Cystic Fibrosis research hub

A phase II trial to assess the activity and tolerability of Thymosin alpha 1 in Cystic Fibrosis Patients

This study is looking at a new treatment called Thymosin alpha 1 (brand name ZADAXIN®) for cystic fibrosis. It wants to see if this treatment can reduce inflammation in the lungs and if it's safe for patients. Researchers will check levels of inflammation markers in sputum and monitor overall health.

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

This study is testing a new medicine, VX-121/TEZ/D-IVA, for children aged 1 to 11 with cystic fibrosis. We want to see how safe and effective it is, how the body uses it, and if it helps improve their health.

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

This study is looking at a new medicine called VNZ/TEZ/D-IVA for people with cystic fibrosis, aged one year and older. It wants to see how safe and effective it is over a long time for those who have already been in a related study.

Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)

This study, called RECOVER, looks at how well the medicine Kaftrio works for people with cystic fibrosis (CF) in real life. It checks many things like breathing, tummy problems, and how people feel overall, for two years, starting with those aged 12 and over.

Analysis of Remote Monitoring/Virtual Clinic Data in Adult Patients With Cystic Fibrosis (Project Breathe)

This study looks at how home monitoring helps adults with Cystic Fibrosis. It checks if using devices like lung function testers and heart rate monitors at home can spot lung infections earlier and how people find using this technology.

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

This study is testing a new inhaled medicine, RCT2100, for cystic fibrosis. It will first check if it's safe for healthy people, then for people with CF, sometimes alongside other CF treatments. We aim to understand its safety and how the body handles it.

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

This study is testing a new medicine, VX-522, for adults with cystic fibrosis (CF) whose current treatments aren't effective. It aims to see if the medicine is safe, well-tolerated, and helpful for improving their condition. This is an important step in finding new ways to manage CF.

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

This study is looking at a new combination medicine, VX-121/TEZ/D-IVA, for people with cystic fibrosis. We want to understand how safe and effective it is over a longer period, especially for those who have been in previous studies with this medication.

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

This study looks at the long-term safety and how well a medicine called ELX/TEZ/IVA works for people with cystic fibrosis, aged 12 months and older. It continues to check on participants who have already been taking the medicine in a previous study.

Exploratory study to evaluate the safety and tolerability of tamoxifen citrate in the treatment of cystic fibrosis in patients without mutations currently eligible for therapy with CFTR modulator drugs Protocol Code: CRCFC-TAMOXI063

This study is looking at a medicine called TAMOXENE for cystic fibrosis in adults who can’t use other modern treatments. We want to see if it’s safe and if it causes any side effects. We'll also check if it helps with breathing and other CF symptoms over 24 weeks.

A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People with Cystic Fibrosis

This study is testing a new medicine, ARCT-032, for people with cystic fibrosis. Researchers want to see if it's safe, how well people tolerate it, and if it helps improve their health, especially their breathing, by giving different doses.

A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects with Chronic Pseudomonas aeruginosa (PsA) Pulmonary Infection

This study looks at a new mist treatment for adults with Cystic Fibrosis (CF) who have ongoing lung infections caused by Pseudomonas aeruginosa bacteria. We want to see if this treatment, called BX004, can reduce the amount of bacteria in their lungs after 8 weeks.

A low-intervention prospective-retrospective study to evaluate the pharmacokinetics of elexacaftor/tezacaftor/ivacaftor combination in a Cystic Fibrosis population

This study looks at how a cystic fibrosis medicine called Kaftrio works in the body. Researchers want to understand how much of the medicine gets into the blood and how long it stays there, to help make sure patients are getting the most benefit.

A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX‑828/Deutivacaftor With and Without Tezacaftor in Subjects Aged 18 Years and Older With Cystic Fibrosis

This study is looking at a new drug combination, VX-828/Deutivacaftor, to see if it's safe and helps adults with cystic fibrosis (CF). Researchers will check how well people respond and if there are any side effects compared to other treatments or placebos. It's an early-stage study to gather important information.

A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON)

This study looks at the long-term health of people with cystic fibrosis who previously took part in a trial for a medicine called BI 3720931. Participants won't receive new medicine in this study, but their health will be monitored for many years to see how they are doing.

A Phase 1/2 Dose-escalation Study Evaluating the Safety, Tolerability, and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy

A Randomised, Double-Blind, Placebo Controlled, Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of a Repeat Dose of Inhaled ETD001 in People with Cystic Fibrosis

An open label, balanced, randomized, single dose, two treatment, two period, two sequence, crossover, bioequivalence study comparing Ivacaftor film coated tablets 150 mg of Sun Pharmaceutical Industries Ltd, India with Kalydeco (Ivacaftor) 150 mg film-coated tablets, Marketing Authorisation Holder: Vertex Pharmaceuticals (Ireland) Limited Unit 49, Block F2, Northwood Court, Santry, Dublin 9, D09 T665, Ireland, in healthy adult, human subjects under fed condition.

A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age

A Phase IIb, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Subjects with Cystic Fibrosis Aged 18 Years or Older (CHOICES)

A seamless Phase I/II trial with an initial open-label dose escalation part and a subsequent randomised, double-blind, placebo-controlled expansion part to evaluate the safety, tolerability, and efficacy of a single dose of BI 3720931, an inhaled lentiviral vector gene therapy, in adult people with cystic fibrosis who are ineligible for CFTR modulators (LenticlairTM 1)

A Phase 3 Open-label Study Evaluating the Long term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older

Adding oxygen to enhance antibiotic treatment of chronic lung infection

YOGA-CF (Yoga Outcomes Get Assessed in Cystic Fibrosis)