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Purpose

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.

Conditions

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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% and 100% of predicted value

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 Design

Phase
Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Intervention Model Description
Multiple ascending dose
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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

Recruiting Locations

University of Arkansas for Medical Sciences
Little Rock 4119403, Arkansas 4099753 72205

Children's Hospital of Los Angeles
Los Angeles 5368361, California 5332921 90027

Central Florida Pulmonary Group
Orlando 4167147, Florida 4155751 32803

The Cystic Fibrosis Institute
Northfield 4904094, Illinois 4896861 60093

Boston Children's Hospital
Boston 4930956, Massachusetts 6254926 02115

Washington University
St Louis 4407066, Missouri 4398678 63144

Vanderbilt University
Nashville 4644585, Tennessee 4662168 37232

UT Health
San Antonio 4726206, Texas 4736286 78229

Seattle Children's Hospital
Seattle 5809844, Washington 5815135 98105

More Details

NCT ID
NCT06747858
Status
Recruiting
Sponsor
Arcturus Therapeutics, Inc.

Study Contact

Clinical Trial Disclosure Manager Central Email Box
858-900-2660
CFclinicaltrials@arcturusrx.com

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 will receive nebulized ARCT-032 daily for 4 weeks, and then followed for safety for a total of 12 weeks.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.