Cystic Fibrosis (CF) is a genetic condition resulting in mainly respiratory and gastrointestinal problems, and is associated with early death from lung damage and respiratory failure. CF is more common in Ireland than anywhere else. Our CF research team led by Professor Paul McNally is passionate about putting Ireland at the forefront of paediatric CF research internationally. Our CF research programme has three main strands – we believe that developing and integrating these strands will drive significant improvements in the care and outcomes for our patients with CF.
Our philosophy is based around establishing a comprehensive national network for high quality CF research, with NCRC at the centre. We want to create strong links between patient care and research to drive improvements in outcomes for children with this debilitating disease.
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| SHIELD CF | The Study of Host Immunity and Early Lung Disease in Children with CF | Non-Interventional Study | NCRC | Academic - Investigator Led |
| VX14-661-110 | A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | Clinical Trial | Vertex | Industry Sponsored |
| VOICE: VX14-CFR-107 | Observational Registry of Cystic Fibrosis Patients in Europe | Non-interventional study | Vertex | Industry Sponsored |
| CUBS (CF Urinary biomarker study) | CF Urinary biomarker study | Non-interventional study | NCRC | Academic - Investigator Led |
| PALM Study | PALM study (Protease activity: Lung measurement) – a longitudinal study in children with CF to establish spectrum and influence of protease antiprotease activity in CF Starting in July | Non-Interventional Study | NCRC | Academic - Investigator Led |
| VX15-770-124 | A Phase 3, 2-Part, Open-label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation | Clinical Trial | Vertex | Industry Sponsored |
| VX15-770-126 | A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation. | Clinical Trial | Vertex | Industry Sponsored |
| Study Name | Study Title | Study Type | Sponsor | Sponsor Type |
|---|---|---|---|---|
| VX15-371-101 | A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi | Clinical Trial | Vertex | Industry Sponsored |
| VX14-661-106 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor | Clinical Trial | Vertex | Industry Sponsored |
| VX14-661-109 | A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation | Clinical Trial | Vertex | Industry Sponsored |
| VX12-809-103 | A Phase 3, Randomised, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination with Ivacaftor in Subjects Aged 12 years and older with Cystic Fibrosis, Homozygous for the F508del CFTR Mutation (TRAFFIC) | Clinical Trial | Vertex | Industry Sponsored |
| VX12-809-105 | A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | Clinical Trial | Vertex | Industry Sponsored |
| MPEX-209 | A Phase 3, Open-label, Randomised Trial to Evaluate the Safety and Efficacy of MP-376 Inhalation solution (Aeroquin) versus Tobramycin Inhalation Solution in stable Cystic Fibrosis patients | Clinical Trial | Mpex Pharmaceuticals Inc. | Industry Sponsored |
| VX08-770-103 | A Phase 3, 2-Part, Randomised, Double-Blind, Placebo-controlled, Parallel Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX-770 in Subjects Aged 6 -11 years with Cystic Fibrosis and G551D mutation | Clinical Trial | Vertex | Industry Sponsored |
| VX08-770-105 | An Open Label, Rollover Study to Evaluate the Long-Term Safety and Efficacy of VX-770 in Subjects with Cystic Fibrosis | Clinical Trial | Vertex | Industry Sponsored |
| TR02-108 | Randomised, Open Label, Active controlled, multicentre study to assess the efficacy, safety and tolerability of ArikaceTM in Cystic Fibrosis Patients with Chronic Infection due to Pseudomonas Aeruginosa | Clinical Trial | Insmed Inc | Industry Sponsored |
| TR02-110 | Long term safety and tolerability study of open-label liposomal amikacin for inhalation (Arikace) in Cystic Fibrosis patients with chronic infection due to Pseudomonas Aeruginosa | Clinical Trial | Insmed Inc | Industry Sponsored |
| PsAer-IgY | Prospective randomised, placebo controlled, double-blind, multicentre study (Phase III) to evaluate clinical efficacy and safety of avian polyclonal anti-Pseudomonas antibodies (IgY) in prevention of recurrence of Pseudomonas aeruginosa infection in cystic fibrosis patients | Clinical Trial | Mukoviszidos | Academic - Collaborative Group |
| Caregiver Burden Study | Caregiver Burden Study in Cystic Fibrosis in the United States, Germany, United Kingdom and Ireland | Non-Interventional Study | Vertex | Industry Sponsored |
| The POOL study | Inflammatory and Microbiological Implications of Pooling of Bronchoalveolar Lavage samples in Preschool Children with Cystic Fibrosis | Non-Interventional Study | NCRC | Academic - Investigator Led |
| Rhinovirus pilot study | The Role of Human Rhinovirus Infections in Young Children with Cystic Fibrosis - A Pilot Study | Non-Interventional Study | NCRC | Academic - Investigator Led |
