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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-15

Study Completion Date

2024-07-29

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT06747858

Cystic Fibrosis CFTR Gene Mutation

RECRUITING PHASE2

Study to Determine the Efficacy&Safety of ARV-1801(ACG-701) for the Treatment of Cystic Fibrosis Pulmonary Exacerbations

NCT05641298

Cystic Fibrosis Cystic Fibrosis Pulmonary Exacerbation

WITHDRAWN PHASE2

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

NCT06237335

Cystic Fibrosis

RECRUITING PHASE2

A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations

NCT03000348

Cystic Fibrosis

COMPLETED PHASE2

A Phase II , Placebo-controlled Study to Assess Efficacy of 28 Day Oral AZD9668 in Patients With Cystic Fibrosis

NCT00757848

Cystic Fibrosis

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Phase 1 of this study is a single ascending dose, first-in-human study to determine the safety, tolerability, and pharmacokinetics (PK) of ARCT-032. After screening, healthy adult participants will be randomized 3:1 to inhale a single dose of nebulized ARCT-032 or placebo. There are 4 planned sequential dose cohorts. After dosing, participants will have follow-up assessments over a 4-week period.

Phase 1b in adults with cystic fibrosis will enroll after Phase 1 is completed and safety data are reviewed. Phase 1b is an open-label, two-dose study in adults with cystic fibrosis to assess the safety, tolerability, and pharmacokinetics of ARCT-032. After completion of screening, each participants will inhale two doses of nebulized ARCT-032 two days apart. Participants will have follow-up assessments over a 4-week period.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind, placebo controlled (Phase 1 only)

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

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.

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ARCT-032, Healthy Adults

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

Group Type EXPERIMENTAL

ARCT-032

Intervention Type DRUG

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

Placebo, Healthy Adults

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Normal saline

ARCT-032,. Adults with Cystic Fibrosis

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

Group Type EXPERIMENTAL

ARCT-032

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ARCT-032

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

Intervention Type DRUG

Placebo

Normal saline

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. 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
2. Body weight between 40-100Kg and body mass index between 16-35 kg/m2
3. 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
4. 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.
5. Phase 1b only: Subjects with CF on CFTR modulator therapy must be on a stable regimen for at least 2 months prior to screening.

Exclusion Criteria

1. History of illness or condition that might pose an additional risk or may confound study results.
2. Pregnant or lactating (breast feeding)
3. History of severe allergic reaction to a liposomal product
4. Clinically significant abnormalities in Screening laboratory results
5. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
6. Treatment with another investigational drug, biological agent, or device within 30 days of screening, or 5 half-lives of investigational drug, whichever is longer
7. Drug or alcohol abuse within the past year
8. 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
9. Systemic or inhaled corticosteroids within 3 months prior to screening (Phase 1 only).
10. Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study

Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes