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Ongoing Clinical Trials for Cystic Fibrosis

There are currently 18 clinical trials underway studying various treatments for cystic fibrosis, a genetic condition affecting the lungs and digestive system. These trials are testing different medications including CFTR modulators, anti-inflammatory drugs, antibiotics, and gene therapy approaches across multiple countries in Europe.

Clinical trial locations

Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis

This study in Germany is testing whether anakinra, an anti-inflammatory medication, can help improve lung function in adults with cystic fibrosis. The medication is given as a daily injection under the skin and works by blocking a protein called interleukin-1 that causes inflammation in the body.

Who can participate: Adults aged 18 and older with confirmed cystic fibrosis are eligible if they have stable lung function with FEV1 (a measure of how much air can be exhaled in one second) of at least 50% of normal. Participants must have adequate blood counts, liver function, and kidney function, and must not be pregnant or breastfeeding. They should be fluent in German to complete study questionnaires.

Who cannot participate: People with active lung infections or worsening respiratory symptoms in the past four weeks cannot join. Those with known allergies to anakinra, pregnant or breastfeeding women, people with severe liver or kidney problems, active cancer, recent serious infections, active tuberculosis, or inability to perform required breathing tests are excluded. People currently taking immunosuppressive medications or with a history of drug or alcohol abuse in the past year also cannot participate.

Study focus: The trial uses a crossover design where participants receive both anakinra (brand name Kineret, 100 mg daily) and placebo for 28-day periods with a break in between. Researchers will monitor lung function using the lung clearance index test, conduct physical examinations and blood tests, and collect sputum samples to analyze inflammation markers. Optional procedures include chest MRI scans to examine lung structure.

Investigational drug: Anakinra is a medication that blocks interleukin-1, a protein involved in inflammation. By reducing inflammation in the airways, it may help improve breathing and lung function in people with cystic fibrosis.

Study of VX-121, Tezacaftor, and Deutivacaftor for Children Aged 1-11 with Cystic Fibrosis

This study in Sweden, the Netherlands, France, and Germany is evaluating a triple combination therapy of VX-121, tezacaftor, and deutivacaftor in children aged 1 to 11 years. These medications work together to improve the function of the CFTR protein, which is defective in people with cystic fibrosis.

Who can participate: Children between 2 and 11 years old with a confirmed diagnosis of cystic fibrosis and at least one specific genetic mutation in the CFTR gene can participate. Their weight must be within certain study limits, and they must have stable symptoms at the start. For some age groups, children must be able to swallow tablets. Parents or legal representatives must provide informed consent, and for female participants who can have children, pregnancy tests must be negative.

Who cannot participate: Children with medical conditions other than cystic fibrosis that might interfere with the study cannot participate. Those outside the specified age range, unable to follow study procedures or take medications as required, who are pregnant or breastfeeding, have participated in another recent clinical trial, or have a history of allergic reactions to study medications are excluded.

Study focus: The study is divided into two parts. Part A focuses on understanding how the body processes these medications, while Part B evaluates safety and tolerability over 24 weeks. Participants will receive the medications as film-coated tablets or in another appropriate form and will undergo regular health assessments including tests of vital signs, laboratory values, and other necessary evaluations.

Investigational drugs: VX-121, tezacaftor, and deutivacaftor are CFTR modulator medications that help the defective CFTR protein work more effectively, potentially improving mucus clearance in the airways and overall lung function.

Study on Long-Term Safety and Effectiveness of Vanzacaftor, Tezacaftor, and Deutivacaftor for Cystic Fibrosis in Patients Aged 1 Year and Older

This long-term study in Germany, the Netherlands, France, and Sweden is evaluating the safety and effectiveness of a triple combination therapy (vanzacaftor, tezacaftor, and deutivacaftor) in individuals aged 1 year and older. The study continues until October 2025.

Who can participate: Patients or their legal representatives must sign an informed consent form and understand study requirements. Participants must not have withdrawn consent from the previous related study and must have either completed study drug treatment or had interruptions but completed all required visits in the previous study. They must be willing to continue their current cystic fibrosis treatment plan throughout the study.

