ACTIVE_NOT_RECRUITING

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

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2024-01-10

Study Completion:2026-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05861453

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.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.

Inclusion Criteria

Exclusion 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.

Contacts and Locations

Study Locations (Sites)

University of Alabama at Birmingham (UAB)

Birmingham, Alabama, 35294

United States

New England Sickle Cell Institute, UConn Health

Farmington, Connecticut, 06030-1163

United States

Medstar Health

Washington D.C., District of Columbia, 20010

United States

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

Atlanta, Georgia, 30303

United States

Emory University - Georgia Comprehensive Sickle Cell Center

Atlanta, Georgia, 30303

United States

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

Atlanta, Georgia, 30329

United States

UI Health Sickle Cell Center

Chicago, Illinois, 60612

United States

The Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205

United States

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

Bethesda, Maryland, 20817

United States

Kaiser Permanente Mid-Atlantic States

Largo, Maryland, 20774

United States

Karmanos Cancer Institute

Detroit, Michigan, 48201

United States

Robert Wood Johnson Medical School Rutgers

New Brunswick, New Jersey, 08901

United States

Newark Beth Israel Medical Center

Newark, New Jersey, 07112

United States

Queens Hospital Center

Jamaica, New York, 11432

United States

Jacobi Medical Center

The Bronx, New York, 10461

United States

UNC Health

Chapel Hill, North Carolina, 27517

United States

East Carolina University

Greenville, North Carolina, 27834

United States

Science 37

Morrisville, North Carolina, 27560

United States

Collaborators and Investigators

Sponsor: 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 Registration Dates

Study Start Date2024-01-10

Study Completion Date2026-03

Study Record Updates

Study Start Date2024-01-10

Study Completion Date2026-03

Terms related to this study

Additional Relevant MeSH Terms

Sickle Cell Disease