Sickle Cell Disease Clinical Trial
Official title:
A Phase 1 Open-Label, Multiple-Dose Study to Evaluate Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of FTX-6058 in Subjects With Sickle Cell Disease (SCD)
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of FTX-6058 in participants with sickle cell disease.
This is a Phase 1 multicenter, open-label study evaluating the safety, tolerability, pharmacokinetics (PK), fetal hemoglobin (HbF) induction and biological activity of FTX-6058 in participants 18-65 years of age, inclusive, with SCD. Participants will receive 12 weeks of dosing with 4 weeks of follow-up. Approximately 10 participants will be enrolled in each cohort. Cohort 1 will receive 6 milligrams (mg) of FTX-6058 by mouth once daily. Doses for subsequent cohorts will be determined following review by the Data Monitoring Committee [DMC]. A total of seven cohorts may be included. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. Additional cohorts using alternative dosing schedules may be considered based on available data. The primary endpoints of the study are to evaluate the safety and tolerability of FTX-6058 as measured by the frequency of adverse events and to evaluate single and multiple-dose pharmacokinetics of FTX-6058 in participants with sickle cell disease. Secondary endpoints include evaluating the effect of FTX-6058 on fetal hemoglobin induction in peripheral blood and evaluating the effects of FTX-6058 on hemolysis in participants with sickle cell disease. ;
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