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.
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 Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
33
CFTR mRNA formulated in lipid nanoparticles
University of Arizona
Tucson, Arizona, United States
RECRUITINGUniversity of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Incidence, severity and dose relationship of adverse events
Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level
Time frame: Cohorts 1-3: 16 weeks and Cohort 4: 24 weeks
Pharmacokinetics - Plasma Concentration
Plasma concentration of ARCT-032 mRNA and lipid components
Time frame: Up to 6 weeks
Pharmacodynamics--Lung Function
Change from baseline in FEV1
Time frame: 4 weeks
Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)
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.
Time frame: 4 weeks
High Resolution Computed Chromatography
Changes from baseline to end of treatment in chest-HRCT-scan scores (e.g., airway wall thickness, air trapping, and mucus plugging scores)
Time frame: 12 weeks
EQ-5D-5L (Cohort 4 only)
Change from baseline to end of treatment (Day 84) in EQ-5D-5L questionnaire scores
Time frame: 12 weeks
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Children's Hospital of Los Angeles
Los Angeles, California, United States
RECRUITINGChildren's National Hospital
Washington D.C., District of Columbia, United States
NOT_YET_RECRUITINGJoe DiMaggio Children's Hospital
Hollywood, Florida, United States
RECRUITINGThe Cystic Fibrosis Institute
Northfield, Illinois, United States
ACTIVE_NOT_RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGWashington University School of Medicine
St Louis, Missouri, United States
RECRUITINGCleveland Clinic Foundation
Cleveland, Ohio, United States
RECRUITINGMedical University of South Carolina
Charleston, South Carolina, United States
RECRUITING...and 3 more locations