Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
Purpose
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.
Conditions
- Cystic Fibrosis
- CFTR Gene Mutation
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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% and 100% of predicted value
Exclusion Criteria
- 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
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Intervention Model Description
- Multiple ascending dose
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Cohort 1 |
Dose Level A of ARCT-032, inhaled daily for 28 days |
|
|
Experimental Cohort 2 |
Dose Level B of ARCT-032, inhaled daily for 28 days |
|
|
Experimental Cohort 3 |
Dose Level C of ARCT-032, inhaled daily for 28 days |
|
Recruiting Locations
Vanderbilt University
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Arcturus Therapeutics, Inc.
Study Contact
Clinical Trial Disclosure Manager Central Email Box858-900-2660
CFclinicaltrials@arcturusrx.com
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 will receive nebulized ARCT-032 daily for 4 weeks, and then followed for safety for a total of 12 weeks.