Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease (NCT05861453) | Clinical Trial Compass
CompletedPhase 2
Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease
United States, Canada35 participantsStarted 2024-01-10
Plain-language summary
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change from baseline in annualized rate of VOCs leading to a healthcare visit, and VOCs that are treated at home
Timeframe: 16 Weeks
2
Changes from baseline in RBC Laminin Adhesion
Timeframe: 16 Weeks
3
Changes from baseline in Vascular Cell Adhesion Molecule 1 (VCAM-1)