Who cannot participate: People without cystic fibrosis, those outside the specified age range, unwilling to follow study procedures, with other interfering medical conditions, who are pregnant or breastfeeding, currently participating in another clinical trial, or with a history of non-compliance with medical treatments cannot participate.

Study focus: This extension study monitors long-term safety and tolerability of the triple combination therapy. Participants will take VX-121/VX-661/VX-561 film-coated tablets orally while continuing their regular cystic fibrosis treatment. Throughout the study, health will be monitored through assessments including laboratory tests, vital signs measurements, sweat chloride levels, lung function tests, and other health indicators.

Investigational drugs: Vanzacaftor, tezacaftor, and deutivacaftor work together as CFTR modulators to enhance the function of the defective protein in cystic fibrosis, helping to improve the balance of salt and water in the lungs and reduce symptoms.

Study on the Safety and Effects of SPL84 for Patients with Cystic Fibrosis

This study across ten countries (Italy, Germany, Czechia, Lithuania, Slovakia, Poland, Netherlands, Hungary, France, and Spain) is testing SPL84, an inhaled medication specifically designed for patients with the 3849 +10 Kb C->T mutation in the CFTR gene.

Who can participate: Adults aged 18 and above with a confirmed diagnosis of cystic fibrosis and two specific genetic mutations (one being the 3849+10 Kb C->T mutation) can participate. Participants must have a body mass index of 17 kg/m² or higher, FEV1 between 40% and 90% of predicted, and stable lung function. They must be non-smokers or non-vapers for at least 180 days before screening.

Who cannot participate: People without the specific genetic mutations, outside the age range, unable to follow study procedures, with other interfering medical conditions, who are pregnant or breastfeeding, taking certain interfering medications, with recent infections or illnesses, or with a history of drug or alcohol abuse cannot participate.

Study focus: The study evaluates the safety and tolerability of SPL84 when given in multiple ascending doses through inhalation using the Aerogen InnoSpire Go nebulizer. Researchers will monitor participants for adverse events, changes in vital signs, lung function, body weight, and sweat chloride levels. The study aims to determine the maximum tolerated dose of this medication.

Investigational drug: SPL84 is administered by inhalation and is being studied for its potential to improve lung function and manage symptoms in patients with the specific 3849 +10 Kb C->T mutation, targeting and modulating pathways involved in the disease process.

Study on the Safety of Inhaled Teicoplanin for Treating Staphylococcus aureus Infections in Cystic Fibrosis Patients

This Italian study is testing whether inhaled teicoplanin is safe and well-tolerated for treating persistent Staphylococcus aureus infections, including MRSA, in people with cystic fibrosis.

Who can participate: Males and females aged 12 years or older with a confirmed diagnosis of cystic fibrosis can participate. They must have a persistent Staphylococcus aureus infection (at least three positive sputum tests in the last 24 months) and lung function (FEV1) between 50% and 90% of predicted. Participants must be able to understand the study and provide informed consent (with guardian consent if under 18).

Who cannot participate: People without persistent Staphylococcus aureus infection, outside the specified age range, unable to follow study procedures, with other interfering medical conditions, who are pregnant or breastfeeding, taking certain interfering medications, or who have participated in another recent clinical trial cannot participate.

Study focus: The treatment involves two 28-day cycles of inhaled teicoplanin at 200 mg twice daily, with a 28-day treatment-free period in between. Throughout the study, researchers will monitor safety and tolerability, measure changes in bacterial load in sputum, assess lung function, oxygen saturation, and any side effects such as coughing or chest discomfort.

Investigational drug: Teicoplanin is a glycopeptide antibiotic that works by inhibiting bacterial cell wall synthesis. When inhaled, it reaches the lungs directly to target Staphylococcus aureus bacteria, including antibiotic-resistant MRSA strains.

