrolled cardiac or pulmonary disease ≤6 months before providing informed consent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ;
• Hepatobiliary disorders;
• Estimated glomerular filtration rate <45 milliliters per minute (mL/min)/1.73 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) ≤16 weeks 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 ≥10 weeks before randomization;
• Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate, Opadry® II Blue [hypromellose, titanium dioxide, lactose monohydrate, triacetin, and FD\&C Blue #2]);
• 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 Also excluded are:
• Participants who are institutionalized by regulatory or court order
• Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor)

Where it is running

• Stanford Medicine — Palo Alto, California, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Weill Cornell Medical Center — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Penn Medicine - University of Pennsylvania Health System — Philadelphia, Pennsylvania, United States
• Universidade de Caxias do Sul — Caxias do Sul, Brazil
• Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP — Ribeirão Preto, Brazil
• HEMORIO Instituto Nacional de Hematologia — Rio de Janeiro, Brazil
• Praxis Pesquisa Medica — Santo André, Brazil
• GSH Banco de Sangue de São Paulo — São Paulo, Brazil
• Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo — São Paulo, Brazil
• MHAT "Dr. Nikola Vasiliev" AD — Kyustendil, Bulgaria
• SHATHD Sofia — Sofia, Bulgaria
• Toronto General Hospital, University Health Network — Toronto, Canada
• Rigshospitalet — Copenhagen, Denmark
• CHU Hôpital Henri Mondor — Créteil, France
• Hopital Edouard Herriot, CHU de Lyon — Lyon, France
• Laiko General Hospital — Athens, Greece
• Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School — Athens, Greece
• University General Hospital of Patras — Rio, Greece
• Ippokrateio General Hospital — Thessaloniki, Greece
• Ospedale "A. Perrino" - Brindisi — Brindisi, Italy
• Ospedale Pediatrico Microcitemico — Cagliari, Italy
• Ospedale Sant'Anna — Ferrara, Italy
• San Diego Hospital, UC San Diego Health — La Jolla, California, United States

Full record on ClinicalTrials.gov

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