Ongoing Clinical Trials for Cystic Fibrosis Lung
This article presents information about 3 ongoing clinical trials investigating new treatments for cystic fibrosis lung disease. These trials are testing gene therapy and mRNA-based treatments designed to help people whose lungs are affected by this genetic condition. The studies are being conducted across several European countries including France, Greece, Italy, Netherlands, Poland, and Spain.
Clinical trial locations
- France
- Greece
- Italy
- Netherlands
- Poland
- Spain
Long-Term Safety Study of BI 3720931 for Patients with Cystic Fibrosis from a Previous Trial
This trial focuses on monitoring the long-term health and safety of people who previously participated in a study testing BI 3720931, an inhaled gene therapy treatment. The study will follow participants for up to 15 years to observe any potential long-term effects after receiving this treatment.
Main inclusion criteria:
- Must have participated in a previous study with BI 3720931 and received at least one dose of the medication or placebo
- Must provide signed and dated written consent according to international and local guidelines
- Open to both males and females of all ages, including children and adults
Main exclusion criteria:
- Any other serious health condition that could affect safety during the trial
- Recent infection that could interfere with the trial
- Currently taking medications that might interact with the trial treatment
- History of allergic reactions to similar treatments
- Pregnancy or breastfeeding
- Recent participation in another clinical trial
- Inability to follow trial procedures or instructions
Focus and goals: The study aims to evaluate the long-term safety profile of BI 3720931 by monitoring for any new health issues such as serious infections, neurological disorders, or autoimmune conditions. Researchers will also track lung function over time, measuring forced expiratory volume in 1 second (FEV1), which indicates how well the lungs are working. The study will record any serious adverse events and assess the time it takes for any loss of treatment effectiveness.
Investigational drug: BI 3720931 is an inhaled gene therapy using a lentiviral vector. It is designed to deliver genetic material directly to the lungs, potentially correcting the underlying genetic defect associated with cystic fibrosis. The treatment is administered as a nebuliser solution through inhalation.
Safety and effectiveness study of inhaled ARCT-032 (mRNA therapy) in adults with Cystic Fibrosis
This trial tests 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 treatment is delivered using a special device called an eFlow Nebulizer System.
Main inclusion criteria:
- Must be at least 18 years old
- Must have a confirmed diagnosis of cystic fibrosis with either two CF-causing mutations or a sweat chloride value of 60 mmol/L or higher
- Must not be eligible for CFTR modulator therapy or not currently taking these medications
- Must have lung function (FEV1) between 40% and 100% of predicted normal value
- Must have stable respiratory condition with no recent chest infections
- 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 level of at least 10 g/dL
- Women of childbearing potential and men with partners who can become pregnant must agree to use effective birth control during the study and for 30 days after the last dose
Main exclusion criteria:
- History of organ transplantation
- 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
- History of mental health conditions that could affect participation
- Unstable medical conditions requiring frequent medication changes
Focus and goals: The study aims to evaluate if ARCT-032 is safe and well-tolerated by patients with cystic fibrosis. Participants will receive increasing doses of the treatment through inhalation to determine the most appropriate and safe amount. The study will monitor how patients respond to the treatment, including any changes in breathing function and overall health. Regular assessments will include physical examinations, vital sign checks, blood tests, lung function tests, and questionnaires about respiratory symptoms.
Investigational drug: ARCT-032 is an inhaled mRNA therapy administered as a liquid vapor through the eFlow Nebulizer System. The treatment contains genetic material designed to help produce a missing or malfunctioning protein in people with cystic fibrosis. This is an experimental therapy specifically for patients who cannot take other available treatments or have not responded well to them.
Study on the Safety and Effectiveness of BI 3720931 for Adults with Cystic Fibrosis Who Cannot Use CFTR Modulators
This trial studies BI 3720931, an inhaled gene therapy, in adults with cystic fibrosis who cannot use other treatments known as CFTR modulators. The study includes an open-label phase to determine the best dose, followed by a randomized, double-blind phase where participants receive either the treatment or a placebo.
Main inclusion criteria:
- Must be a male or female who cannot have children and has cystic fibrosis affecting the lungs with a confirmed diagnosis
- Must have a positive sweat test result showing 60 or more units, or have two known genetic mutations causing cystic fibrosis with certain symptoms if the sweat test result is between 30 and 59 units
- Must not be eligible for specific cystic fibrosis treatment due to genetic makeup and not expected to become eligible during the study
- Must be able to perform breathing tests properly according to specific health organization standards
- Must have a lung function test result (FEV1pp) between 50% and 100% of what is considered normal
- Must have stable cystic fibrosis with no worsening of lung symptoms for at least 4 weeks before the study starts
- Can either have never received gene therapy or have received gene therapy for cystic fibrosis before, with specific time gaps since last treatment (6 months for non-viral or 24 months for viral gene therapy)
Main exclusion criteria:
- History of severe allergic reactions to any of the ingredients in the study medication
- Currently participating in another clinical trial or have participated in one within the last 30 days
- History of lung transplant
- Significant medical condition that could interfere with study results
- Pregnancy or breastfeeding
- History of drug or alcohol abuse within the past year
- Unable to comply with study procedures or follow-up visits
Focus and goals: The study evaluates the safety, tolerability, and effectiveness of a single dose of BI 3720931 in adults who cannot use CFTR modulators. Participants will be monitored for changes in lung function, specifically FEV1 measurements at various intervals including weeks 4, 6, 8, and 24. The primary outcome is the change in lung function from the start of the study to week 8. The study also tracks any side effects that occur after receiving the treatment, with monitoring up to 24 weeks after dosing.
Investigational drug: BI 3720931 is an inhaled gene therapy using a lentiviral vector delivery system. The therapy is administered as a nebulizer solution, delivering genetic material directly to the lungs. The goal is to improve lung function by correcting the underlying genetic defect at the molecular level. The trial assesses how safe and tolerable this therapy is, as well as its potential effectiveness in improving breathing in patients.
Summary
Currently, there are 3 ongoing clinical trials for cystic fibrosis lung disease across Europe. These studies are exploring innovative treatment approaches, including gene therapy and mRNA-based therapies, aimed at addressing the underlying genetic causes of the condition rather than just managing symptoms.
A notable observation is that two of the three trials are testing BI 3720931, an inhaled gene therapy using a lentiviral vector. One of these trials focuses on long-term safety monitoring over up to 15 years for patients who previously received the treatment, while the other evaluates its safety and effectiveness in adults who cannot use CFTR modulators. This concentration of research on a single therapy suggests it may be a promising approach for patients who have limited treatment options.
The third trial investigates ARCT-032, an mRNA-based therapy that represents a different technological approach to treating the condition. This trial is specifically designed for adults who cannot take or have not responded well to existing treatments.
Geographically, the trials show concentration in Western European countries. France, Italy, Netherlands, and Spain each host two trials testing BI 3720931, while Greece and Poland host the ARCT-032 mRNA therapy trial. This distribution reflects the collaborative nature of European clinical research and provides multiple access points for potentially eligible patients across the continent.
All three trials focus on patients who have limited treatment options, particularly those who cannot use CFTR modulators. This represents an important research direction for addressing unmet medical needs in the cystic fibrosis patient community. The studies are expected to continue through 2026 and 2027, with the long-term safety study extending potentially until the late 2030s.
