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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Epeleuton

Detailed Description

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

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V6E 1M7
      • St Paul's Hospital Hematology/Oncology Research
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Toronto General Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama at Birmingham (UAB)
  • Connecticut
    • Farmington, Connecticut, United States, 06030-1163
      • New England Sickle Cell Institute, UConn Health
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • MedStar Health
  • Georgia
    • Atlanta, Georgia, United States, 30303
      • Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding
    • Atlanta, Georgia, United States, 30303
      • Emory University - Georgia Comprehensive Sickle Cell Center
    • Atlanta, Georgia, United States, 30329
      • Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital
  • Illinois
    • Chicago, Illinois, United States, 60612
      • UI Health Sickle Cell Center
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • The Johns Hopkins University School of Medicine
    • Bethesda, Maryland, United States, 20817
      • The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA
    • Largo, Maryland, United States, 20774
      • Kaiser Permanente Mid-Atlantic States
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Karmanos Cancer Institute
  • New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Robert Wood Johnson Medical School Rutgers
    • Newark, New Jersey, United States, 07112
      • Newark Beth Israel Medical Center
  • New York
    • The Bronx, New York, United States, 10461
      • Jacobi Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27517
      • UNC Health
    • Morrisville, North Carolina, United States, 27560
      • Science 37

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Epeleuton 4g/day	Drug: Epeleuton; Participants will receive 2000mg Epeleuton (DS102) capsules twice daily.; Other Names: DS102 Capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes from baseline in P-selectin	Change in P-selectin from baseline at Week 16.	16 Weeks
Changes from baseline in Hemoglobin	Change in hemoglobin from baseline at Week 16	16 Weeks
Changes from baseline in absolute reticulocyte count	Change in absolute reticulocyte count from baseline at Week 16.	16 Weeks
Changes from baseline in E-selectin	Change in E-selectin from baseline at Week 16.	16 Weeks
Changes from baseline in Phosphatidylserine	Change in Phosphatidlyserine from baseline to week 16.	16 Weeks
Changes from baseline in RBC Laminin Adhesion	Changes in RBC Laminin Adhesion from baseline to week 16	16 Weeks
Changes from baseline in Leukocytes	Changes in Leukocytes from baseline to Week 16	16 Weeks
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)	Changes in VCAM-1 from baseline to Week 16	16 Weeks
Changes from baseline in Dense Red Blood Cells	Changes in Dense Red Blood Cells from baseline to Week 16	16 Weeks
Changes from baseline in Osmoscan	Changes in Osmoscan from baseline to Week 16	16 Weeks
Changes from baseline in Oxygen Point of Sickling	Changes in Oxygen Point of Sickling from baseline to Week 16	16 Weeks
Changes from baseline in D-dimer	Changes in D-dimer from baseline to Week 16	16 Weeks
Change from baseline in PROMIS Pain Interference Short Form	Change in PROMIS Pain Interference from baseline to Week 16	16 Weeks
Change from baseline in PROMIS Physical Activity Short Form	Change in PROMIS Physical Activity from baseline to Week 16	16 Weeks
Trough plasma concentrations of total and unesterified 15 HEPE	Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16	16 Weeks
Determination of exploratory biomarkers from baseline	Determination of exploratory biomarkers at baseline and Week 16	16 Weeks
Change from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home	Change in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16	16 Weeks

Collaborators and Investigators

• Sponsor: Afimmune

Study record dates

Study Major Dates

• Study Start (Actual): January 10, 2024
• Primary Completion (Actual): January 21, 2026
• Study Completion (Actual): February 11, 2026

Study Registration Dates

• First Submitted: April 26, 2023
• First Submitted That Met QC Criteria: May 15, 2023
• First Posted (Actual): May 16, 2023

Study Record Updates

• Last Update Posted (Actual): May 4, 2026
• Last Update Submitted That Met QC Criteria: April 28, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell; epeleuton
• Other Study ID Numbers: DS102A-10-RD2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No