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

NCT04770779 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 258

Last updated 2026-04-24

Study results available
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Summary

The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.

Conditions

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
2021-11-30
Primary Completion
2024-04-11
Completion
2029-06-30
FDA Drug
Yes

Countries

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

Study Locations

More Related Trials

Entities

Diseases

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 NCT04770779 on ClinicalTrials.gov