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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Placebo Matching Mitapivat — DRUG
  Tablets
• Mitapivat — DRUG
  Tablets

Study Details

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 Dates

Start date

Dec 20, 2021

Status verified

May 2026

Primary completion

Nov 13, 2023

Completion

Dec 31, 2028

Study Design

Enrollment

194 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Mitapivat
  Mitapivat 100 milligrams (mg), orally, twice daily (BID) for 24 weeks in double blind (DB) period and for up to 5 years in open label extension (OLE) period.
• Placebo Comparator: Placebo
  Placebo matching mitapivat, orally, BID for 24 weeks in double blind period followed by Mitapivat 100 mg, orally, BID for up to 5 years in open label extension period.

Primary Outcome Measure

Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline [ Time Frame: Double-Blind Period: Baseline up to Week 12 through Week 24 ]

Locations (6)

Related coverage on Hipa.ai

• Mitapivat Phase 3 for Thalassemia Hits Primary Hemoglobin Response Endpoint
  Mitapivat · Nov 13, 2023 · ClinicalTrials.gov

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