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

An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease

To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.

The trial will consist of a 28-day screening period, 16 weeks of active treatment and a 30-day post-treatment follow-up period.

Inclusion Criteria:

• Patients with sickle cell disease (SCD) including:

  • 2 sickle hemoglobin genes [HbSS]
  • HbSβ0 thalassemia
  • HbSβ+ thalassemia
  • Heterozygous for hemoglobin S and hemoglobin C [HbSC]

• 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 15 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.

Exclusion Criteria:

• 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 baseline visit
• 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.