Study on VX-522 and Ivacaftor for Adults with Cystic Fibrosis Unresponsive to CFTR Modulator Therapy

This study across six countries (Italy, Spain, Belgium, Germany, Netherlands, and Sweden) is testing VX-522, delivered as an inhaled mist, either alone or in combination with ivacaftor tablets for adults with specific genetic types of cystic fibrosis that don’t respond to existing CFTR modulator therapies.

Who can participate: Adults aged 18 to 65 with a body mass index below 30 and weighing more than 50 kg can participate. They must have specific CFTR mutations unresponsive to current therapies, stable disease with FEV1 at least 40% of predicted, and be on a stable treatment regimen for 28 days before dosing. Participants must be nonsmokers or have quit for at least three months.

Who cannot participate: People without the specific unresponsive genetic mutations, outside the age or weight ranges, unable to follow study procedures, with other interfering medical conditions, who are pregnant or breastfeeding, currently in another clinical trial, with allergic reactions to study medications, or with recent surgery plans cannot participate.

Study focus: The study has two phases: a single ascending dose phase to evaluate safety and tolerability, and a multiple ascending dose phase with two treatment arms. One arm receives VX-522 alone via inhalation using the eFlow Nebuliser System, while the other receives VX-522 plus ivacaftor (Kalydeco 150 mg tablets) taken orally. Regular monitoring includes lung function tests, safety assessments, and measurements of how the body processes these medications.

Investigational drugs: VX-522 is an inhaled gene therapy agent being studied for its potential to improve CFTR protein function in patients with mutations unresponsive to current treatments. Ivacaftor enhances CFTR protein activity by helping protein channels stay open longer, improving salt and water movement in and out of cells.

Study of elexacaftor, tezacaftor and ivacaftor combination therapy to evaluate blood levels in patients with Cystic Fibrosis

This Italian study is measuring how the triple combination therapy of elexacaftor, tezacaftor, and ivacaftor (marketed as Kaftrio) circulates in the blood of patients already taking this treatment, and how blood levels relate to treatment response.

Who can participate: Males and females aged 6 years and older with cystic fibrosis and at least one F508del mutation can participate. They must be currently receiving this combination therapy for at least 8 days to ensure stable medication levels in the body. Parents or legal guardians must provide informed consent for children.

Who cannot participate: People with a history of organ or stem cell transplant, moderate or severe liver problems, abnormal liver function tests, abnormal kidney function, history of alcohol or drug abuse within the past year, pregnant or nursing mothers, participation in another recent clinical trial, known allergies to study medications, serious interfering medical conditions, inability to swallow tablets, or mental health conditions affecting compliance cannot participate.

Study focus: The study runs from August 2025 to July 2027 and measures medication levels in the blood after at least 8 days of treatment. Researchers will assess lung function using ppFEV1 tests, measure chloride levels, and classify participants as responders, non-responders, or unresolved responders based on their response to treatment. The goal is to understand the relationship between medication levels and treatment effectiveness.

Investigational drugs: Elexacaftor, tezacaftor, and ivacaftor work together as CFTR modulators. Elexacaftor and tezacaftor help the defective CFTR protein reach the correct location and fold properly in cells, while ivacaftor increases the protein’s activity once in place, improving lung function and breathing.

Study on Improving Bone Health in Children with Cystic Fibrosis Using Cholecalciferol

This Czech study is testing whether a higher dose of vitamin D (cholecalciferol) can improve bone health, bone density, and muscle strength in children with cystic fibrosis over two years.

Who can participate: Children aged 6 to 16 years with a confirmed diagnosis of cystic fibrosis can participate. Parents or legal representatives must provide informed consent. If the participant is a girl who can have children, she must agree to use appropriate pregnancy prevention methods during the study.

Who cannot participate: Children without cystic fibrosis, without problems with bone metabolism, bone density, or skeletal muscle strength, outside the specified age range, unable to take study medication as required, with other interfering medical conditions, taking medications affecting study results, who have participated in another recent clinical trial, or whose parents/guardians do not give consent cannot participate.

