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

Description

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

Conditions

Sickle Cell Disease

Talk to us

Study Overview

On this page

Study Details

Study overview

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

An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients with Sickle Cell Disease

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

Condition
Sickle Cell Disease
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama at Birmingham (UAB), Birmingham, Alabama, United States, 35294

Farmington

New England Sickle Cell Institute, UConn Health, Farmington, Connecticut, United States, 06030-1163

Washington

Medstar Health, Washington, District of Columbia, United States, 20010

Atlanta

Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding, Atlanta, Georgia, United States, 30303

Atlanta

Emory University - Georgia Comprehensive Sickle Cell Center, Atlanta, Georgia, United States, 30303

Atlanta

Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital, Atlanta, Georgia, United States, 30329

Chicago

UI Health Sickle Cell Center, Chicago, Illinois, United States, 60612

Baltimore

The Johns Hopkins University School of Medicine, Baltimore, Maryland, United States, 21205

Bethesda

The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA, Bethesda, Maryland, United States, 20817

Largo

Kaiser Permanente Mid-Atlantic States, Largo, Maryland, United States, 20774

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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.

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Afimmune,

Study Record Dates

2025-09