A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
NCT ID: NCT04987489
Last Updated: 2025-12-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
53 participants
INTERVENTIONAL
2022-03-28
2025-09-24
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Etavopivat 400 mg daily - SCD with transfusions
Patients with sickle cell disease on chronic red blood cell transfusions
Etavopivat tablets
Etavopivat 400 mg once daily
Etavopivat 400 mg daily - Thalassemia with transfusions
Patients with thalassemia on chronic red blood cell transfusions
Etavopivat tablets
Etavopivat 400 mg once daily
Etavopivat 400 mg daily - Thalassemia
Patients with thalassemia not on chronic red blood cell transfusions
Etavopivat tablets
Etavopivat 400 mg once daily
Interventions
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Etavopivat tablets
Etavopivat 400 mg once daily
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Female patients of childbearing potential must use acceptable methods of contraception, male patients are willing to use barrier methods of contraception
Cohort A (Sickle Cell Disease Transfusion Cohort)
* Confirmed diagnosis of sickle cell disease
* Chronically red blood cell transfused (sample or exchange \[manual or via electrophoresis\]) for primary stroke prevention or due to previous stroke. Chronic red blood cell transfusion is defined as: ≥ 6 red blood cell units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for \> 35 days during that period
* At least 24 months of chronic monthly red blood cell transfusions for secondary stroke prevention/treatment of primary stroke (initial completed overt clinical stroke with documented infarction on brain computed tomography \[CT\] or magnetic resonance imaging \[MRI\])
* Prior to screening OR at least 12 months of chronic RBC transfusions for primary stroke prevention (abnormal TCD) prior to screening
* Documented adequate monthly transfusions with average HbS ≤ 45% (the upper limit of the established academic community standard) for the previous 12 weeks of red blood cell transfusions before the first dose of study treatment
Cohort B (Thalassemia Transfusion Cohort)
* Documented diagnosis of β-thalassemia, Hemoglobin E/ β-thalassemia or Hemoglobin H (α-thalassemia), or other thalassemia variant
* Chronically transfused, defined as: ≥ 6 red blood cell units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for \> 35 days during that period
Cohort C (Thalassemia Non-transfused Cohort)
* Documented diagnosis of β-thalassemia, Hemoglobin E/ β-thalassemia or Hemoglobin H (α-thalassemia), or other thalassemia variant
* Hemoglobin ≤ 10 g/dL
Exclusion Criteria
* Hepatic dysfunction characterized by:
* Alanine aminotransferase (ALT) \> 4.0 × upper limit of normal (ULN)
* Direct bilirubin \> 3.0 × ULN
* History of cirrhosis
* Known human immunodeficiency virus (HIV) positivity
* Active hepatitis B or hepatitis C infection
* Severe renal dysfunction or on chronic dialysis
* History of malignancy within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation.
* Patients with malignancy considered surgically cured are eligible (eg, non- melanoma skin cancer, cancer of the cervix in-situ, ductal carcinoma in situ \[Stage 1\], Grade 1 endometrial cancer)
* History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
* Unstable angina pectoris or myocardial infarction or elective coronary intervention
* Congestive heart failure requiring hospitalization
* Uncontrolled clinically significant arrhythmias
* Symptomatic pulmonary hypertension
12 Years
65 Years
ALL
No
Sponsors
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Forma Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Transparency (dept. 2834)
Role: STUDY_DIRECTOR
Novo Nordisk A/S
Locations
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TOI Clinical Research
Cerritos, California, United States
[Legal] Children's Hospital Los Angeles
Los Angeles, California, United States
University of Californ LA-UCLA
Los Angeles, California, United States
UCSF Oakland Benioff ChildHosp
Oakland, California, United States
[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County
Orange, California, United States
UCI Health
Orange, California, United States
Children's National Health Hospital
Washington D.C., District of Columbia, United States
Children's Hospital of Atlanta
Atlanta, Georgia, United States
[Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.
Detroit, Michigan, United States
Weill Medical College of Cornell University
New York, New York, United States
Duke University
Durham, North Carolina, United States
East Carolina University
Greenville, North Carolina, United States
[Legal] Children's Hospital Medical Center dba Cincinnati Children's
Cincinnati, Ohio, United States
Children's Hosptl Philadelphia
Philadelphia, Pennsylvania, United States
Master Centre for Canada
Mississauga, Ontario, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
University Health Network - Toronto General Hospital
Toronto, Ontario, Canada
CHU Sainte-Justine Mother and Child University Hospital
Montreal, Quebec, Canada
Cairo University
Cairo, , Egypt
Abu El-Reesh El-Mounira Children University Hospital
Cairo, Egypt, , Egypt
Galliera Hospital Centro Anemie Congenite
Genova, , Italy
Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico
Milan, , Italy
A.O.U. Università Studi della Campania "Luigi Vanvitelli"
Naples, , Italy
AORN A. Cardarelli
Napoli, , Italy
Chronic Care Center
Baabda, , Lebanon
Hospital Nini
Tripoli, , Lebanon
Sandwell and West Birmingham NHS Trust SCAT/ haematology
Birmingham, , United Kingdom
Barts Health NHS Trust - The Royal London Hospital
London, , United Kingdom
University College Hospital - University College London Hospitals NHS Foundation Trust
London, , United Kingdom
Kings College London
London, , United Kingdom
Hammersmith Hospital - London
London, , United Kingdom
Manchester University NHS Foundation Trust
Manchester, , United Kingdom
Countries
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Other Identifiers
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4202-HEM-201
Identifier Type: -
Identifier Source: org_study_id