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 in the world. Our group is committed to bringing high quality clinical research and clinical trials to children in Ireland with CF. Starting in NCRC and CHI at Crumlin in 2010, we have grown our portfolio of clinical trials and clinical research projects. We currently have active clinical trials running in 3 of the 6 CF specialist centres and investigator led studies in 4 of the 6 centres. We have developed processes to allow us to take advantage of significant overlap in study design and reduce duplication by running the same study in several sites in parallel. Our goal is to have full time clinical research coordinators in place in each of the CF centres to allow increased opportunity to enroll in future trials and ensure high participation in clinical research.
There are a number of specialist CF centres for children in this country covering all children referred through the CF national newborn screening programme. These centres have excellent channels of communication and we meet as a group on a regular basis. We collaborate on a number of CF and non-CF service development and guideline projects. Our goal is to collectively share resources and expertise to expand and develop CF research in children in Ireland and to bring clinical trials to all children in all centres. This strategy has the following benefits:
CF Centres:
Patients and Families:
Pharmaceutical Companies:
For more information on Cystic Fibrosis research supported by the NCRC please click here.
| 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 | ||
| VX17-661-116 | A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation | Clinical Trial | Vertex | Industry Sponsored |
| VX17-659-105 | A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation | Clinical Trial | Vertex | Industry Sponsored |
| PREDiCT | Predicting Response in Disease outcomes in CF using iPSCs for new CFTR Therapies (PREDiCT) | Non-interventional study | ||
| CFORMS | Children’s Follow up Orkambi Real world MBW Study (CFORMS) | Non-interventional study | ||
| 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 |
| RECAP | The role of exosomes in cystic fibrosis airway pathogenesis | Non-interventional study | ||
| VX18-445-104 | A phase 3, randomized, double blind, controlled study evaluating the efficacy and safety of VX-445 combination therapy in subjects with cystic fibrosis who are heterozygous for the FR508del mutation and a gating or residual function mutation (F/G and F/RF genotypes) | Clinical Trial | Vertex | Industry Sponsored |
| VX18-445-106 | A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age. | Clinical Trial | Vertex | Industry Sponsored |
| VX18-445-110 | A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes). | Clinical Trial | Vertex | Industry Sponsored |
| VX18-445-113 | A Phase 3, Open-label Study evaluating the long-term Safety of VX-445 Combination Therapy in Subjects with Cystic Fibrosis. | Clinical Trial | Vertex | Industry Sponsored |
| VX19-445-107 | A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEX/IVA Combination Therapy in Subjects with Cystic Fibrosis Who are 6 years of age and older. | Clinical Trial | Vertex | Industry Sponsored |
| RECOVER | Real World Clinical Outcomes with Novel Modifier Therapy Combinations in People with CF. | Non-Interventional Study | RCSI | Academic- Investigator Led |
| SAINTS | Study of Airway Infection in the Nose, Throat and Sinuses in Children with CF. | Non-Interventional Study | ||
| Seagulls | Study to Evaluate the Additional Gains of Upper and Lower Lobe Sampling in Children with CF. | Non-Interventional Study |
| 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 |
| VX14-661-110 | 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 |
| VX16-661-115 | A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | Clinical Trial | Vertex | Industry Sponsored |
| VX17-659-103 | A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) | Clinical Trial | Vertex | Industry Sponsored |
| VOICE: VX14-CFR-107 | Observational Registry of Cystic Fibrosis Patients in Europe | Non-interventional study | Vertex | Industry Sponsored |
| VX17-659-102 | A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) | 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 | ||
| 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 | ||
| Rhinovirus pilot study | The Role of Human Rhinovirus Infections in Young Children with Cystic Fibrosis - A Pilot Study | Non-Interventional Study | ||
| BLOC-WORC | Baseline lung clearance index of children with or without CF | Non-interventional study | ||
| CUBS (CF Urinary biomarker study) | CF Urinary biomarker study | Non-interventional study |
