Skip to navigation menu Skip to content
Illustration of a calendarIllustration of a document pageIllustration of a heart over a handIllustration of an envelopeIllustration of the letter i inside a circleIllustration of a map markerIllustration of a caduceusIllustration of a user with a plus signIllustration of a telephoneIllustration of a question mark inside a circleIllustration of a video cameraDocument with a PDF label
Current Research Studies

A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People with Cystic Fibrosis

Arcturus Phase 2 Study of ARCT-032 in CF

  • Phase: II

What is the goal of the study?

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. ARCT-032 is a mutation-agnostic messenger RNA (mRNA) encoding full-length CFTR and encapsulated in a lipid nanoparticle for topical delivery by aerosolization to the airways. Translation of the mRNA in the cells could lead to the generation of functional CFTR to help restore lung homeostasis. Potential participants will have a screening visit to determine eligibility for dosing. If eligible, ARCT-032 will be administered by nebulization at the Day 1 visit followed by 6 additional visits over 56 days for safety and efficacy assessments. Participants will administer the study drug at home for 28 days. Study procedures will include pulmonary function testing, safety labs, ECG, and quality of life questionnaires. A baseline HRCT will be obtained with an optional follow up HRCT on Day 28. Adverse events will be recorded throughout the study period with ongoing monitoring by the data safety monitoring board. Participants will be compensated for time spent at study visits and will be reimbursed for travel, lodging, and food related to study participation.

Who can participate in the study?

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.

Study Team: