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Trial record 1 of 1 for:    NCT05861453
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Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT05861453
Recruitment Status : Recruiting
First Posted : May 16, 2023
Last Update Posted : March 15, 2024
Sponsor:
Information provided by (Responsible Party):
Afimmune

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

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Epeleuton Phase 2

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease
Actual Study Start Date : January 10, 2024
Estimated Primary Completion Date : August 2024
Estimated Study Completion Date : September 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Epeleuton 4g/day Drug: Epeleuton
Participants will receive 2000mg Epeleuton (DS102) capsules twice daily.
Other Name: DS102 Capsules


Outcome Measures
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Primary Outcome Measures :
  1. Changes from baseline in P-selectin [ Time Frame: 16 Weeks ]
    Change in P-selectin from baseline at Week 16.

  2. Changes from baseline in Hemoglobin [ Time Frame: 16 Weeks ]
    Change in hemoglobin from baseline at Week 16

  3. Changes from baseline in absolute reticulocyte count [ Time Frame: 16 Weeks ]
    Change in absolute reticulocyte count from baseline at Week 16.

  4. Changes from baseline in E-selectin [ Time Frame: 16 Weeks ]
    Change in E-selectin from baseline at Week 16.

  5. Changes from baseline in Phosphatidylserine [ Time Frame: 16 Weeks ]
    Change in Phosphatidlyserine from baseline to week 16.

  6. Changes from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home [ Time Frame: 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

  7. Changes from baseline in RBC Laminin Adhesion [ Time Frame: 16 Weeks ]
    Changes in RBC Laminin Adhesion from baseline to week 16

  8. Changes from baseline in Leukocytes [ Time Frame: 16 Weeks ]
    Changes in Leukocytes from baseline to Week 16

  9. Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1) [ Time Frame: 16 Weeks ]
    Changes in VCAM-1 from baseline to Week 16

  10. Changes from baseline in Dense Red Blood Cells [ Time Frame: 16 Weeks ]
    Changes in Dense Red Blood Cells from baseline to Week 16

  11. Changes from baseline in Osmoscan [ Time Frame: 16 Weeks ]
    Changes in Osmoscan from baseline to Week 16

  12. Changes from baseline in Oxygen Point of Sickling [ Time Frame: 16 Weeks ]
    Changes in Oxygen Point of Sickling from baseline to Week 16

  13. Changes from baseline in D-dimer [ Time Frame: 16 Weeks ]
    Changes in D-dimer from baseline to Week 16

  14. Change from baseline in PROMIS Pain Interference Short Form [ Time Frame: 16 Weeks ]
    Change in PROMIS Pain Interference from baseline to Week 16

  15. Change from baseline in PROMIS Physical Activity Short Form [ Time Frame: 16 Weeks ]
    Change in PROMIS Physical Activity from baseline to Week 16

  16. Trough plasma concentrations of total and unesterified 15 HEPE [ Time Frame: 16 Weeks ]
    Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16

  17. Determination of exploratory biomarkers from baseline [ Time Frame: 16 Weeks ]
    Determination of exploratory biomarkers at baseline and Week 16


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with sickle cell disease (SCD) including: 2 sickle hemoglobin genes [HbSS] and HbSβ0-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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05861453


Locations
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United States, Alabama
University of Alabama at Birmingham (UAB) Recruiting
Birmingham, Alabama, United States, 35294
Contact: Study Coordinator    205-975-6215    info@afimmune.com   
United States, California
David Geffen School of Medicine at UCLA Not yet recruiting
Los Angeles, California, United States, 90095-1678
Contact: Study Coordinator    310-794-0242    info@afimmune.com   
United States, Connecticut
New England Sickle Cell Institute, UConn Health Recruiting
Farmington, Connecticut, United States, 06030-1163
Contact: Study Coordinator    860-679-7879    info@afimmune.com   
United States, District of Columbia
MedStar Health Not yet recruiting
Washington, District of Columbia, United States, 20010
Contact: Study Coordinator       info@afimmune.com   
United States, Georgia
Emory University - Georgia Comprehensive Sickle Cell Center Not yet recruiting
Atlanta, Georgia, United States, 30303
Contact: Study Coordinator    404-712-8895    info@afimmune.com   
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta Not yet recruiting
Atlanta, Georgia, United States, 30342
Contact: Study Coordinator    404-785-6274    info@afimmune.com   
United States, Illinois
UI Health Sickle Cell Center Not yet recruiting
Chicago, Illinois, United States, 60612
Contact: Study Coordinator    312-413-0242    info@afimmune.com   
United States, Maryland
The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA Recruiting
Bethesda, Maryland, United States, 20817
Contact: Study Coordinator    301-571-2016    info@afimmune.com   
Kaiser Permanente Mid-Atlantic States Not yet recruiting
Largo, Maryland, United States, 20774
Contact: Study Coordinator    301-386-6692    info@afimmune.com   
United States, Michigan
Karmanos Cancer Institute Recruiting
Detroit, Michigan, United States, 48201
Contact: Study Coordinator    313-576-8694    info@afimmune.com   
United States, New Jersey
Robert Wood Johnson Medical School Rutgers Not yet recruiting
New Brunswick, New Jersey, United States, 08901
Contact: Study Coordinator    732-235-7678    info@afimmune.com   
Newark Beth Israel Medical Center Recruiting
Newark, New Jersey, United States, 07112
Contact: Study Coordinator    973-926-7231    info@afimmune.com   
United States, New York
Jacobi Medical Center Recruiting
Bronx, New York, United States, 10461
Contact: Study Coordinator    718-918-6039    info@afimmune.com   
Queens Hospital Center Not yet recruiting
Jamaica, New York, United States, 11432
Contact: Study Coordinator       info@afimmune.com   
United States, North Carolina
UNC Health Recruiting
Chapel Hill, North Carolina, United States, 27514
Contact: Study Coordinator    919-966-1178    info@afimmune.com   
East Carolina University Not yet recruiting
Greenville, North Carolina, United States, 27834
Contact: Study Coordinator    252-744-3617    info@afimmune.com   
Sponsors and Collaborators
Afimmune
More Information
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Responsible Party: Afimmune
ClinicalTrials.gov Identifier: NCT05861453    
Other Study ID Numbers: DS102A-10-RD2
First Posted: May 16, 2023    Key Record Dates
Last Update Posted: March 15, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn