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

Cancer Categories

Karmanos Trial ID

NCT ID

Age Group

Scope

Phase

Principal Investigator

Pharmacokinetic Objective:

To assess the PK of orally administered Epeleuton capsules, in the treatment of patients with SCD.

Pharmacodynamic Objective:

To assess the PD and clinical activity of orally administered Epeleuton capsules, in the treatment of patients with SCD.

Safety Objective:

To assess the safety of orally administered Epeleuton capsules in the treatment of patients with SCD.

Eligibility

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.

Locations

4100 John R
Detroit, MI 48201
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Phone: 800-527-6266

31995 Northwestern Hwy
Farmington Hills, MI 48334
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Phone: 800-527-6266

Applicable Disease Site

Therapies, Drugs, Devices