Study focus: The study compares two daily doses of cholecalciferol: 6000 IU versus 1000 IU, given as oral drops (Vigantol 0.5 mg/ml) for two years. Regular visits are scheduled to monitor health and progress. Blood tests will measure vitamin D and parathormone levels, and assessments of lumbar spine bone density and dynamic muscle force and power will be performed at the final visit.

Investigational drug: Cholecalciferol (Vitamin D3) promotes calcium absorption in the gut and maintains adequate calcium and phosphate levels in the blood, which are necessary for normal bone mineralization and healthy bone development.

Study on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis

This study across nine countries (Belgium, Italy, Denmark, Germany, Netherlands, Czechia, Portugal, Sweden, and Spain) is testing a triple combination of dirocaftor, posenacaftor, and nesolicaftor in adults with rare genetic mutations associated with cystic fibrosis.

Who can participate: Males and females aged 18 and older who completed the HIT-CF Organoid Study can participate. They must have confirmed cystic fibrosis with a sweat chloride value of 60 mmol/L or higher or two CF-causing genetic mutations, along with chronic lung or digestive issues. Participants need clinically stable disease, lung function (FEV1) between 40% and 90% of predicted, BMI between 16 and 30 kg/m², and must be non-smokers for at least 30 days before the study.

Who cannot participate: People without cystic fibrosis, outside the specified age range, unwilling to follow study procedures, with other serious interfering health conditions, who are pregnant or breastfeeding, or considered part of a vulnerable population cannot participate.

Study focus: This double-blind, crossover study lasts 16 weeks total, with participants receiving either the triple combination therapy or placebo for 8 weeks, then switching to the other treatment for another 8 weeks. Regular assessments include lung function tests (FEV1), sweat chloride measurements, weight monitoring, and respiratory symptom questionnaires.

Investigational drugs: Dirocaftor, posenacaftor, and nesolicaftor are CFTR modulators designed to correct the function of the defective CFTR protein. Together, they aim to improve protein function, reduce symptoms such as breathing difficulties and lung infections, and improve quality of life.

Study on the Safety and Tolerability of Tamoxifen in Cystic Fibrosis Patients Not Eligible for CFTR Modulator Therapy

This Italian study is exploring whether tamoxifen citrate is safe and well-tolerated in patients with cystic fibrosis who don’t have mutations currently eligible for CFTR modulator drug treatment.

Who can participate: Adults aged 18 and older with cystic fibrosis who can perform reliable lung function tests and communicate well with the study team can participate. They must not have genetic mutations eligible for CFTR modulator drugs. Lung function (ppFEV1) must be between 40% and 90% of expected, and they must have stable respiratory health for at least 3 weeks and a stable routine treatment for at least 28 days. Female participants must have negative pregnancy tests and use non-hormonal birth control during the study and for 2 months after.

Who cannot participate: People with mutations eligible for modulator drug therapy, outside the age range, unable to understand or comply with procedures, who are pregnant or breastfeeding, participated in another recent trial, or have other conditions making participation unsafe cannot participate.

Study focus: Participants will take tamoxifen citrate (TAMOXENE 20 mg) as one film-coated tablet daily for 24 weeks. The study will monitor safety and tolerability, track any side effects, measure changes in lung function, frequency of lung infections, hospitalizations, antibiotic use, body weight, and sweat chloride levels.

Investigational drug: Tamoxifen citrate is a selective estrogen receptor modulator not typically used for cystic fibrosis, but researchers are exploring whether it can help patients who cannot use existing CFTR modulator drugs. Its exact mechanism in cystic fibrosis is still under investigation.

Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor for Cystic Fibrosis in Patients Aged 12 Months and Older

This study in the Netherlands, Germany, and Denmark is evaluating the long-term safety and effectiveness of the triple combination of elexacaftor, tezacaftor, and ivacaftor in patients aged 12 months and older, continuing until August 2027.

