Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients with Sickle Cell Disease
- Registration Number
- NCT05861453
- Lead Sponsor
- Afimmune
- Brief Summary
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
- Detailed Description
The trial will consist of a 28-day screening period, 16 weeks of active treatment and a 30-day post-treatment follow-up period.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 30
-
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Epeleuton 4g/day Epeleuton -
- Primary Outcome Measures
Name Time Method Changes from baseline in Phosphatidylserine 16 Weeks Change in Phosphatidlyserine from baseline to week 16.
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1) 16 Weeks Changes in VCAM-1 from baseline to Week 16
Change from baseline in PROMIS Pain Interference Short Form 16 Weeks Change in PROMIS Pain Interference from baseline to Week 16
Determination of exploratory biomarkers from baseline 16 Weeks Determination of exploratory biomarkers at baseline and Week 16
Changes from baseline in Leukocytes 16 Weeks Changes in Leukocytes from baseline to Week 16
Change from baseline in PROMIS Physical Activity Short Form 16 Weeks Change in PROMIS Physical Activity from baseline to Week 16
Changes from baseline in P-selectin 16 Weeks Change in P-selectin from baseline at Week 16.
Changes from baseline in absolute reticulocyte count 16 Weeks Change in absolute reticulocyte count from baseline at Week 16.
Changes from baseline in RBC Laminin Adhesion 16 Weeks Changes in RBC Laminin Adhesion from baseline to week 16
Changes from baseline in Hemoglobin 16 Weeks Change in hemoglobin from baseline at Week 16
Changes from baseline in Dense Red Blood Cells 16 Weeks Changes in Dense Red Blood Cells from baseline to Week 16
Changes from baseline in Osmoscan 16 Weeks Changes in Osmoscan from baseline to Week 16
Changes from baseline in Oxygen Point of Sickling 16 Weeks Changes in Oxygen Point of Sickling from baseline to Week 16
Changes from baseline in D-dimer 16 Weeks Changes in D-dimer from baseline to Week 16
Changes from baseline in E-selectin 16 Weeks Change in E-selectin from baseline at Week 16.
Changes from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home 16 Weeks Changes in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16
Trough plasma concentrations of total and unesterified 15 HEPE 16 Weeks Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (21)
University of Alabama at Birmingham (UAB)
🇺🇸Birmingham, Alabama, United States
New England Sickle Cell Institute, UConn Health
🇺🇸Farmington, Connecticut, United States
Medstar Health
🇺🇸Washington, 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
UI Health Sickle Cell Center
🇺🇸Chicago, Illinois, United States
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital
🇺🇸Atlanta, Georgia, 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
🇺🇸Bronx, New York, United States
Queens Hospital Center
🇺🇸Jamaica, New York, United States
UNC Health
🇺🇸Chapel Hill, North Carolina, United States
East Carolina University
🇺🇸Greenville, North Carolina, United States
Science 37
🇺🇸Morrisville, North Carolina, United States
Foothills Medical Center
🇨🇦Calgary, Alberta, Canada
St Paul's Hospital Hematology/Oncology Research
🇨🇦Vancouver, British Columbia, Canada
Toronto General Hospital
🇨🇦Toronto, Ontario, Canada