A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People With Cystic Fibrosis
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- Arcturus Therapeutics, Inc.
- Enrollment
- 33
- Locations
- 19
- Primary Endpoint
- Incidence, severity and dose relationship of adverse events
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Confirmed diagnosis of Cystic Fibrosis
- •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)
- •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
- •History of illness or medical condition that might pose an additional risk or may confound study results
- •Recent moderate or severe hemoptysis
- •Recent major surgery
- •Solid organ or hematologic transplant
- •Requirement of supplemental oxygen while awake or > 2L per minute while sleeping.
- •Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day
- •Adequate liver and kidney function as determined by lab tests
Arms & Interventions
Cohort 1
Dose Level A of ARCT-032, inhaled daily for 28 days
Intervention: ARCT-032 (Biological)
Cohort 3
Dose Level C of ARCT-032, inhaled daily for 28 days
Intervention: ARCT-032 (Biological)
Cohort 4
Dose level B or C of ARCT-032, inhaled once daily for 12 weeks
Intervention: ARCT-032 (Biological)
Cohort 2
Dose Level B of ARCT-032, inhaled daily for 28 days
Intervention: ARCT-032 (Biological)
Outcomes
Primary Outcomes
Incidence, severity and dose relationship of adverse events
Time Frame: 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 Outcomes
- Pharmacodynamics--Lung Function(4 weeks)
- Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)(4 weeks)
- Pharmacokinetics - Plasma Concentration(Up to 6 weeks)
- High Resolution Computed Chromatography(12 weeks)
- EQ-5D-5L (Cohort 4 only)(12 weeks)