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

NCT04770753 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 194

Last updated 2026-04-24

Study results available
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Summary

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).

Conditions

  • Non-Transfusion-dependent Alpha-Thalassemia
  • Non-Transfusion-dependent Beta-Thalassemia

Interventions

DRUG

Placebo Matching Mitapivat

Tablets

DRUG

Mitapivat

Tablets

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Affairs · Agios Pharmaceuticals, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-20
Primary Completion
2023-11-13
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Bulgaria
  • Canada
  • Denmark
  • France
  • Greece
  • Italy
  • Lebanon
  • Malaysia
  • Netherlands
  • Saudi Arabia
  • Spain
  • Taiwan
  • Thailand
  • Turkey (Türkiye)
  • United Arab Emirates
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04770753 on ClinicalTrials.gov