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RECRUITING
NCT06395285
PHASE1

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients

Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)

View on ClinicalTrials.gov

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.

Official title: A Phase Ib, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Orally Administered DF-003 in ROSAH Syndrome Patients

Key Details

Gender

All

Age Range

18 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

12

Start Date

2025-05-27

Completion Date

2026-05

Last Updated

2025-08-26

Healthy Volunteers

No

Conditions

Interventions

DRUG

DF-003

140 mg on Days 1, 2, and 3 followed by a maintenance dose of 45 mg QD starting on Day 4 through Day 28. DF-003 will be administered PO with approximately 240 mL of water in the morning once daily for 28 consecutive days.

Locations (4)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Duke Eye Center - Duke University Hospital

Durham, North Carolina, United States

John A. Moran Eye Center - University of Utah Health

Salt Lake City, Utah, United States

Save Sight Institute - University of Sydney Eye Hospital

Sydney, New South Wales, Australia