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

Completed · Phase 2

Conditions studied: Sickle Cell Disease

In brief

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

Key facts

Study ID

NCT05861453

Run by

Afimmune

People needed

35

Starts

2024-01-10

Expected to finish

2026-02-11

Last updated by the study team

2026-05-04

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

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.

Where it is running

• University of Alabama at Birmingham (UAB) — Birmingham, Alabama, United States
• New England Sickle Cell Institute, UConn Health — Farmington, Connecticut, United States
• Medstar Health — Washington D.C., District of Columbia, United States
• Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding — Atlanta, Georgia, United States
• Emory University - Georgia Comprehensive Sickle Cell Center — Atlanta, Georgia, United States
• Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital — Atlanta, Georgia, United States
• UI Health Sickle Cell Center — Chicago, Illinois, United States
• The Johns Hopkins University School of Medicine — Baltimore, Maryland, United States
• The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA — Bethesda, Maryland, United States
• Kaiser Permanente Mid-Atlantic States — Largo, Maryland, United States
• Karmanos Cancer Institute — Detroit, Michigan, United States
• Robert Wood Johnson Medical School Rutgers — New Brunswick, New Jersey, United States
• Newark Beth Israel Medical Center — Newark, New Jersey, United States
• Jacobi Medical Center — The Bronx, New York, United States
• UNC Health — Chapel Hill, North Carolina, United States
• Science 37 — Morrisville, North Carolina, United States
• St Paul's Hospital Hematology/Oncology Research — Vancouver, British Columbia, Canada
• Toronto General Hospital — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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