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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.
Full description
Etavopivat is a potent, selective, orally bioavailable, small-molecule activator of pyruvate kinase red blood cell (PKR) being developed by Forma Therapeutics, Inc and is intended for use as a treatment for patients with sickle cell disease (SCD) or other inherited hemoglobinopathies or refractory anemias. This study is a multicenter, Phase 2, open-label, multiple-cohort study examining the safety and efficacy of etavopivat for the treatment of patients, age 12 to 65 years, with SCD or thalassemia. Three treatment cohorts based on the patients hemoglobinopathy (SCD or thalassemia) and transfusion requirements will be evaluated.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Cohort A (Sickle Cell Disease Transfusion Cohort)
Cohort B (Thalassemia Transfusion Cohort)
Cohort C (Thalassemia Non-transfused Cohort)
Exclusion criteria
Female who is breast feeding or pregnant
Hepatic dysfunction characterized by:
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.
History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
Primary purpose
Allocation
Interventional model
Masking
60 participants in 3 patient groups
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Central trial contact
Novo Nordisk
Data sourced from clinicaltrials.gov
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