Phase Ib Study of Oral DF-003 to Evaluate Safety and Tolerability in Adults With ROSAH Syndrome

What this Trial Is About

Researchers are evaluating the safety and tolerability of an oral drug called DF-003 in patients with ROSAH syndrome, a condition involving retinal dystrophy, optic nerve swelling, enlarged spleen, lack of sweating, and migraine headaches. This Phase Ib, open-label study will enroll up to 12 patients to investigate how the drug is processed in the body and its effects on the disease. The study aims to better understand DF-003s safety profile and biological activity in this rare condition. Participants will receive DF-003 orally once daily for 28 days. The dosing starts with higher loading doses of 140 mg on the first three days, followed by a maintenance dose of 45 mg from Day 4 through Day 28. The drug is taken in the morning with water. After completing the dosing period, patients will be followed for an additional 8 weeks to monitor ongoing effects and safety. During the study, patients will have regular assessments to monitor eye inflammation signs such as macular edema and optic nerve swelling, along with blood tests measuring inflammatory markers and drug levels. Safety will be closely monitored by recording adverse events from baseline through Day 78. The study includes detailed pharmacokinetic sampling to understand how DF-003 behaves in the body. Participation spans about 11 weeks from the start of treatment through follow-up evaluations.

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients