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

RecruitingPhase 1Ages 18 Years - 65 YearsAllShanghai Yao Yuan Biotechnology Ltd. (also known โ€ฆ
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ค 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)
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What it tests

  • DF-003
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Where

5 sites
National Institutes of Health Clinical Center
Bethesda, United States
Duke Eye Center - Duke University Hospital
Durham, United States
John A. Moran Eye Center - University of Utah Health
Salt Lake City, United States
Save Sight Institute - University of Sydney Eye Hospital
Sydney, Australia
Peking Union Medical College Hospital
Beijing, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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