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

Phase 2

Recruiting

• Conditions: Cystic Fibrosis; CFTR Gene Mutation
• Interventions: Biological: ARCT-032

• Registration Number: NCT06747858
• Lead Sponsor: Arcturus Therapeutics, Inc.

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.

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.

Study Timeline

• Status Verified: 2024-08
• Study Start: 2024-12-12
• Study First Submitted: 2024-12-17
• Study First Submitted QC: 2024-12-20
• Last Update Submitted: 2024-12-20
• Study First Posted: 2024-12-24
• Last Update Posted: 2024-12-24
• Primary Completion: 2025-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	ARCT-032	Dose Level A of ARCT-032, inhaled daily for 28 days
Cohort 2	ARCT-032	Dose Level B of ARCT-032, inhaled daily for 28 days
Cohort 3	ARCT-032	Dose Level C of ARCT-032, inhaled daily for 28 days

Primary Outcome Measures

Name	Time	Method
Incidence, severity and dose relationship of adverse events	16 weeks	Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics	Up to 6 weeks	Plasma concentration of ARCT-032 mRNA and lipid components
Pharmacodynamics--Lung Function	4 weeks	Change from baseline in FEV1
Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)	4 weeks	Change from baseline in CFQ-R RSS (Respiratory Symptoms Scale) Score. Overall CFQ-R score ranges from 0-100, with higher scores indicating better health.

Trial Locations

Locations (1)

The Cystic Fibrosis Institute; 🇺🇸 Northfield, Illinois, United States