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Tests treatment safety and results for ROSAH
Official title Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
ClinicalTrials.gov ID: NCT06395285
What this study is testing
What is DF-003?
DF-003 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for rosah.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- 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).
- Patients must be deemed healthy except for diagnosis of ROSAH syndrome and its clinical manifestation.
You likely can't join if
- 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...
- 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...
- Strong CYP3A4 inhibitors: e.g., ceritinib, clarithromycin, cobicistat, elvitegravir/ritonavir, idelalisib, indinavir/ritonavir, itraconazole...
See the full eligibility criteria
- 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).
- Patients must be deemed healthy except for diagnosis of ROSAH syndrome and its clinical manifestation.
- Patients must be at least 18 years of age but no older than 65 years of age at the time of Screening.
- 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.
- 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...
- Strong CYP3A4 inhibitors: e.g., ceritinib, clarithromycin, cobicistat, elvitegravir/ritonavir, idelalisib, indinavir/ritonavir, itraconazole, ketoconazole, lopinavir/ritonavir, nefazodone, nelfinavir...
- 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...
- 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...
- 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.
- 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%.
- Moderate or severe hepatic impairment (categorized as Child-Pugh class B and C, respectively, on the Child-Pugh Score for Cirrhosis Mortality)
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
- Durham, North Carolina, United States
- Salt Lake City, Utah, United States
- Sydney, New South Wales, Australia
- Beijing, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States; Durham, North Carolina, United States; Salt Lake City, Utah, United States; Sydney, New South Wales, Australia; Beijing, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.