NCT06747858

Brief Summary

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
33

participants targeted

Target at P25-P50 for phase_2

Timeline
10mo left

Started Dec 2024

Geographic Reach
1 country

13 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress67%
Dec 2024Jun 2027

Study Start

First participant enrolled

December 12, 2024

Completed
5 days until next milestone

First Submitted

Initial submission to the registry

December 17, 2024

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 24, 2024

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2027

Last Updated

May 8, 2026

Status Verified

May 1, 2026

Enrollment Period

2.5 years

First QC Date

December 17, 2024

Last Update Submit

May 6, 2026

Conditions

Keywords

CFTRmRNA

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (5)

  • Pharmacokinetics - Plasma Concentration

    Up to 6 weeks

  • Pharmacodynamics--Lung Function

    4 weeks

  • Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)

    4 weeks

  • High Resolution Computed Chromatography

    12 weeks

  • EQ-5D-5L (Cohort 4 only)

    12 weeks

Study Arms (4)

Cohort 1

EXPERIMENTAL

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

Biological: ARCT-032

Cohort 2

EXPERIMENTAL

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

Biological: ARCT-032

Cohort 3

EXPERIMENTAL

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

Biological: ARCT-032

Cohort 4

EXPERIMENTAL

Dose level B or C of ARCT-032, inhaled once daily for 12 weeks

Biological: ARCT-032

Interventions

ARCT-032BIOLOGICAL

CFTR mRNA formulated in lipid nanoparticles

Cohort 1Cohort 2Cohort 3Cohort 4

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Confirmed diagnosis of Cystic Fibrosis
  • 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)
  • FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height

You may not qualify if:

  • History of illness or medical condition that might pose an additional risk or may confound study results
  • Recent moderate or severe hemoptysis
  • Recent major surgery
  • Solid organ or hematologic transplant
  • Requirement of supplemental oxygen while awake or \> 2L per minute while sleeping.
  • Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day
  • Adequate liver and kidney function as determined by lab tests

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

University of Arizona

Tucson, Arizona, 85724, United States

RECRUITING

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

NOT YET RECRUITING

Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States

RECRUITING

The Cystic Fibrosis Institute

Northfield, Illinois, 60093, United States

ACTIVE NOT RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63144, United States

RECRUITING

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

UT Health San Antonio

San Antonio, Texas, 78229, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Central Study Contacts

Clinical Trial Disclosure Manager Central Email Box

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Multiple ascending dose
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 17, 2024

First Posted

December 24, 2024

Study Start

December 12, 2024

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

June 1, 2027

Last Updated

May 8, 2026

Record last verified: 2026-05

Data Sharing

IPD Sharing
Will not share

Locations