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

Sponsor
Afimmune (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05861453
Collaborator
(none)
30
Enrollment
1
Arm
12
Anticipated Duration (Months)

Study Details

Study Description

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
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
30 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open-label Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease
Anticipated Study Start Date :
Sep 1, 2023
Anticipated Primary Completion Date :
Aug 1, 2024
Anticipated Study Completion Date :
Sep 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Epeleuton 4g/day

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Changes from baseline in P-selectin [16 Weeks]

      Change in P-selectin from baseline at Week 16.

    2. Changes from baseline in Hemoglobin [16 Weeks]

      Change in hemoglobin from baseline at Week 16

    3. Changes from baseline in absolute reticulocyte count [16 Weeks]

      Change in absolute reticulocyte count from baseline at Week 16.

    4. Changes from baseline in E-selectin [16 Weeks]

      Change in E-selectin from baseline at Week 16.

    5. Changes from baseline in Phosphatidylserine [16 Weeks]

      Change in Phosphatidlyserine from baseline to week 16.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Patients with homozygous sickle cell disease (SCD) including: 2 sickle hemoglobin genes [HbSS]

    • Male or female patients aged 18 years and older on the day of signing the informed consent form (ICF)

    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.

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Afimmune

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Afimmune
    ClinicalTrials.gov Identifier:
    NCT05861453
    Other Study ID Numbers:
    • DS102A-10-RD2
    First Posted:
    May 16, 2023
    Last Update Posted:
    May 16, 2023
    Last Verified:
    May 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of May 16, 2023