A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Part of paid clinical trials in Cerritos, California.

Sponsor
Forma Therapeutics, Inc.
Study ID
NCT04987489
Phase
PHASE2
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin.

Key Dates

Start date
Mar 28, 2022
Status verified
Dec 2025
Primary completion
Sep 3, 2025
Completion
Sep 24, 2025

Study Design

Enrollment
53 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Etavopivat 400 mg daily - SCD with transfusions
    Patients with sickle cell disease on chronic red blood cell transfusions
  • Experimental: Etavopivat 400 mg daily - Thalassemia with transfusions
    Patients with thalassemia on chronic red blood cell transfusions
  • Experimental: Etavopivat 400 mg daily - Thalassemia
    Patients with thalassemia not on chronic red blood cell transfusions

Primary Outcome Measure

Cohorts A: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history [ Time Frame: 12 weeks ]

Locations (14)

FacilityCityStateZIPSite coordinators
TOI Clinical ResearchCerritosCalifornia90703-
[Legal] Children's Hospital Los AngelesLos AngelesCalifornia90027-
University of Californ LA-UCLALos AngelesCalifornia90095-
UCSF Oakland Benioff ChildHospOaklandCalifornia94609-
[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange CountyOrangeCalifornia92868-
UCI HealthOrangeCalifornia92868-
Children's National Health HospitalWashington D.C.District of Columbia20010-
Children's Hospital of AtlantaAtlantaGeorgia30342-
[Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.DetroitMichigan48201-2018-
Weill Medical College of Cornell UniversityNew YorkNew York10065-
Duke UniversityDurhamNorth Carolina27710-
East Carolina UniversityGreenvilleNorth Carolina27858-
[Legal] Children's Hospital Medical Center dba Cincinnati Children'sCincinnatiOhio45229-
Children's Hosptl PhiladelphiaPhiladelphiaPennsylvania19104-

Find similar trials in Cerritos, CA

By condition
Sickle cell disease
By research site
TOI Clinical Research· Cerritos, CA[Legal] Children's Hospital Los Angeles· Los Angeles, CAUniversity of Californ LA-UCLA· Los Angeles, CAUCSF Oakland Benioff ChildHosp· Oakland, CA[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County· Orange, CAUCI Health· Orange, CA

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