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Evaluation of Safety, Tolerability, and Biodistribution of Inhaled RCT2100 in Cystic Fibrosis: A Phase 1/2 Multicenter Study

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Cystic Fibrosis, a genetic condition that affects the lungs and other organs. The trial will use a treatment called RCT2100, which is a new type of medication designed to be inhaled. This medication is a form of mRNA therapy, which involves using tiny particles to deliver genetic instructions to cells in the body. The goal of this study is to evaluate the safety and how well people tolerate different doses of RCT2100 when inhaled through a nebulizer, a device that turns liquid medicine into a mist.

The study will be conducted in two parts. Initially, healthy participants will receive single doses of RCT2100 to assess its safety. Following this, participants with Cystic Fibrosis will receive multiple doses to further evaluate safety and to see how the medication is distributed in the body. The trial will also explore whether the treatment can improve lung function and quality of life for those with Cystic Fibrosis. Participants will be monitored for any side effects and changes in their condition throughout the study.

The trial aims to provide valuable information about the potential of RCT2100 as a treatment for Cystic Fibrosis. By understanding how the medication works in the body and its effects on the disease, researchers hope to develop a new option for managing this challenging condition. The study is expected to continue until early 2025, with recruitment starting in mid-2024.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Study participation and medication administration
The study involves the use of a test medication called rct2100, which is taken by inhalation using a nebulizer (a machine that turns liquid medicine into a fine mist to be breathed into the lungs).
The medication rct2100 is administered in multiple-ascending doses, meaning the amount of medicine increases over time to monitor safety.
A comparator medication, kalydeco (ivacaftor) 150 mg film-coated tablets, may also be used and is taken by oral use (swallowing).
Step 2
Monitoring and evaluation
Safety and tolerability (how well the body handles the medicine) are monitored by tracking any adverse events (unexpected medical problems) or serious adverse events (more severe medical problems).
For those in the escalation cohorts (groups receiving increasing doses), lung function and respiratory health scores are measured through week 4.
For those in the expansion cohort, lung function and respiratory health scores are measured through week 12.
Blood samples may be collected to study biodistribution (how the medicine moves through the body) and to measure the concentration of the medication in the blood.

Who can join the trial?

6 criteria

Both men and women who are between 18 and 60 years old on the day they agree to join the study.
A confirmed diagnosis of Cystic Fibrosis, which is a genetic condition that affects the lungs and digestive system.
People who cannot use CFTR modulators, which are specific medicines designed to fix the faulty protein caused by the genetic mutation in Cystic Fibrosis. This applies if the person has certain types of gene mutations (changes in the DNA instructions) on both copies of the gene that do not respond to these medicines.
People who are allowed to use CFTR modulators but cannot take them because they cause bad side effects or because the medicine is not safe for them to use due to other health issues, known as contraindications.
A Forced expiratory volume in 1 second level that is between 50% and 100% of what is expected for a person of their age, sex, and height. This measurement, taken during a spirometry test (a breathing test used to check lung function), shows how much air a person can exhale quickly.
The breathing test results must meet specific standards for acceptability and repeatability, meaning the test must be performed correctly and the results must be consistent each time they are measured.

Who cannot join the trial?

9 criteria

You cannot participate if you have liver cirrhosis, which is permanent scarring of the liver, accompanied by portal hypertension, a condition where blood pressure increases in the vein that carries blood to the liver.
You are excluded if you have moderate or severe hepatic impairment, meaning your liver is not working well enough to perform its normal functions.
You cannot join if you have had a recent acute upper or lower respiratory infection (such as a sudden cold, flu, or lung infection), a pulmonary exacerbation (a sudden worsening of lung symptoms), or if you have changed your medications, including antibiotics, for lung or sinus problems within 4 weeks before the first dose.
You are not eligible if you have a lung infection caused by specific bacteria like Burkholderia cenocepacia, Burkholderia dolosa, or Mycobacterium abscessus, which are germs that can cause a person's lung health to decline very quickly.
If you have had these specific bacteria in the past, you must have had at least two negative test results for them within the last 12 months, with specific timing requirements to ensure the infection is gone.
You cannot participate if your arterial oxygen saturation (the amount of oxygen in your blood) is less than 94% while breathing normal air.
You are excluded if your estimated glomerular filtration rate, which is a measurement used to check how well your kidneys are filtering waste from your blood, is less than 30 mL/minute.
You cannot take part if you have used a CFTR modulator (a type of medication used to treat the underlying cause of certain lung conditions, such as Kalydeco or Trikafta) within the 12 weeks before joining the study.
You are ineligible if you have a history of poorly controlled diabetes, which is a condition where blood sugar levels remain consistently too high.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Centre Hospitalier Universitaire De Montpellier
Montpellier, France
Verified
Hopital Necker Enfants Malades
Paris, France
Responsive
Universitair Medisch Centrum Utrecht
Utrecht, The Netherlands

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Investigated drugs

RCT2100
is an inhaled therapy being tested to see if it is safe and how it works in people with cystic fibrosis. It is delivered through a nebulizer, which turns the liquid medicine into a mist that is breathed directly into the lungs.
Kalydeco
is a medication taken by mouth that is used as a comparison in this study to help evaluate the effectiveness of the new treatment.

What is already known about the treatment

Ivacaftor
This medication is taken orally in the form of film-coated tablets and is currently an approved treatment used to manage certain symptoms of cystic fibrosis. It belongs to a class of drugs known as CFTR potentiators, which work by helping a specific protein in the body's cells stay open longer. By doing this, the drug allows salt and water to move more easily in and out of cells, which helps thin the thick mucus often found in the lungs of patients with this condition.
RCT2100
This experimental substance is administered through inhalation using a nebulizer and is currently being studied in clinical trials to evaluate its safety and effectiveness. It is classified as an investigational inhaled therapy designed specifically for people living with cystic fibrosis. At a molecular level, it is intended to target and improve the function of proteins in the lungs to help manage the disease, though its full medical status is still being determined through ongoing research.

Investigated diseases

Cystic Fibrosis - This is a genetic disorder that affects the cells that produce mucus, sweat, and digestive juices. It causes the body to produce thick, sticky mucus that can clog various organs. This buildup primarily affects the lungs and the digestive system. Over time, the thick mucus can lead to breathing difficulties and blockages in the intestines. The condition is caused by mutations in a specific protein that regulates the movement of salt and water in and out of cells.