Sickle Cell DiseasePhase 2NCT05861453
Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease
A Phase 2 study testing Epeleuton in people with Sickle Cell Disease, run by Afimmune at 18 sites in 2 countries.
Trial summary
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Epeleuton
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 18 sitesin 2 countries
- Study dates
- 10 Jan 2024to 11 Feb 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
18 sites in 2 countries.
18 sites in 2 countries
United States16 sites
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Hughes Spalding
Atlanta, Georgia, 30303
Emory University - Georgia Comprehensive Sickle Cell Center
Atlanta, Georgia, 30303
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta at Arthur M. Blank Hospital
Atlanta, Georgia, 30329
The Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
The Center for Cancer and Blood Disorders, A Division of American Oncology Partners, PA
Bethesda, Maryland, 20817
University of Alabama at Birmingham (UAB)
Birmingham, Alabama, 35294
UNC Health
Chapel Hill, North Carolina, 27517
UI Health Sickle Cell Center
Chicago, Illinois, 60612
Karmanos Cancer Institute
Detroit, Michigan, 48201
New England Sickle Cell Institute, UConn Health
Farmington, Connecticut, 06030-1163
Kaiser Permanente Mid-Atlantic States
Largo, Maryland, 20774
Science 37
Morrisville, North Carolina, 27560
Robert Wood Johnson Medical School Rutgers
New Brunswick, New Jersey, 08901
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Jacobi Medical Center
The Bronx, New York, 10461
Medstar Health
Washington D.C., District of Columbia, 20010
Canada2 sites
Toronto General Hospital
Toronto, Ontario, M5G 2C4
St Paul's Hospital Hematology/Oncology Research
Vancouver, British Columbia, V6E 1M7
About this study
Official title: An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered 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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Epeleuton
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EpeleutonDrug
Also called DS102 Capsules
Participants will receive 2000mg Epeleuton (DS102) capsules twice daily.
| Study group | Role | What they receive |
|---|---|---|
| Epeleuton 4g/day | Experimental | Not described Receives: Drug: Epeleuton |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home
Change in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home from baseline to week 16
Measured: 16 Weeks
Changes from baseline in RBC Laminin Adhesion
Changes in RBC Laminin Adhesion from baseline to week 16
Measured: 16 Weeks
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
Changes in VCAM-1 from baseline to Week 16
Measured: 16 Weeks
Changes from baseline in P-selectin
Change in P-selectin from baseline at Week 16.
Measured: 16 Weeks
Changes from baseline in E-selectin
Change in E-selectin from baseline at Week 16.
Measured: 16 Weeks
Changes from baseline in Dense Red Blood Cells
Changes in Dense Red Blood Cells from baseline to Week 16
Measured: 16 Weeks
Changes from baseline in Oxygen Point of Sickling
Changes in Oxygen Point of Sickling from baseline to Week 16
Measured: 16 Weeks
Changes from baseline in Osmoscan
Changes in Osmoscan from baseline to Week 16
Measured: 16 Weeks
Changes from baseline in absolute reticulocyte count
Change in absolute reticulocyte count from baseline at Week 16.
Measured: 16 Weeks
Changes from baseline in D-dimer
Changes in D-dimer from baseline to Week 16
Measured: 16 Weeks
Changes from baseline in Phosphatidylserine
Change in Phosphatidlyserine from baseline to week 16.
Measured: 16 Weeks
Changes from baseline in Hemoglobin
Change in hemoglobin from baseline at Week 16
Measured: 16 Weeks
Changes from baseline in Leukocytes
Changes in Leukocytes from baseline to Week 16
Measured: 16 Weeks
Change from baseline in PROMIS Pain Interference Short Form
Change in PROMIS Pain Interference from baseline to Week 16
Measured: 16 Weeks
Change from baseline in PROMIS Physical Activity Short Form
Change in PROMIS Physical Activity from baseline to Week 16
Measured: 16 Weeks
Trough plasma concentrations of total and unesterified 15 HEPE
Trough plasma concentrations of total and unesterified 15 HEPE at baseline and Week 16
Measured: 16 Weeks
Determination of exploratory biomarkers from baseline
Determination of exploratory biomarkers at baseline and Week 16
Measured: 16 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2023
- Study start10 Jan 2024
- Primary completion21 Jan 2026
- Study completion11 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Afimmune
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05861453
- Sponsor's protocol ID
- DS102A-10-RD2
- Status last verified
- Apr 2026
- First submitted
- 26 Apr 2023
- Last updated
- 4 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT05861453
- Acronym / sponsor ID
- DS102A-10-RD2
- Conditions
- Sickle Cell Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- AfimmuneIndustry (pharma, biotech, devices)
- Study start
- 10 Jan 2024 (actual)
- Primary completion
- 21 Jan 2026 (actual)
- Study completion
- 11 Feb 2026 (actual)
- First posted
- 16 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Epeleuton 4g/day | Experimental | Drug: Epeleuton |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients with sickle cell disease (SCD) including:
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EpeleutonAlso: DS102 CapsulesParticipants will receive 2000mg Epeleuton (DS102) capsules twice daily. | Drug | Epeleuton 4g/day |
Primary outcome
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
Changes from baseline in RBC Laminin Adhesion
Time frame: 16 Weeks
Changes in RBC Laminin Adhesion from baseline to week 16
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)
Time frame: 16 Weeks
Changes in VCAM-1 from baseline to Week 16
Changes from baseline in P-selectin
Time frame: 16 Weeks
Change in P-selectin from baseline at Week 16.
Changes from baseline in E-selectin
Time frame: 16 Weeks
Change in E-selectin from baseline at Week 16.
Changes from baseline in Dense Red Blood Cells
Time frame: 16 Weeks
Changes in Dense Red Blood Cells from baseline to Week 16
Changes from baseline in Oxygen Point of Sickling
Time frame: 16 Weeks
Changes in Oxygen Point of Sickling from baseline to Week 16
Changes from baseline in Osmoscan
Time frame: 16 Weeks
Changes in Osmoscan from baseline to Week 16
Changes from baseline in absolute reticulocyte count
Time frame: 16 Weeks
Change in absolute reticulocyte count from baseline at Week 16.
Changes from baseline in D-dimer
Time frame: 16 Weeks
Changes in D-dimer from baseline to Week 16
Changes from baseline in Phosphatidylserine
Time frame: 16 Weeks
Change in Phosphatidlyserine from baseline to week 16.
Changes from baseline in Hemoglobin
Time frame: 16 Weeks
Change in hemoglobin from baseline at Week 16
Changes from baseline in Leukocytes
Time frame: 16 Weeks
Changes in Leukocytes from baseline to Week 16
Change from baseline in PROMIS Pain Interference Short Form
Time frame: 16 Weeks
Change in PROMIS Pain Interference from baseline to Week 16
Change from baseline in PROMIS Physical Activity Short Form
Time frame: 16 Weeks
Change in PROMIS Physical Activity from baseline to Week 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
Determination of exploratory biomarkers from baseline
Time frame: 16 Weeks
Determination of exploratory biomarkers at baseline and Week 16
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Epeleuton in people with Sickle Cell Disease, run by Afimmune at 18 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home (time frame: 16 Weeks). The study lists 17 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 May 2026.