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

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Agios Pharmaceuticals, Inc.
Study ID
NCT04770779
Phase
PHASE3
Status
Active Not Recruiting

Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Nov 30, 2021
Status verified
May 2026
Primary completion
Apr 11, 2024
Completion
Jun 30, 2029

Study Design

Enrollment
258 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Mitapivat
    Participants randomized to receive mitapivat 100 milligrams (mg), orally, twice daily (BID) for 48 weeks in the double-blind (DB) period and for up to 5 years in the open label extension (OLE) period.
  • Placebo Comparator: Placebo
    Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the DB period followed by mitapivat 100 mg, orally, BID for up to 5 years in the OLE period.

Primary Outcome Measure

Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) [ Time Frame: Double-blind Period: Baseline through Week 48 ]

Locations (10)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016-7710-
San Diego Hospital, UC San Diego HealthLa JollaCalifornia92093-
Children's Hospital OaklandOaklandCalifornia94609-1809-
Stanford MedicinePalo AltoCalifornia94304-1601-
Boston Children's HospitalBostonMassachusetts02115-
Children's Hospital of MichiganDetroitMichigan48201-2196-
Weill Cornell Medical CenterNew YorkNew York10065-4870-
Duke University Medical CenterDurhamNorth Carolina27710-3038-
Penn Medicine - University of Pennsylvania Health SystemPhiladelphiaPennsylvania19104-
Seattle Cancer Care Alliance, University of WashingtonSeattleWashington98101-

Related coverage on Hipa.ai

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Phoenix Children's Hospital· Phoenix, AZSan Diego Hospital, UC San Diego Health· La Jolla, CAChildren's Hospital Oakland· Oakland, CAStanford Medicine· Palo Alto, CABoston Children's Hospital· Boston, MAChildren's Hospital of Michigan· Detroit, MI

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