Actively Recruiting
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
Led by Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) · Updated on 2025-08-26
12
Participants Needed
4
Research Sites
48 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Understand the study purpose and procedures
- Body mass index (BMI) between 18.0 and 35.0 kg/m2
- Confirmed genetic ALPK1 mutations associated with ROSAH syndrome (e.g., T237M, Y254C, or T237A)
- Signs of uveitis in the eye (e.g., macula edema, optic nerve edema, retinal vasculitis, or retinal vascular leakage)
- Healthy except for ROSAH syndrome and its symptoms
- Aged between 18 and 65 years at screening
You will not qualify if you...
- Males planning to father a child or donate sperm during the study or within 90 days after last dose
- Females who are pregnant, breastfeeding, planning pregnancy, or donating eggs during the study or within 90 days after last dose
- Use of prohibited medications including systemic anti-inflammatories, certain CYP3A4 inhibitors/inducers, digoxin, agents causing Torsade de Pointes, investigational agents, vaccines, or invasive devices within specified timeframes
- History of severe allergic reactions to DF-003 or related products
- Recent significant lab abnormalities such as low platelet count, elevated liver enzymes, bilirubin, INR, low albumin, impaired kidney function, or high HbA1c
- Moderate or severe liver impairment (Child-Pugh class B or C)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Withdrawn
2
Duke Eye Center - Duke University Hospital
Durham, North Carolina, United States, 27705
Actively Recruiting
3
John A. Moran Eye Center - University of Utah Health
Salt Lake City, Utah, United States, 84132
Actively Recruiting
4
Save Sight Institute - University of Sydney Eye Hospital
Sydney, New South Wales, Australia, 2000
Actively Recruiting
Research Team
N
Neil Solomons, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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