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

ConditionsCystic FibrosisCFTR Gene Mutation
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorArcturus Therapeutics, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • ARCT-032

    Biological/Vaccine

    CFTR mRNA formulated in lipid nanoparticles

Treatment groups

33 Participants
are divided into 4 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 3Experimental treatment 1 intervention
Group D: Cohort 4Experimental treatment 1 intervention

Trial outcomes

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

Time frame
Cohorts 1-3: 16 weeks and Cohort 4: 24 weeks

Secondary outcomes

1

Pharmacokinetics - Plasma Concentration

Plasma concentration of ARCT-032 mRNA and lipid components

Time frame
Up to 6 weeks
2

Pharmacodynamics--Lung Function

Change from baseline in FEV1

Time frame
4 weeks
3

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

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.

Time frame
4 weeks
4

High Resolution Computed Chromatography

Changes from baseline to end of treatment in chest-HRCT-scan scores (e.g., airway wall thickness, air trapping, and mucus plugging scores)

Time frame
12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Arcturus Therapeutics, Inc.

Lead sponsor

Source ClinicalTrials.gov
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