Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis (LunairCF)

A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People With Cystic Fibrosis

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.

Study Overview

• Status: Recruiting
• Conditions: Cystic Fibrosis; CFTR Gene Mutation
• Intervention / Treatment: Biological: ARCT-032

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 Type: Interventional
• Enrollment (Estimated): 33
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Clinical Trial Disclosure Manager Central Email Box; Phone Number: 858-900-2660; Email: CFclinicaltrials@arcturusrx.com

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States, 85724
      • Recruiting
      • University of Arizona
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Recruiting
      • University of Arkansas for Medical Sciences
  • California
    • Los Angeles, California, United States, 90027
      • Recruiting
      • Children's Hospital of Los Angeles
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Not yet recruiting
      • Children's National Hospital
  • Florida
    • Hollywood, Florida, United States, 33021
      • Recruiting
      • Joe DiMaggio Children's Hospital
  • Illinois
    • Northfield, Illinois, United States, 60093
      • Active, not recruiting
      • The Cystic Fibrosis Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Boston Children's Hospital
  • Missouri
    • St Louis, Missouri, United States, 63144
      • Recruiting
      • Washington University School of Medicine
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic Foundation
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt University Medical Center
  • Texas
    • San Antonio, Texas, United States, 78229
      • Recruiting
      • UT Health San Antonio
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; Dose Level A of ARCT-032, inhaled daily for 28 days	Biological: ARCT-032; CFTR mRNA formulated in lipid nanoparticles
Experimental: Cohort 2; Dose Level B of ARCT-032, inhaled daily for 28 days	Biological: ARCT-032; CFTR mRNA formulated in lipid nanoparticles
Experimental: Cohort 3; Dose Level C of ARCT-032, inhaled daily for 28 days	Biological: ARCT-032; CFTR mRNA formulated in lipid nanoparticles
Experimental: Cohort 4; Dose level B or C of ARCT-032, inhaled once daily for 12 weeks	Biological: ARCT-032; CFTR mRNA formulated in lipid nanoparticles

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Cohorts 1-3: 16 weeks and Cohort 4: 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacodynamics--Lung Function	Change from baseline in FEV1	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.	4 weeks
Pharmacokinetics - Plasma Concentration	Plasma concentration of ARCT-032 mRNA and lipid components	Up to 6 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)	12 weeks
EQ-5D-5L (Cohort 4 only)	Change from baseline to end of treatment (Day 84) in EQ-5D-5L questionnaire scores	12 weeks

Collaborators and Investigators

• Sponsor: Arcturus Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 12, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: December 17, 2024
• First Submitted That Met QC Criteria: December 20, 2024
• First Posted (Actual): December 24, 2024

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 6, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: mRNA; CFTR
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cystic Fibrosis
• Other Study ID Numbers: ARCT-032-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No