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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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
• Joe DiMaggio Children's Hospital
  Hollywood, Florida, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• Washington University School of Medicine
  St Louis, Missouri, United States Recruiting

Study contacts

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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.