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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT06395285
Registration number
NCT06395285
Ethics application status
Date submitted
25/04/2024
Date registered
2/05/2024
Date last updated
23/05/2024
Titles & IDs
Public title
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
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Scientific title
A Phase Ib, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Orally Administered DF-003 in ROSAH Syndrome Patients
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Secondary ID [1]
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DF-003-1002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
ROSAH
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Condition category
Condition code
Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - DF-003
Experimental: DF-003 - Oral (PO) doses of 140 mg DF-003 on Days 1, 2, and 3 followed by a maintenance dose of 45 mg DF-003 once daily (QD) starting on Day 4 through Day 28.
Treatment: Drugs: 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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Frequency and Severity of Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
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Assessment method [1]
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Timepoint [1]
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Baseline to Day 78 (±2) days
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Primary outcome [2]
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Frequency and Severity of Serious Adverse Events (SAEs) (Local and Systemic) as Assessed by CTCAE v5.0
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Assessment method [2]
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Timepoint [2]
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Baseline to Day 78 (±2) days
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Secondary outcome [1]
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Eye Uveitis as Measured by Changes in Macular Edema
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Assessment method [1]
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Timepoint [1]
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Baseline to Day 78 (±2) days
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Secondary outcome [2]
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Eye Uveitis as Measured by Changes in Optic Nerve Edema
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Assessment method [2]
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Timepoint [2]
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Baseline to Day 78 (±2) days
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Secondary outcome [3]
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Eye Uveitis as Measured by Changes in Retinal Vasculitis
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Assessment method [3]
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Timepoint [3]
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Baseline to Day 78 (±2) days
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Secondary outcome [4]
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Eye Uveitis as Measured by Changes in Retinal Vascular Leakage
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Assessment method [4]
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Timepoint [4]
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Baseline to Day 78 (±2) days
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Secondary outcome [5]
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Changes in Plasma Chemokine and Cytokine Levels
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Assessment method [5]
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Monocyte Chemoattractant Protein (MCP)-1, Chemokine (C-C motif) ligand CCL2, CCL4, Interleukin (IL)-176, IL-186, IL-1b, IL-6, IL-8, IL-17, IL-18, Tumor Necrosis Factor (TNF)-Alpha (a), Interferon-Gamma (?)6, Interferon-ß12, Serum Amyloid A (SAA)-6, SAA-12, and high sensitivity C-reactive protein (hs-CRP)
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Timepoint [5]
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Baseline to Day 78 (±2) days
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Secondary outcome [6]
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Area Under the Concentration-Time Curve (AUC) of DF-003 from time zero to the time of the last quantifiable concentration (AUC0-last)
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Assessment method [6]
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Timepoint [6]
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Day 1, 2, 8(±2), 15(±2), 22(±2), 28(±2), 29(±2)
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Secondary outcome [7]
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Area Under the Concentration-Time Curve (AUC) of DF-003 from time zero to time t (AUC0-t)
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Assessment method [7]
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Timepoint [7]
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Day 1, 2, 8(±2), 15(±2), 22(±2), 28(±2), 29(±2)
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Secondary outcome [8]
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Area Under the Concentration-Time Curve (AUC) of DF-003 from time zero to infinity (AUC0-inf)
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Assessment method [8]
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Timepoint [8]
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Day 1, 2, 8(±2), 15(±2), 22(±2), 28(±2), 29(±2)
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Secondary outcome [9]
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Peak Plasma Concentration (Cmax) of DF-003
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Assessment method [9]
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Timepoint [9]
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Day 1, 2, 8(±2), 15(±2), 22(±2), 28(±2), 29(±2)
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Secondary outcome [10]
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Time to Reach Peak Plasma Concentration (Tmax) of DF-003
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Assessment method [10]
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Timepoint [10]
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Day 1, 2, 8(±2), 15(±2), 22(±2), 28(±2), 29(±2)
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Eligibility
Key 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 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.
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Minimum age
18
Years
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Maximum age
65
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
- Strong CYP3A4 inhibitors: 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%.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Not yet recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
20/05/2024
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/11/2025
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Actual
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Sample size
Target
12
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Save Sight Institute - University of Sydney Eye Hospital - Sydney
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Recruitment hospital [2]
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The Royal Melbourne Hospital - Parkville
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Recruitment postcode(s) [1]
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2000 - Sydney
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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North Carolina
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Country [2]
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United States of America
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State/province [2]
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Utah
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to assess the safety and tolerability of DF-003 in retinal
dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH)
syndrome patients.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT06395285
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Jeysen Z Yogaratnam, MB.BCh, MRCSEd, PhD, MBA
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Address
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Country
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Phone
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+12039097551
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT06395285
Download to PDF