A study to assess the pharmacokinetics, pharmacodynamics, & safety of study drug (Epeleuton) in patients with sickle cell disease

Purpose of trial

The purpose of this study is to investigate the pharmacokinetic, pharmacodynamic, and safety of orally administrated Epeleuton in patients with Sickle Cell Disease.

There is a clear need for additional disease-modifying therapeutic options for SCD and Epeleuton, has a novel mechanism of action and has demonstrated effects in preclinical studies on a SCD humanized mouse model and SCD patient red blood cells (RBCs), indicating that it simultaneously targets multiple aspects of the underlying pathophysiology of SCD including:

1. Red blood cell health and hemolytic anemia
2. Vascular inflammation and endothelial adhesion
3. Organ damage

This treatment could provide a safe, orally administered and readily accessible alternative disease modifying therapy for patients with this debilitating disease.

Eligibility

Key Inclusion Criteria:

• Patients with Sickle Cell Disease
• Male or Female patients aged 18 years and older on the day of signing the ICF
• Patients who have had between 2 and 10 episodes of vaso-occlusive crisis in the past year
• Are able to understand and voluntarily sign an informed consent form prior to any study-related assessments/procedures being conducted
• Are willing and able to adhere to the study visit schedule and other protocol requirements
• If you are a woman, you cannot be pregnant or breastfeeding an infant

Key Exclusion Criteria:

• Patients who are receiving regularly scheduled blood transfusion therapy
• Patients who have received a hematopoietic stem cell transplant
• Known positive status for HIV or active hepatitis A, B, or C
• Men who are planning on conceiving a child with their partner during the duration of the study