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

April 28, 2026 updated by: Afimmune

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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
35

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Changes from baseline in P-selectin
Time Frame: 16 Weeks
Change in P-selectin from baseline at Week 16.
16 Weeks
Changes from baseline in Hemoglobin
Time Frame: 16 Weeks
Change in hemoglobin from baseline at Week 16
16 Weeks
Changes from baseline in absolute reticulocyte count
Time Frame: 16 Weeks
Change in absolute reticulocyte count from baseline at Week 16.
16 Weeks
Changes from baseline in E-selectin
Time Frame: 16 Weeks
Change in E-selectin from baseline at Week 16.
16 Weeks
Changes from baseline in Phosphatidylserine
Time Frame: 16 Weeks
Change in Phosphatidlyserine from baseline to week 16.
16 Weeks
Changes from baseline in RBC Laminin Adhesion
Time Frame: 16 Weeks
Changes in RBC Laminin Adhesion from baseline to week 16
16 Weeks
Changes from baseline in Leukocytes
Time Frame: 16 Weeks
Changes in Leukocytes from baseline to Week 16
16 Weeks
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
Time Frame: 16 Weeks
Changes in VCAM-1 from baseline to Week 16
16 Weeks
Changes from baseline in Dense Red Blood Cells
Time Frame: 16 Weeks
Changes in Dense Red Blood Cells from baseline to Week 16
16 Weeks
Changes from baseline in Osmoscan
Time Frame: 16 Weeks
Changes in Osmoscan from baseline to Week 16
16 Weeks
Changes from baseline in Oxygen Point of Sickling
Time Frame: 16 Weeks
Changes in Oxygen Point of Sickling from baseline to Week 16
16 Weeks
Changes from baseline in D-dimer
Time Frame: 16 Weeks
Changes in D-dimer from baseline to Week 16
16 Weeks
Change from baseline in PROMIS Pain Interference Short Form
Time Frame: 16 Weeks
Change in PROMIS Pain Interference from baseline to Week 16
16 Weeks
Change from baseline in PROMIS Physical Activity Short Form
Time Frame: 16 Weeks
Change in PROMIS Physical Activity from baseline to Week 16
16 Weeks
Trough plasma concentrations of total and unesterified 15 HEPE
Time Frame: 16 Weeks
Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16
16 Weeks
Determination of exploratory biomarkers from baseline
Time Frame: 16 Weeks
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
Time Frame: 16 Weeks
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

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Afimmune

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 10, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 21, 2026

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 11, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 26, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 15, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 16, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 4, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 28, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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