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

NCT05861453 | Completed Phase 2 FDA Drug

• 1 other identifier: DS102A-10-RD2
• Study Type: interventional
• Target: 35
• Locations: 2 countries
• Sites: 18

Brief Summary

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

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (17)

• 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

• Changes from baseline in RBC Laminin Adhesion

  Changes in RBC Laminin Adhesion 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 P-selectin

  Change in P-selectin 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 Dense Red Blood Cells

  Changes in Dense Red Blood Cells 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 Osmoscan

  Changes in Osmoscan from baseline to 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 D-dimer

  Changes in D-dimer from baseline to Week 16

  16 Weeks

• Changes from baseline in Phosphatidylserine

  Change in Phosphatidlyserine from baseline to week 16.

  16 Weeks

• Changes from baseline in Hemoglobin

  Change in hemoglobin from baseline at Week 16

  16 Weeks

• Changes from baseline in Leukocytes

  Changes in Leukocytes 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

Study Arms (1)

Epeleuton 4g/day

EXPERIMENTAL

Drug: Epeleuton

Interventions

Epeleuton DRUG

Participants will receive 2000mg Epeleuton (DS102) capsules twice daily.

Also known as: DS102 Capsules

Epeleuton 4g/day

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with sickle cell disease (SCD) including:
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Afimmune: lead

Study Sites (18)

University of Alabama at Birmingham (UAB)

Birmingham, Alabama, 35294, United States

Location

New England Sickle Cell Institute, UConn Health

Farmington, Connecticut, 06030-1163, United States

Location

Medstar Health

Washington D.C., District of Columbia, 20010, United States

Location

Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding

Atlanta, Georgia, 30303, United States

Location

Emory University - Georgia Comprehensive Sickle Cell Center

Atlanta, Georgia, 30303, United States

Location

Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital

Atlanta, Georgia, 30329, United States

Location

UI Health Sickle Cell Center

Chicago, Illinois, 60612, United States

Location

The Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

Location

The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA

Bethesda, Maryland, 20817, United States

Location

Kaiser Permanente Mid-Atlantic States

Largo, Maryland, 20774, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Robert Wood Johnson Medical School Rutgers

New Brunswick, New Jersey, 08901, United States

Location

Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States

Location

Jacobi Medical Center

The Bronx, New York, 10461, United States

Location

UNC Health

Chapel Hill, North Carolina, 27517, United States

Location

Science 37

Morrisville, North Carolina, 27560, United States

Location

St Paul's Hospital Hematology/Oncology Research

Vancouver, British Columbia, V6E 1M7, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

epeleuton

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2023
• First Posted: May 16, 2023
• Study Start: January 10, 2024
• Primary Completion: January 21, 2026
• Study Completion: February 11, 2026
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (16); CA (2)