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

Primary Purpose

Sickle Cell Disease

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Epeleuton
Sponsored by
Afimmune
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama at Birmingham (UAB)Recruiting
  • New England Sickle Cell Institute, UConn Health
  • Emory University - Georgia Comprehensive Sickle Cell Center
  • Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta
  • UI Health Sickle Cell Center
  • The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PARecruiting
  • Karmanos Cancer Institute
  • Jacobi Medical Center
  • UNC Health
  • Versiti, Inc - Wisconsin

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Epeleuton 4g/day

Arm Description

Outcomes

Primary Outcome Measures

Changes from baseline in P-selectin
Change in P-selectin from baseline at Week 16.
Changes from baseline in Hemoglobin
Change in hemoglobin from baseline at Week 16
Changes from baseline in absolute reticulocyte count
Change in absolute reticulocyte count from baseline at Week 16.
Changes from baseline in E-selectin
Change in E-selectin from baseline at Week 16.
Changes from baseline in Phosphatidylserine
Change in Phosphatidlyserine from baseline to week 16.
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
Changes from baseline in RBC Laminin Adhesion
Changes in RBC Laminin Adhesion from baseline to week 16
Changes from baseline in Leukocytes
Changes in Leukocytes from baseline to Week 16
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
Changes in VCAM-1 from baseline to Week 16
Changes from baseline in Dense Red Blood Cells
Changes in Dense Red Blood Cells from baseline to Week 16
Changes from baseline in Osmoscan
Changes in Osmoscan from baseline to Week 16
Changes from baseline in Oxygen Point of Sickling
Changes in Oxygen Point of Sickling from baseline to Week 16
Changes from baseline in D-dimer
Changes in D-dimer from baseline to Week 16
Change from baseline in PROMIS Pain Interference Short Form
Change in PROMIS Pain Interference from baseline to Week 16
Change from baseline in PROMIS Physical Activity Short Form
Change in PROMIS Physical Activity from baseline to Week 16
Trough plasma concentrations of total and unesterified 15 HEPE
Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16
Determination of exploratory biomarkers from baseline
Determination of exploratory biomarkers at baseline and Week 16

Secondary Outcome Measures

Full Information

First Posted
April 26, 2023
Last Updated
October 18, 2023
Sponsor
Afimmune
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1. Study Identification

Unique Protocol Identification Number
NCT05861453
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 2023 (Anticipated)
Primary Completion Date
August 2024 (Anticipated)
Study Completion Date
September 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Afimmune

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Epeleuton 4g/day
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Epeleuton
Other Intervention Name(s)
DS102 Capsules
Intervention Description
Participants will receive 2000mg Epeleuton (DS102) capsules twice daily.
Primary Outcome Measure Information:
Title
Changes from baseline in P-selectin
Description
Change in P-selectin from baseline at Week 16.
Time Frame
16 Weeks
Title
Changes from baseline in Hemoglobin
Description
Change in hemoglobin from baseline at Week 16
Time Frame
16 Weeks
Title
Changes from baseline in absolute reticulocyte count
Description
Change in absolute reticulocyte count from baseline at Week 16.
Time Frame
16 Weeks
Title
Changes from baseline in E-selectin
Description
Change in E-selectin from baseline at Week 16.
Time Frame
16 Weeks
Title
Changes from baseline in Phosphatidylserine
Description
Change in Phosphatidlyserine from baseline to week 16.
Time Frame
16 Weeks
Title
Changes from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home
Description
Changes in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16
Time Frame
16 Weeks
Title
Changes from baseline in RBC Laminin Adhesion
Description
Changes in RBC Laminin Adhesion from baseline to week 16
Time Frame
16 Weeks
Title
Changes from baseline in Leukocytes
Description
Changes in Leukocytes from baseline to Week 16
Time Frame
16 Weeks
Title
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
Description
Changes in VCAM-1 from baseline to Week 16
Time Frame
16 Weeks
Title
Changes from baseline in Dense Red Blood Cells
Description
Changes in Dense Red Blood Cells from baseline to Week 16
Time Frame
16 Weeks
Title
Changes from baseline in Osmoscan
Description
Changes in Osmoscan from baseline to Week 16
Time Frame
16 Weeks
Title
Changes from baseline in Oxygen Point of Sickling
Description
Changes in Oxygen Point of Sickling from baseline to Week 16
Time Frame
16 Weeks
Title
Changes from baseline in D-dimer
Description
Changes in D-dimer from baseline to Week 16
Time Frame
16 Weeks
Title
Change from baseline in PROMIS Pain Interference Short Form
Description
Change in PROMIS Pain Interference from baseline to Week 16
Time Frame
16 Weeks
Title
Change from baseline in PROMIS Physical Activity Short Form
Description
Change in PROMIS Physical Activity from baseline to Week 16
Time Frame
16 Weeks
Title
Trough plasma concentrations of total and unesterified 15 HEPE
Description
Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16
Time Frame
16 Weeks
Title
Determination of exploratory biomarkers from baseline
Description
Determination of exploratory biomarkers at baseline and Week 16
Time Frame
16 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
University of Alabama at Birmingham (UAB)
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator
Phone
205-975-6215
Email
info@afimmune.com
Facility Name
New England Sickle Cell Institute, UConn Health
City
Farmington
State/Province
Connecticut
ZIP/Postal Code
06030-1163
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
860-679-7879
Facility Name
Emory University - Georgia Comprehensive Sickle Cell Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30303
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
404-712-8895
Facility Name
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
404-785-6274
Facility Name
UI Health Sickle Cell Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
312-413-0242
Facility Name
The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Study Coordinator
Phone
301-571-2016
Email
info@afimmune.com
Facility Name
Karmanos Cancer Institute
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
313-576-8694
Facility Name
Jacobi Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
718-918-6039
Facility Name
UNC Health
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
919-966-1178
Facility Name
Versiti, Inc - Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
Phone
414-805-2220

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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