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

NCT ID: NCT05861453

Last Updated: 2025-10-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-01-10

Study Completion Date

2026-03-31

Brief Summary

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

Detailed Description

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

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Epeleuton 4g/day

Group Type EXPERIMENTAL

Epeleuton

Intervention Type DRUG

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

Interventions

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Epeleuton

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

Intervention Type DRUG

Other Intervention Names

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DS102 Capsules

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Afimmune

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham (UAB)

Birmingham, Alabama, United States

Site Status

New England Sickle Cell Institute, UConn Health

Farmington, Connecticut, United States

Site Status

Medstar Health

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

Site Status

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

Atlanta, Georgia, United States

Site Status

Emory University - Georgia Comprehensive Sickle Cell Center

Atlanta, Georgia, United States

Site Status

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

Atlanta, Georgia, United States

Site Status

UI Health Sickle Cell Center

Chicago, Illinois, United States

Site Status

The Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

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

Bethesda, Maryland, United States

Site Status

Kaiser Permanente Mid-Atlantic States

Largo, Maryland, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Robert Wood Johnson Medical School Rutgers

New Brunswick, New Jersey, United States

Site Status

Newark Beth Israel Medical Center

Newark, New Jersey, United States

Site Status

Jacobi Medical Center

The Bronx, New York, United States

Site Status

UNC Health

Chapel Hill, North Carolina, United States

Site Status

Science 37

Morrisville, North Carolina, United States

Site Status

St Paul's Hospital Hematology/Oncology Research

Vancouver, British Columbia, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Countries

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United States Canada

Other Identifiers

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DS102A-10-RD2

Identifier Type: -

Identifier Source: org_study_id

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