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

Completed · Phase 2

Conditions studied: Sickle Cell Disease, Thalassemia

In brief

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 facts

Study ID

NCT04987489

Run by

Forma Therapeutics, Inc.

People needed

53

Starts

2022-03-28

Expected to finish

2025-09-24

Last updated by the study team

2025-12-16

Who can join

Age: 12 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Provision of consent
• 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

You may not qualify if…

• Female who is breast feeding or pregnant
• 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

Where it is running

• 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
• AORN A. Cardarelli — Naples, Italy
• A.O.U. Università Studi della Campania "Luigi Vanvitelli" — Naples, Italy
• Chronic Care Center — Baabda, Lebanon

Full record on ClinicalTrials.gov

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