Overview
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.
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.
Eligibility
Key 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% and 100% of predicted value
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