Evaluating ARCT-032 for treating cystic fibrosis in patients not on CFTR modulators

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

This study is testing a new treatment called ARCT-032 to see if it can help adults with cystic fibrosis who can't take standard medications feel better and improve their lung function.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Arcturus Therapeutics, Inc. (Industry-sponsored)
• Drugs / interventions: prednisone
• Locations: 13 sites (Tucson, Arizona and 12 other locations)
• Trial ID: NCT06747858 on ClinicalTrials.gov

What this trial studies

This Phase 2, open-label study investigates the safety, tolerability, and efficacy of ARCT-032 in adults with cystic fibrosis who are not eligible for or are intolerant to CFTR modulator therapy. Participants will receive nebulized ARCT-032 daily for four weeks, followed by a 12-week safety monitoring period. The study aims to assess the drug's impact on patients' respiratory function and overall health. It involves multiple ascending doses to determine the optimal dosage for efficacy and safety.

Who should consider this trial

Good fit: Ideal candidates are adults with cystic fibrosis who are not on CFTR modulators due to intolerance, poor response, or lack of access.

Not a fit: Patients with severe comorbid conditions or those requiring significant supplemental oxygen may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for cystic fibrosis patients who currently have limited or no access to effective therapies.

How similar studies have performed: While there have been studies on CFTR modulators, the approach of using ARCT-032 is novel and has not been extensively tested in this specific patient population.

Eligibility criteria

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

Where this trial is running

Tucson, Arizona and 12 other locations

• University of Arizona — Tucson, Arizona, United States (Recruiting)
• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States (Recruiting)
• Children's Hospital of Los Angeles — Los Angeles, California, United States (Recruiting)
• Children's National Hospital — Washington D.C., District of Columbia, United States (Not_yet_recruiting)
• Joe DiMaggio Children's Hospital — Hollywood, Florida, United States (Recruiting)
• The Cystic Fibrosis Institute — Northfield, Illinois, United States (Active_not_recruiting)
• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
• Washington University School of Medicine — St Louis, Missouri, United States (Recruiting)
• Cleveland Clinic Foundation — Cleveland, Ohio, United States (Recruiting)
• Medical University of South Carolina — Charleston, South Carolina, United States (Recruiting)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
• UT Health San Antonio — San Antonio, Texas, United States (Recruiting)
• Seattle Children's Hospital — Seattle, Washington, United States (Recruiting)

Study contacts

• Study coordinator: Clinical Trial Disclosure Manager Central Email Box

Conditions Cystic Fibrosis; CFTR Gene Mutation; CFTR; mRNA