Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients (NCT06395285) | Clinical Trial Compass
RecruitingPhase 1
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
United States, Australia, China12 participantsStarted 2025-05-27
Plain-language 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
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Sufficient understanding of the purpose and procedures required for the study.
. Body mass index (BMI) of 18.0 to 35.0 kg/m2, inclusive.
. Genetic testing for ALPK1 mutations that has been shown to be associated with ROSAH syndrome (e.g. T237M or Y254C, or T237A mutations).
. Signs of uveitis (anterior and/or posterior) in the eye (e.g. macula edema, optic nerve edema, retinal vasculitis, or retinal vascular leakage).
. Patients must be deemed healthy except for diagnosis of ROSAH syndrome and its clinical manifestation.
. Patients must be at least 18 years of age but no older than 65 years of age at the time of Screening.
Exclusion criteria
. Males who plan to father a child or donate sperm while enrolled in this study or within 90 days after the last dose of study drug.
. Females who are pregnant, breastfeeding, planning to become pregnant, or planning to donate eggs while on study medication or within 90 days after the last dose of study drug.
. Use of any of the following prohibited medications:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1 trial focused primarily on safety and tolerability of DF-003, what does that mean for what we actually know — or don't yet know — about whether it helps people with ROSAH syndrome?
2The trial is measuring adverse events using a standardized scale called CTCAE v5.0 — can you explain what kinds of side effects have been seen with drugs like DF-003, and how serious they've been in earlier research?
3ROSAH syndrome is a very rare condition, so I'm wondering: are there any existing standard-of-care treatments I should consider trying first before exploring an early-phase experimental trial like this one?
4Given that this trial involves oral dosing, how frequent would my visits or check-ins need to be to monitor for adverse events, and is that realistic given my current situation?
5Because ROSAH syndrome affects multiple organ systems, are there specific aspects of my health right now that might make participating in a Phase 1 safety study like this more or less appropriate for me?'}
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Frequency and Severity of Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
Timeframe: Baseline to Day 78 (±2)
2
Frequency and Severity of Serious Adverse Events (SAEs) (Local and Systemic) as Assessed by CTCAE v5.0
Timeframe: Baseline to Day 78 (±2)
Trial details
NCT IDNCT06395285
SponsorShanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
. History of significant hypersensitivity to products related to DF-003 (including excipients of the formulations) as well as severe hypersensitivity reactions (like angioedema) to any drugs.
. Recent (within 3 months prior to screening) or acute changes in the following laboratory values:
. Moderate or severe hepatic impairment (categorized as Child-Pugh class B and C, respectively, on the Child-Pugh Score for Cirrhosis Mortality)