Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
Start Date
5/27/2025
Completion Date
11/1/2026
Summary
The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.
Detailed Description
This is a Phase Ib open-label, single-arm, single-dose study that will be conducted in up to 12 ROSAH syndrome patients. The study will investigate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DF-003 (study drug). DF-003 will be administered orally (PO), once daily (QD) for 28 days (4 weeks). Patients will be followed up for 8 weeks after administration of the last dose of study drug. A total of 8 patients will be evaluated in one cohort. The cohort will have a minimum of 6 patients. Additional patients (maximum of 12 patients) may be enrolled in the event of insufficient data after a review of safety data by the Study Safety Committee. Patients will receive loading doses of 140 mg DF-003 on Days 1, 2, and 3, followed by a maintenance dose of 45 mg DF-003 starting on Day 4 through Day 28. Individual dose modification is not allowed in this study.
Eligibility Criteria
Age Range: 18 years to 65 years
Interventions
DF-003
Conditions
Locations
National Institutes of Health Clinical Center
Bethesda, Maryland 20892
United States
Duke Eye Center - Duke University Hospital
Durham, North Carolina 27705
United States
John A. Moran Eye Center - University of Utah Health
Salt Lake City, Utah 84132
United States
Save Sight Institute - University of Sydney Eye Hospital
Sydney, New South Wales 2000
Australia
Peking Union Medical College Hospital
Beijing, 100730
China