Who can participate: Patients or their legal representatives must sign informed consent and understand study requirements. Participants must not have withdrawn consent from the previous related study and must have either completed study drug treatment or had interruptions but completed all required visits in that study. They must be willing to continue with a stable cystic fibrosis treatment plan throughout this study.

Who cannot participate: People without cystic fibrosis, outside the specified age range, unwilling to follow procedures, with other interfering medical conditions, who are pregnant or breastfeeding, taking interfering medications, participated in another recent trial, or with a history of non-compliance with treatments cannot participate.

Study focus: This open-label extension study lasts up to 96 weeks. Participants will receive elexacaftor, tezacaftor, and ivacaftor in the form of granules or oral solution (products include Kaftrio and Kalydeco). Regular monitoring includes checking for adverse events, conducting laboratory tests, measuring vital signs, and performing sweat chloride tests to evaluate treatment effectiveness.

Investigational drugs: Elexacaftor, tezacaftor, and ivacaftor are CFTR modulators that work together to improve the function of the faulty protein in cystic fibrosis. Elexacaftor and tezacaftor help the protein work better and reach the right place in cells, while ivacaftor helps the protein stay open longer, improving lung function and reducing symptoms.

Summary

The 18 ongoing clinical trials for cystic fibrosis represent a diverse range of therapeutic approaches being tested across Europe. A significant portion of these studies focuses on CFTR modulator therapies, which target the underlying genetic defect in cystic fibrosis. Multiple trials are evaluating different combinations of modulators such as elexacaftor, tezacaftor, ivacaftor, vanzacaftor, and deutivacaftor, both in new patients and as long-term extension studies.

Germany, the Netherlands, Italy, and France emerge as the countries with the highest concentration of trials, reflecting their strong research infrastructure for rare diseases. Several studies specifically target patients with rare genetic mutations or those who don’t respond to existing CFTR modulator therapies, addressing an important unmet medical need.

Beyond CFTR modulators, the trials explore other therapeutic approaches including anti-inflammatory medications (anakinra, thymosin alpha 1), antibiotics for persistent infections (teicoplanin), nutritional supplements for bone health (cholecalciferol), and novel compounds like SPL84 for specific genetic mutations. Age ranges vary widely, from infants as young as 12 months to adults, with some trials specifically designed for pediatric populations.

The studies employ various administration routes including oral tablets, inhaled medications, subcutaneous injections, and oral granules, offering potential options suitable for different patient preferences and clinical situations. Many trials emphasize long-term safety monitoring, recognizing that cystic fibrosis requires lifelong treatment. These ongoing studies collectively aim to expand treatment options, improve outcomes, and address the needs of diverse patient populations living with cystic fibrosis.

Ongoing Clinical Trials on Cystic fibrosis

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

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France The Netherlands

  • Study of Piperacillin/Tazobactam and Levofloxacin for Patients with COPD or Cystic Fibrosis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria

  • Study of VX-121, Tezacaftor, and Deutivacaftor for Children Aged 1-11 with Cystic Fibrosis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany The Netherlands Sweden

  • Study on Improving Bone Health in Children with Cystic Fibrosis Using Cholecalciferol

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia

  • Study on the Safety of Inhaled Teicoplanin for Treating Staphylococcus aureus Infections in Cystic Fibrosis Patients

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • Study on Long-Term Safety and Effectiveness of Vanzacaftor, Tezacaftor, and Deutivacaftor for Cystic Fibrosis in Patients Aged 1 Year and Older

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany The Netherlands Sweden

  • Study of elexacaftor, tezacaftor and ivacaftor combination therapy to evaluate blood levels in patients with Cystic Fibrosis

    Not yet recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on the Safety and Tolerability of Tamoxifen in Cystic Fibrosis Patients Not Eligible for CFTR Modulator Therapy

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on the Safety and Effects of SPL84 for Patients with Cystic Fibrosis

    Not yet recruiting

    1
    Investigated diseases:
    Czechia France Germany Hungary Italy Lithuania +4

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