CFTR Gene Mutation, Cystic Fibrosis
Conditions
Keywords
CFTR, mRNA
Brief summary
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.
Detailed description
This is an open-label, multiple-ascending dose study of ARCT-032 in adults with CF who are not on CFTR modulator therapy. After successful screening, eligible participants enrolled in cohorts 1-3 will receive nebulized ARCT-032 daily for 4 weeks and then will be followed for safety for a total of 12 weeks. In cohort 4, eligible participants will receive nebulized ARCT-032 for 12 weeks and will then be followed for safety for an additional 12 weeks.
Interventions
BIOLOGICALARCT-032
CFTR mRNA formulated in lipid nanoparticles
Sponsors
Arcturus Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Multiple ascending dose
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Confirmed diagnosis of Cystic Fibrosis 2. Not eligible for CFTR modulator therapy or not taking CFTR modulators for at least 60 days prior to dosing (e.g. due to intolerance, poor response, or lack of access to modulators) 3. FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height
Exclusion criteria
1. History of illness or medical condition that might pose an additional risk or may confound study results 2. Recent moderate or severe hemoptysis 3. Recent major surgery 4. Solid organ or hematologic transplant 5. Requirement of supplemental oxygen while awake or \> 2L per minute while sleeping. 6. Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day 7. Adequate liver and kidney function as determined by lab tests
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence, severity and dose relationship of adverse events | Cohorts 1-3: 16 weeks and Cohort 4: 24 weeks | Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics - Plasma Concentration | Up to 6 weeks | Plasma concentration of ARCT-032 mRNA and lipid components |
| Pharmacodynamics--Lung Function | 4 weeks | Change from baseline in FEV1 |
| Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R) | 4 weeks | Change from baseline in CFQ-R RSS (Respiratory Symptoms Scale) Score. Overall CFQ-R score ranges from 0-100, with higher scores indicating better health. |
| High Resolution Computed Chromatography | 12 weeks | Changes from baseline to end of treatment in chest-HRCT-scan scores (e.g., airway wall thickness, air trapping, and mucus plugging scores) |
| EQ-5D-5L (Cohort 4 only) | 12 weeks | Change from baseline to end of treatment (Day 84) in EQ-5D-5L questionnaire scores |
Countries
United States
Contacts
CONTACTClinical Trial Disclosure Manager Central Email Box
Outcome results
None listed