Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults With Cystic Fibrosis.

NCT05712538 | Completed Phase 1 DMC

• 1 other identifier: ARCT-032-01
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-032 in healthy adult subjects (Phase 1) and of two doses in Adults with Cystic Fibrosis (Phase 1b).

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis, CF

Outcome Measures

Primary Outcomes (1)

• Incidence, severity and dose-relationship of AEs

  Safety and tolerability of ARCT-032 assessed by determining the incidence, severity and dose-relationship of AEs by dose

  4 weeks

Secondary Outcomes (7)

• Change in plasma area under the curve after single dose of ARCT-032

  Up to 2 Weeks

• Maximum observed plasma concentration (Cmax) after single dose of ARCT-032

  Up to 2 Weeks

• Time at which Cmax occurred after single dose of ARCT-032

  Up to 2 Weeks

• AUC0-inf after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032

  Up to 2 Weeks

• T1/2 after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032

  Up to 2 Weeks

Study Arms (3)

ARCT-032, Healthy Adults

EXPERIMENTAL

Ascending single doses of ARCT-032 administered to healthy adults via nebulizer

Drug: ARCT-032

Placebo, Healthy Adults

PLACEBO COMPARATOR

Single doses of 0.9% Saline administered to healthy adults via nebulizer

Other: Placebo

ARCT-032,. Adults with Cystic Fibrosis

EXPERIMENTAL

Two doses of ARCT-032 administered to adults with Cystic Fibrosis via nebulizer

Drug: ARCT-032

Interventions

ARCT-032 DRUG

ARCT-032 is messenger RNA (mRNA) coding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, formulated in a lipid nanoparticle (LNP).

ARCT-032, Healthy Adults; ARCT-032,. Adults with Cystic Fibrosis

Placebo OTHER

Normal saline

Placebo, Healthy Adults

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Phase 1: Healthy males or females aged 18 to 65 years at the time of informed consent.
• Phase 1b: Males or females aged 18 to 65 years with confirmed diagnosis of CF documented in subject's medical record
• Body weight between 40-100Kg and body mass index between 16-35 kg/m2
• Phase 1: Forced expiratory volume (FEV1) at screening \>85% of predicted value for age, sex, and height.
• Phase 1b: FEV1 at screening between 50% and 100% of predicted value
• Surgically sterile or using an acceptable contraceptive method from the time of signing the informed consent form until at least 30 days after the last dose of study drug.
• Phase 1b only: Subjects with CF on CFTR modulator therapy must be on a stable regimen for at least 2 months prior to screening.

You may not qualify if:

• History of illness or condition that might pose an additional risk or may confound study results.
• Pregnant or lactating (breast feeding)
• History of severe allergic reaction to a liposomal product
• Clinically significant abnormalities in Screening laboratory results
• Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
• Treatment with another investigational drug, biological agent, or device within 30 days of screening, or 5 half-lives of investigational drug, whichever is longer
• Drug or alcohol abuse within the past year
• History of moderate to heavy smoking or vaping (\>10 cigarettes/sessions per day) within 6 months prior to the dose of study drug. Participants must be willing to refrain from smoking or vaping within 1 week of dosing through Day 15
• Systemic or inhaled corticosteroids within 3 months prior to screening (Phase 1 only).

Sponsors & Collaborators

• Arcturus Therapeutics, Inc.: lead
• Novotech CRO: collaborator

Study Sites (1)

New Zealand Clinical Research

Christchurch, 8011, New Zealand

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Clinical Program Director

  Arcturus Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Participant and investigator masking only applies to Phase 1 healthy volunteers only. For Phase 1b, there is no masking, None (Open Label) should be applied.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double-blind, placebo controlled (Phase 1 only)

Study Record Dates

• First Submitted: January 26, 2023
• First Posted: February 3, 2023
• Study Start: February 15, 2023
• Primary Completion: July 29, 2024
• Study Completion: July 29, 2024
• Last Updated: November 13, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (1)