Actively Recruiting
A Phase Ib Open-Label Study Evaluating Safety and Pharmacokinetics of Oral DF-003 in Patients With ROSAH Syndrome
Led by Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) · Updated on 2026-06-05
12
Participants Needed
5
Research Sites
17 weeks
Total Duration
On this page
AI-Summary
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-003's 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.
CONDITIONS
Brief 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...
- Understanding of the study purpose and procedures
- Body mass index (BMI) between 18.0 and 35.0 kg/m2
- Genetic confirmation of ALPK1 mutations linked to ROSAH syndrome
- Presence of eye inflammation signs such as macular edema or optic nerve edema
- Healthy except for ROSAH syndrome diagnosis
- Age 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 with anti-inflammatory effects or risk of kidney/liver toxicity
- Use of moderate or strong CYP3A4 inhibitors or inducers
- Use of digoxin or medications known to cause Torsade de Pointes
- Use of investigational agents, vaccines, or invasive devices within specified time frames
- History of severe allergic reactions to DF-003 or related products
- Recent significant lab abnormalities including low platelets, elevated liver enzymes, bilirubin, INR, low albumin, reduced 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 28 days (4 weeks)
Participants take oral doses of DF-003 once daily for 28 days, starting with loading doses on Days 1 to 3 followed by maintenance doses from Day 4 through Day 28.
Multiple visits during dosing period for assessments
Duration - 8 weeks
Participants are monitored for safety and treatment effects for 8 weeks after the last dose of DF-003.
Periodic visits during follow-up period for assessments
Trial Site Locations
Total: 5 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
5
Peking Union Medical College Hospital
Beijing, China, 100730
Actively Recruiting
Research Team
K
Kenneth Truitt, MD
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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