Clinical trial NCT05861453: Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients with Sickle Cell Disease
Official title: An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients with Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Sickle Cell Disease
Lead sponsor: Afimmune
Sponsor class: INDUSTRY
First posted: 2023-05-16
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Changes from baseline in P-selectin
timeFrame: 16 Weeks
measure: Changes from baseline in Hemoglobin
timeFrame: 16 Weeks
measure: Changes from baseline in absolute reticulocyte count
timeFrame: 16 Weeks
measure: Changes from baseline in E-selectin
timeFrame: 16 Weeks
measure: Changes from baseline in Phosphatidylserine
timeFrame: 16 Weeks
measure: Changes from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home
timeFrame: 16 Weeks
measure: Changes from baseline in RBC Laminin Adhesion
timeFrame: 16 Weeks
measure: Changes from baseline in Leukocytes
timeFrame: 16 Weeks
measure: Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
timeFrame: 16 Weeks
measure: Changes from baseline in Dense Red Blood Cells
timeFrame: 16 Weeks
measure: Changes from baseline in Osmoscan
timeFrame: 16 Weeks
measure: Changes from baseline in Oxygen Point of Sickling
timeFrame: 16 Weeks
measure: Changes from baseline in D-dimer
timeFrame: 16 Weeks
measure: Change from baseline in PROMIS Pain Interference Short Form
timeFrame: 16 Weeks
measure: Change from baseline in PROMIS Physical Activity Short Form
timeFrame: 16 Weeks
measure: Trough plasma concentrations of total and unesterified 15 HEPE
timeFrame: 16 Weeks
measure: Determination of exploratory biomarkers from baseline
timeFrame: 16 Weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham (UAB)
state: Alabama
city: Farmington
country: United States
facility: New England Sickle Cell Institute, UConn Health
state: Connecticut
city: Washington
country: United States
facility: Medstar Health
state: District of Columbia
city: Atlanta
country: United States
facility: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding
state: Georgia
city: Atlanta
country: United States
facility: Emory University - Georgia Comprehensive Sickle Cell Center
state: Georgia
city: Atlanta
country: United States
facility: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital
state: Georgia
city: Chicago
country: United States
facility: UI Health Sickle Cell Center
state: Illinois
city: Baltimore
country: United States
facility: The Johns Hopkins University School of Medicine
state: Maryland
city: Bethesda
country: United States
facility: The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA
state: Maryland
city: Largo
country: United States
facility: Kaiser Permanente Mid-Atlantic States
state: Maryland
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: New Brunswick
country: United States
facility: Robert Wood Johnson Medical School Rutgers
state: New Jersey
city: Newark
country: United States
facility: Newark Beth Israel Medical Center
state: New Jersey
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Jamaica
country: United States
facility: Queens Hospital Center
state: New York
city: Chapel Hill
country: United States
facility: UNC Health
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Morrisville
country: United States
facility: Science 37
state: North Carolina
city: Calgary
country: Canada
facility: Foothills Medical Center
state: Alberta
city: Vancouver
country: Canada
facility: St Paul's Hospital Hematology/Oncology Research
state: British Columbia
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
Study officials:
References: