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Completed

NCT Number: NCT05861453

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

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

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

St Paul's Hospital Hematology/Oncology Research, Vancouver, British Columbia, Canada

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About this study

The trial will consist of a 28-day screening period, 16 weeks of active treatment and a 30-day post-treatment follow-up period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Epeleuton

Drug

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

Other names: DS102 Capsules

Primary outcomes

  1. Change from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home

    Time frame: 16 Weeks

    Change in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16

  2. Changes from baseline in RBC Laminin Adhesion

    Time frame: 16 Weeks

    Changes in RBC Laminin Adhesion from baseline to week 16

  3. Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)

    Time frame: 16 Weeks

    Changes in VCAM-1 from baseline to Week 16

  4. Changes from baseline in P-selectin

    Time frame: 16 Weeks

    Change in P-selectin from baseline at Week 16.

  5. Changes from baseline in E-selectin

    Time frame: 16 Weeks

    Change in E-selectin from baseline at Week 16.

  6. Changes from baseline in Dense Red Blood Cells

    Time frame: 16 Weeks

    Changes in Dense Red Blood Cells from baseline to Week 16

  7. Changes from baseline in Oxygen Point of Sickling

    Time frame: 16 Weeks

    Changes in Oxygen Point of Sickling from baseline to Week 16

  8. Changes from baseline in Osmoscan

    Time frame: 16 Weeks

    Changes in Osmoscan from baseline to Week 16

  9. Changes from baseline in absolute reticulocyte count

    Time frame: 16 Weeks

    Change in absolute reticulocyte count from baseline at Week 16.

  10. Changes from baseline in D-dimer

    Time frame: 16 Weeks

    Changes in D-dimer from baseline to Week 16

  11. Changes from baseline in Phosphatidylserine

    Time frame: 16 Weeks

    Change in Phosphatidlyserine from baseline to week 16.

  12. Changes from baseline in Hemoglobin

    Time frame: 16 Weeks

    Change in hemoglobin from baseline at Week 16

  13. Changes from baseline in Leukocytes

    Time frame: 16 Weeks

    Changes in Leukocytes 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

Sponsors and collaborators

Lead sponsor

Afimmune

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 16, 2023
Registry last updated
May 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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