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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05861453
• Other study ID #: DS102A-10-RD2
• Status: Recruiting
• Phase: Phase 2
• Start date: January 10, 2024
• Est. completion date: September 2024

Study information

• Verified date: May 2024
• Source: Afimmune
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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 information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: September 2024
• Est. primary completion date: August 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients with sickle cell disease (SCD) including: 2 sickle hemoglobin genes [HbSS] and HbSß0-thalassemia
• 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 10 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 randomization visit (baseline/day 0) or have a hemoglobin A level >20% of the total hemoglobin.
• 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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Epeleuton
Participants will receive 2000mg Epeleuton (DS102) capsules twice daily.

Locations

Country	Name	City	State
United States	Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Emory University - Georgia Comprehensive Sickle Cell Center	Atlanta	Georgia
United States	The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA	Bethesda	Maryland
United States	University of Alabama at Birmingham (UAB)	Birmingham	Alabama
United States	Jacobi Medical Center	Bronx	New York
United States	UNC Health	Chapel Hill	North Carolina
United States	UI Health Sickle Cell Center	Chicago	Illinois
United States	Karmanos Cancer Institute	Detroit	Michigan
United States	New England Sickle Cell Institute, UConn Health	Farmington	Connecticut
United States	East Carolina University	Greenville	North Carolina
United States	Queens Hospital Center	Jamaica	New York
United States	Kaiser Permanente Mid-Atlantic States	Largo	Maryland
United States	Robert Wood Johnson Medical School Rutgers	New Brunswick	New Jersey
United States	Newark Beth Israel Medical Center	Newark	New Jersey
United States	MedStar Health	Washington	District of Columbia
United States	Medstar Health	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Afimmune

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes from baseline in P-selectin	Change in P-selectin from baseline at Week 16.	16 Weeks
Primary	Changes from baseline in Hemoglobin	Change in hemoglobin from baseline at Week 16	16 Weeks
Primary	Changes from baseline in absolute reticulocyte count	Change in absolute reticulocyte count from baseline at Week 16.	16 Weeks
Primary	Changes from baseline in E-selectin	Change in E-selectin from baseline at Week 16.	16 Weeks
Primary	Changes from baseline in Phosphatidylserine	Change in Phosphatidlyserine from baseline to week 16.	16 Weeks
Primary	Changes from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home	Changes in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16	16 Weeks
Primary	Changes from baseline in RBC Laminin Adhesion	Changes in RBC Laminin Adhesion from baseline to week 16	16 Weeks
Primary	Changes from baseline in Leukocytes	Changes in Leukocytes from baseline to Week 16	16 Weeks
Primary	Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)	Changes in VCAM-1 from baseline to Week 16	16 Weeks
Primary	Changes from baseline in Dense Red Blood Cells	Changes in Dense Red Blood Cells from baseline to Week 16	16 Weeks
Primary	Changes from baseline in Osmoscan	Changes in Osmoscan from baseline to Week 16	16 Weeks
Primary	Changes from baseline in Oxygen Point of Sickling	Changes in Oxygen Point of Sickling from baseline to Week 16	16 Weeks
Primary	Changes from baseline in D-dimer	Changes in D-dimer from baseline to Week 16	16 Weeks
Primary	Change from baseline in PROMIS Pain Interference Short Form	Change in PROMIS Pain Interference from baseline to Week 16	16 Weeks
Primary	Change from baseline in PROMIS Physical Activity Short Form	Change in PROMIS Physical Activity from baseline to Week 16	16 Weeks
Primary	Trough plasma concentrations of total and unesterified 15 HEPE	Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16	16 Weeks
Primary	Determination of exploratory biomarkers from baseline	Determination of exploratory biomarkers at baseline and Week 16	16 Weeks