Safety and effectiveness study of inhaled ARCT-032 (mRNA therapy) in adults with Cystic Fibrosis

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What is this study about?

This clinical trial focuses on Cystic Fibrosis, a genetic condition that affects the lungs and other organs. The study will test a new inhaled treatment called ARCT-032, which contains genetic material (mRNA) designed to help produce a protein that is missing or not working properly in people with Cystic Fibrosis. The purpose is to evaluate if this treatment is safe and well-tolerated by patients with this condition.

The treatment will be delivered using a special device called an eFlow Nebulizer System, which turns the liquid medication into a fine mist that can be inhaled into the lungs. This portable, battery-operated device is quiet and lightweight, making it convenient for patients to use. The study will involve multiple doses of the treatment given over time to determine the most appropriate and safe amount.

During the study, participants will receive increasing doses of ARCT-032 through inhalation. This is particularly important for patients who cannot take other available treatments for Cystic Fibrosis or have not responded well to them. The study will monitor how patients respond to the treatment, including any changes in their breathing function and overall health.

1 Initial medical assessment

Your eligibility for the study will be evaluated through medical tests and examinations

The assessment includes lung function tests to confirm your FEV1 (breathing capacity) is between 40% and 100% of predicted value

Laboratory tests will check your liver function, kidney function, and hemoglobin levels

An electrocardiogram (ECG) will be performed to check your heart rhythm

2 Treatment period preparation

You must not have any respiratory infections or lung problems for a specified period before starting treatment

If you were previously taking CFTR modulator therapy, you must have stopped it for the required period

Baseline measurements of your health status will be recorded

3 Treatment administration

You will receive ARCT-032, an inhaled medication that contains genetic material (mRNA)

The medication will be given through inhalation in liquid vapor form

Multiple doses will be administered during the study period

Your lung function will be checked before and after each dose to monitor for any breathing difficulties

4 Monitoring and assessments

Regular physical examinations and vital sign checks will be performed

Blood tests will be conducted to measure medication levels

Your lung function will be regularly tested

You will complete questionnaires about your respiratory symptoms

Any side effects or health changes will be recorded and monitored

5 Study completion

Final health assessments will be performed

Follow-up monitoring will continue for 30 days after your last dose

The study is expected to run until May 2026

Who Can Join the Study?

Must be at least 18 years old
Must have a confirmed diagnosis of Cystic Fibrosis documented in medical records with either:
– Two CF-causing mutations, or
– Sweat chloride value of 60 mmol/L or higher with history of chronic lung and sinus disease
Must not be eligible for CFTR modulator therapy (a type of treatment that helps improve the function of the defective protein in CF) or not currently taking these medications
Must have lung function test (FEV1 – the amount of air that can be forcefully exhaled in one second) between 40% and 100% of the predicted normal value
Must have stable respiratory condition with no recent chest infections or changes in treatment
Must have liver function tests less than 3 times the normal limit
Must have kidney function (eGFR) greater than 45 L/min/1.73m2
Must have hemoglobin (oxygen-carrying protein in blood) level of at least 10 g/dL
Must have normal heart rhythm on ECG (heart electrical activity test)
For women who can become pregnant: must agree to use effective birth control during the study and for 30 days after the last dose
For men with partners who can become pregnant: must agree to have their partner use effective birth control during the study and for 30 days after the last dose
Must be willing and able to:
– Sign an informed consent form
– Follow study procedures
– Perform acceptable breathing tests
– Complete all study visits

Who Cannot Join the Study?

History of organ transplantation (surgical procedure where an organ is removed from one person and placed in another)
Active lung infection or significant worsening of lung symptoms in the past 4 weeks
Current smoking or use of tobacco products
Known allergic reactions to any medications used in the study
Participation in another clinical trial within the past 30 days
Presence of any other serious medical condition that could interfere with the study
Current pregnancy or breastfeeding
Liver problems or abnormal liver function tests
History of alcohol abuse or illegal drug use
Unable to follow study procedures or attend scheduled visits
Taking medications that could interact with the study drug
History of mental health conditions that could affect participation in the study
Unstable medical conditions that require frequent medication changes

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country
Geniko Nosokomeio Thessalonikis George Papanikolaou	Thessaloniki	Greece
Ippokratio General Hospital Of Thessaloniki	Thessaloniki	Greece
Niwdnkwz Ixdijibwx Ox Tsdxcrysunel Ayj Ltak Dqrmvpig	Warsaw	Poland
Pekdfjqtsko Oxyqogd Pngfwcwiitab i Ahdbknfvxqc Sbw z oovn	Rzeszow	Poland

Trial status

Country	Status	Recruitment Start
Greece	Not recruiting	01.05.2025
Poland	Not recruiting	01.05.2025

Trial locations

ARCT-032 is an investigational therapy being studied for people with Cystic Fibrosis (CF). It is a new treatment that is being evaluated to determine if it is safe and well-tolerated by patients with CF. The medication is being tested in different doses to understand how patients respond to the treatment. This is an experimental therapy that aims to help people with Cystic Fibrosis, but its effectiveness and safety are still being studied through this clinical trial.

Investigated diseases:

Cystic fibrosis lung

Cystic Fibrosis – A genetic disorder that affects cells producing mucus, sweat, and digestive fluids. The condition causes these fluids to become thick and sticky instead of thin and slippery. This buildup of thick, sticky secretions can affect various organs, particularly the lungs and digestive system. In the lungs, the thick mucus clogs the airways and traps bacteria, leading to frequent infections and breathing problems. The condition also affects the pancreas, limiting the body’s ability to break down and absorb nutrients from food. People with cystic fibrosis often experience persistent coughing, recurring chest infections, and digestive difficulties. The disease is present from birth and is inherited in an autosomal recessive pattern.

Trial ID:

2024-517663-23-01

Protocol code:

ARCT-032-02

NCT ID:

NCT06747858

Trial Phase:

Therapeutic exploratory (Phase II)

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Safety and effectiveness study of inhaled ARCT-032 (mRNA therapy) in adults with Cystic Fibrosis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?