Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
Recruiting
Phase 1
Interventional Study
ROSAH
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 18 – 65
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 12 (estimated)
- Sponsor
- Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
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).…
Contacts