Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Pat
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.
| Condition(s) | ROSAH |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| 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. |
| Who can participate | Inclusion Criteria: 1. Sufficient understanding of the purpose and procedures required for the study. 2. Body mass index (BMI) of 18.0 to 35.0 kg/m2, inclusive. 3. Genetic testing for ALPK1 mutations that has been shown to be associated with ROSAH syndrome (e.g. T237M or Y254C, or T237A mutations). 4. Signs of uveitis (anterior and/or posterior) in the eye (e.g. macula edema, optic nerve edema, retinal vasculitis, or retinal vascular leakage). 5. Patients must be deemed healthy except for diagnosis of ROSAH syndrome and its clinical manifestation. 6. Patients must be at least 18 years of age but no older than 65 years of age at the time of Screening. Exclusion Criteria: 1. Males who plan to father a child or donate sperm while enrolled in this study or within 90 days after the last dose of |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Lead sponsor | Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) |
| Locations | Bethesda, Maryland, United States; Durham, North Carolina, United States; Salt Lake City, Utah, United States; Sydney, New South Wales, Australia; Beijing, China |
| Start date | 2025-05-27 |
| NCT ID | NCT06395285 |
| Official listing | https://clinicaltrials.gov/study/NCT06395285 |