Haematological Disorders
Conditions
Interventions
Treatment group
Placebo group
Sponsors
Forma Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient has provided documented informed consent or assent (the informed consent form [ICF] must be reviewed and signed by each patient; in the case of adolescent patients, both the consent of the patient’s legal representative or legal guardian, and the patient’s assent must be obtained) 2. Age 3. Patient has a confirmed diagnosis of sickle cell disease • Documentation of SCD genotype (HbSS, HbSß0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing; if unavailable, must be confirmed by laboratory testing during screening 4. Patient has had at least 2 episodes of VOC in the past 12 months • For study eligibility, VOC is defined as a previously documented episode of ACS or acute painful crisis (for which there was no explanation other than VOC) which required prescription or healthcare professional-instructed use of analgesics for moderate to severe pain (documentation must exist in the patient medical record prior to Screening) 5. Hemoglobin = 5.5 and = 10 g/dL (= 55 and = 100 g/L) during screening 6. For participants taking HU, the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator 7. Patients, who if female and of child bearing potential, are using highly effective methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male are willing to use barrier methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.
Exclusion criteria
Exclusion criteria: 1. More than 10 VOCs (as defined in Inclusion Criterion 4) within the past 12 months 2. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF 3. Female who is breast feeding or pregnant 4. Hepatic dysfunction characterized by: • Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN) • Direct bilirubin > 3.0 × ULN 5. Patients with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy • Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening/enrollment until active therapy has been completed Note: Infection prophylaxis is allowed (see concomitant medication restrictions) 6. Known human immunodeficiency virus (HIV) positivity 7. Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive) 8. Active hepatitis C infection 9. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory < 30 mL/min/1.73 m2) or on chronic dialysis 10. 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) 11. 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. History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage 13. Among others
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. To assess the efficacy of etavopivat in adolescents and adults with SCD as compared to placebo as measured by improvement in hemoglobin (Hb) 2. To assess the efficacy of etavopivat as compared to placebo on the annualized vaso-occlusive crisis (VOC) rate | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. To measure the effects of etavopivat on clinical measures and sequelae of hemolysis 2. To evaluate the effects of etavopivat on the sequelae of VOC 3. To assess changes in fatigue of sickle cell patients taking etavopivat | — |
Countries
Egypt, Ghana, Kenya, Nigeria
Contacts
Public ContactEdeghonghon Olayemi
Consultant Haematologist Associate Professor
Outcome results
None listed