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.
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).
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.
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
Single group
DF-003
Drug140 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.
Frequency and Severity of Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
Frequency and Severity of Serious Adverse Events (SAEs) (Local and Systemic) as Assessed by CTCAE v5.0
Eye Uveitis as Measured by Changes in Macular Edema
Eye Uveitis as Measured by Changes in Optic Nerve Edema
Eye Uveitis as Measured by Changes in Retinal Vasculitis
Eye Uveitis as Measured by Changes in Retinal Vascular Leakage
Changes in Eye Anterior Chamber Aqueous Fluid Chemokine Levels
Eye anterior chamber fluid collection is optional and will be collected for assessment of chemokine (C-C motif) ligand CCL2, CCL3, CCL4, CCL5, C-X-C motif chemokine ligand (CXCL)-1, CXCL9, CXCL10
Changes in Eye Anterior Chamber Aqueous Fluid Cytokine Levels
Eye anterior chamber fluid collection is optional and will be collected for assessment of Interleukin (IL)-1b, IL-6, IL-8, IL-10, IL-2RA soluble, IL-18, Tumor Necrosis Factor (TNF)-Alpha, Serum Amyloid A (SAA), and high-sensitivity C-reactive protein (hs-CRP) levels
Changes in Eye Anterior Chamber Aqueous Fluid Serum Amyloid A (SAA) Levels
Eye anterior chamber fluid collection is optional and will be collected for assessment of Serum Amyloid A (SAA), and high-sensitivity C-reactive protein (hs-CRP) levels
Changes in Eye Anterior Chamber Aqueous Fluid High-Sensitivity C-reactive protein (hs-CRP) levels
Eye anterior chamber fluid collection is optional and will be collected for assessment of high-sensitivity C-reactive protein (hs-CRP) levels
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