Evaluating Epeleuton for treating Sickle Cell Disease
An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients with Sickle Cell Disease
PHASE2 · Afimmune · NCT05861453
This study is testing a new medication called Epeleuton to see how it works and if it helps adults with sickle cell disease feel better.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Afimmune (industry) |
| Locations | 21 sites (Birmingham, Alabama and 20 other locations) |
| Trial ID | NCT05861453 on ClinicalTrials.gov |
What this trial studies
This trial assesses the pharmacokinetics, pharmacodynamics, and safety of Epeleuton capsules in adults with sickle cell disease. It includes a 28-day screening period, followed by 16 weeks of active treatment, and a 30-day post-treatment follow-up. The study aims to determine how the drug behaves in the body and its effects on patients suffering from this condition.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with sickle cell disease who have experienced between 2 and 15 vaso-occlusive crises in the past year.
Not a fit: Patients receiving regular blood transfusions or those who have had a transfusion within the past 30 days may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of sickle cell disease and reduce the frequency of painful crises.
How similar studies have performed: Other studies have explored treatments for sickle cell disease, but the specific approach with Epeleuton is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Birmingham, Alabama and 20 other locations
- University of Alabama at Birmingham (UAB) — Birmingham, Alabama, United States (RECRUITING)
- New England Sickle Cell Institute, UConn Health — Farmington, Connecticut, United States (RECRUITING)
- Medstar Health — Washington, District of Columbia, United States (RECRUITING)
- Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding — Atlanta, Georgia, United States (RECRUITING)
- Emory University - Georgia Comprehensive Sickle Cell Center — Atlanta, Georgia, United States (RECRUITING)
- Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital — Atlanta, Georgia, United States (RECRUITING)
- UI Health Sickle Cell Center — Chicago, Illinois, United States (RECRUITING)
- The Johns Hopkins University School of Medicine — Baltimore, Maryland, United States (NOT_YET_RECRUITING)
- The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA — Bethesda, Maryland, United States (RECRUITING)
- Kaiser Permanente Mid-Atlantic States — Largo, Maryland, United States (RECRUITING)
- Karmanos Cancer Institute — Detroit, Michigan, United States (RECRUITING)
- Robert Wood Johnson Medical School Rutgers — New Brunswick, New Jersey, United States (RECRUITING)
- Newark Beth Israel Medical Center — Newark, New Jersey, United States (RECRUITING)
- Jacobi Medical Center — Bronx, New York, United States (RECRUITING)
- Queens Hospital Center — Jamaica, New York, United States (NOT_YET_RECRUITING)
- UNC Health — Chapel Hill, North Carolina, United States (RECRUITING)
- East Carolina University — Greenville, North Carolina, United States (NOT_YET_RECRUITING)
- Science 37 — Morrisville, North Carolina, United States (RECRUITING)
- Foothills Medical Center — Calgary, Alberta, Canada (RECRUITING)
- St Paul's Hospital Hematology/Oncology Research — Vancouver, British Columbia, Canada (RECRUITING)
- Toronto General Hospital — Toronto, Ontario, Canada (RECRUITING)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Sickle Cell Disease