ROSAH Clinical Trial
Official title:
A Phase Ib, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Orally Administered DF-003 in ROSAH Syndrome Patients
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DF-003 for treatment of ROSAH syndrome.
After being informed about the study and potential risks, all patients giving written informed consent will undergo a 49 day screening period to determine eligibility for study entry. At Week 0, patients who meet eligibility requirements will be enrolled into Cohort 1 to receive DF-003 (study drug). DF-003 will be administered orally once daily (QD) for 28 days (4 weeks). The doses to be evaluated are loading doses of 140 mg on Days 1, 2, and 3 followed by a maintenance dose of 45 mg QD starting on Day 4 through Day 28. Doses in subsequent cohorts in this study will be determined based on review of all the available safety and PK data from Cohort 1. Subsequent cohorts will only be dosed if it is considered safe to do so by the study safety committee. ;