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.
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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

Kenneth Truitt, MD

732-221-7104

kenneth.truitt@drug-farm.com

CONTACT