Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease

Open to people ages 18 years and up

• Patients with sickle cell disease (SCD) including: 2 sickle hemoglobin genes [HbSS] and HbSβ0-thalassemia
• Male or female patients aged 18 years and older on the day of signing the informed consent form (ICF)
• Patients who have had between 2 and 10 episodes of vaso-occlusive crisis (VOC) in the past year (12 months)
• For patients taking hydroxyurea (HU), the dose of HU must be stable for at least 3 months prior to signing the ICD and with no anticipated need for dose adjustment during the study.
• Female patients and male patients with female partners of childbearing potential must use highly effective contraceptive methods for the duration of the study.

• Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion), have received an RBC transfusion for any reason within three months of the randomization visit (baseline/day 0) or have a hemoglobin A level >20% of the total hemoglobin.
• Patients who have received a hematopoietic stem cell transplant.
• Patients with inadequate venous access as determined by the Investigator
• Patients who are pregnant, planning pregnancy, breastfeeding and/or are unwilling to use adequate contraception during the trial.