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

NCT ID: NCT06747858

Last Updated: 2025-10-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-12

Study Completion Date

2025-12-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Cystic Fibrosis CFTR Gene Mutation

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Multiple ascending dose
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Dose Level A of ARCT-032, inhaled daily for 28 days

Group Type EXPERIMENTAL

ARCT-032

Intervention Type BIOLOGICAL

CFTR mRNA formulated in lipid nanoparticles

Cohort 2

Dose Level B of ARCT-032, inhaled daily for 28 days

Group Type EXPERIMENTAL

ARCT-032

Intervention Type BIOLOGICAL

CFTR mRNA formulated in lipid nanoparticles

Cohort 3

Dose Level C of ARCT-032, inhaled daily for 28 days

Group Type EXPERIMENTAL

ARCT-032

Intervention Type BIOLOGICAL

CFTR mRNA formulated in lipid nanoparticles

Interventions

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ARCT-032

CFTR mRNA formulated in lipid nanoparticles

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Arcturus Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Arizona

Tucson, Arizona, United States

Site Status RECRUITING

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

Joe DiMaggio Children's Hospital

Hollywood, Florida, United States

Site Status RECRUITING

Central Florida Pulmonary Group

Orlando, Florida, United States

Site Status ACTIVE_NOT_RECRUITING

The Cystic Fibrosis Institute

Northfield, Illinois, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Washington University

St Louis, Missouri, United States

Site Status RECRUITING

Vanderbilt University

Nashville, Tennessee, United States

Site Status RECRUITING

UT Health

San Antonio, Texas, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Clinical Trial Disclosure Manager Central Email Box

Role: CONTACT

858-900-2660

Other Identifiers

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ARCT-032-02

Identifier Type: -

Identifier Source: org_study_id

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