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Clinical Trials in China / NCT06395285
Recruiting Phase 1

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

NCT06395285 · tracked via the Priya Life Science China tracker
Sponsor
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Phase
Phase 1
Started
2025-05-27
Last updated
2026-06-05

Condition(s) studied

ROSAH

Investigational drug(s) / intervention(s)

DF-003

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.

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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). 4. Signs of uveitis (anterior and/or posterior) in the eye (e.g. macula edema, optic nerve edema, retinal vasculitis, or retinal vascular leakage). 5. Patients must be deemed healthy except for diagnosis of ROSAH syndrome and its clinical manifestation. 6. Patients must be at least 18 years of age but no older than 65 years of age at the time of Screening. Exclusion Criteria: 1. 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. 2. 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. 3. Use of any of the following prohibited medications: * Agents that are known to have systemic anti-inflammatory responses or high risk for nephrotoxicity or hepatotoxicity * Moderate CYP3A4 inhibitors: e.g., amiodarone, amprenavir, conivaptan, delavirdine, diltiazem, erythromycin, fluconazole, fosamprenavir, imatinib, miconazole, verapamil, grapefruit juice, cat's claw (Dolichandra unguis-cati), Echinacea augustifolia, wild cherry, chamomile, licorice * Strong CYP3A4 inhibitors: e.g., ceritinib, clarithromycin, cobicistat, elvitegravir/ritonavir, idelalisib, indinavir/ritonavir, itraconazole, ketoconazole, lopinavir/ritonavir, nefazodone, nelfinavir, paritaprevir/ritonavir, ombitasvir/paritaprevir/ritonavir (and/or dasabuvir), posaconazole, ritonavir, saquinavir/ritonavir, telithromycin, tipranavir/ritonavir, voriconazole. * Strong CYP3A4 inducers: apalutamide, carbamazepine, enzalutamide, ivosidenib, lumacaftor/ivacaftor, mitotane, phenytoin, rifampin, St. John's wort. * Digoxin * Agents known to cause Torsade de Pointes: Disopyramide, procainamide, quinidine, sotalol, azithromycin, clarithromycin, erythromycin, ciprofloxacin, levofloxacin, moxifloxacin, fluconazole, ketoconazole, pentamidine, voriconazole, haloperidol, thioridazine, ziprasidone, citalopram, escitalopram, dolasetron, droperidol, granisetron, and ondansetron * Investigational agents (small molecules and oligonucleotides), vaccines, or invasive medical devices within 28 days (4 weeks, or 5 half-lives, whichever is longer) prior to enrollment or having received a biological product within 6 months prior to enrollment. 4. 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. 5. Recent (within 3 months prior to screening) or acute changes in the following laboratory values: * Platelet count ≤ 120,000/mm3, or * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> ULN * Bilirubin (total, direct) \> ULN or * International Normalization Ratio (INR) \> ULN, or * Serum albumin less than the lower limit of normal, or * Estimated creatinine clearance \< 70 mL/min/1.73 m2 at Screening, calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula, or * Hemoglobin A1c (HbA1c) \> 8%. 6. Moderate or severe hepatic impairment (categorized as Child-Pugh class B and C, respectively, on the Child-Pugh Score for Cirrhosis Mortality)

Primary outcome measure(s)

Trial sites (5)

FacilityCityRegionStatus
National Institutes of Health Clinical Center Bethesda Maryland Withdrawn
Duke Eye Center - Duke University Hospital Durham North Carolina Recruiting
John A. Moran Eye Center - University of Utah Health Salt Lake City Utah Recruiting
Save Sight Institute - University of Sydney Eye Hospital Sydney New South Wales Recruiting
Peking Union Medical College Hospital Beijing China Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06395285 on ClinicalTrials.gov ↗ ← All trials in China