Respiratory Medicine


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:

  • We can ensure each paediatric CF centre in Ireland can offer clinical trials opportunities to their patients.
  • Cross cover arrangements between centres depending on how many children in each centre can participate in a given trial.
  • Ongoing facilitated access to clinical trials will improve centre level knowledge, expertise and experience in clinical trials, making future investigator led trials more realistic.

Patients and Families:

  • All children in Ireland will be afforded the opportunity to participate in clinical trials in their own centre
  • As a community, CF families will take comfort from the knowledge that the paediatric CF care network in Ireland is unified at the forefront of advances in therapies

Pharmaceutical Companies:

  • The centres together look after more than 500 children with CF in Ireland
  • We will provide a central access point through which companies can gather information about potential numbers of subjects for prospective studies.
  • The operation of the same study across multiple sites has advantages in terms of education of CRAs, uniformity of practice and peer support.
  • The adoption of national agreed SOPs in terms of operation of clinical trials in centres will enhance and standardise quality of practice.
  • The drive of the CF centres involved to maintain a consistent presence of clinical trials in their centres operated at a high standard will ensure reliable access to patient groups longitudinally for future studies.
  • Having several centres in the same country involved in trials provides efficiencies in terms of ethics applications, monitoring and communication.

For more information on Cystic Fibrosis research supported by the NCRC please click here.

 

Current Trials & Studies

Study NameStudy TitleStudy TypeSponsorSponsor Type
SHIELD CFThe Study of Host Immunity and Early Lung Disease in Children with CFNon-Interventional Study
VX17-661-116A 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 MutationClinical TrialVertexIndustry Sponsored
VX17-659-105A 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 MutationClinical TrialVertexIndustry Sponsored
PREDiCTPredicting Response in Disease outcomes in CF using iPSCs for new CFTR Therapies (PREDiCT)Non-interventional study
CFORMSChildren’s Follow up Orkambi Real world MBW Study (CFORMS)Non-interventional study
VX15-770-124A 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 MutationClinical TrialVertexIndustry Sponsored
VX15-770-126A 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 TrialVertexIndustry Sponsored
RECAPThe role of exosomes in cystic fibrosis airway pathogenesisNon-interventional study
VX18-445-104A 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 TrialVertexIndustry Sponsored
VX18-445-106A 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 TrialVertexIndustry Sponsored
VX18-445-110A 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 TrialVertexIndustry Sponsored
VX18-445-113A Phase 3, Open-label Study evaluating the long-term Safety of VX-445 Combination Therapy in Subjects with Cystic Fibrosis.Clinical TrialVertexIndustry Sponsored
VX19-445-107A 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 TrialVertexIndustry Sponsored
RECOVERReal World Clinical Outcomes with Novel Modifier Therapy Combinations in People with CF.Non-Interventional StudyRCSIAcademic- Investigator Led
SAINTSStudy of Airway Infection in the Nose, Throat and Sinuses in Children with CF.Non-Interventional Study
SeagullsStudy to Evaluate the Additional Gains of Upper and Lower Lobe Sampling in Children with CF.Non-Interventional Study

Past Trials & Studies

Study NameStudy TitleStudy TypeSponsorSponsor Type
VX15-371-101A 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 OrkambiClinical TrialVertexIndustry Sponsored
VX14-661-110A 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 MutationClinical TrialVertexIndustry Sponsored
VX16-661-115A 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 MutationClinical TrialVertexIndustry Sponsored
VX17-659-103A 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 TrialVertexIndustry Sponsored
VOICE: VX14-CFR-107Observational Registry of Cystic Fibrosis Patients in EuropeNon-interventional studyVertexIndustry Sponsored
VX17-659-102A 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 TrialVertexIndustry Sponsored
VX14-661-106A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With IvacaftorClinical TrialVertexIndustry Sponsored
VX14-661-109A 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 MutationClinical TrialVertexIndustry Sponsored
VX12-809-103A 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 TrialVertexIndustry Sponsored
VX12-809-105A 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 MutationClinical TrialVertexIndustry Sponsored
MPEX-209A 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 patientsClinical TrialMpex Pharmaceuticals Inc.Industry Sponsored
VX08-770-103A 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 mutationClinical TrialVertexIndustry Sponsored
VX08-770-105An Open Label, Rollover Study to Evaluate the Long-Term Safety and Efficacy of VX-770 in Subjects with Cystic FibrosisClinical TrialVertexIndustry Sponsored
TR02-108Randomised, 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 AeruginosaClinical TrialInsmed IncIndustry Sponsored
TR02-110Long term safety and tolerability study of open-label liposomal amikacin for inhalation (Arikace) in Cystic Fibrosis patients with chronic infection due to Pseudomonas AeruginosaClinical TrialInsmed IncIndustry Sponsored
PsAer-IgYProspective 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 patientsClinical TrialMukoviszidosAcademic - Collaborative Group
Caregiver Burden StudyCaregiver Burden Study in Cystic Fibrosis in the United States, Germany, United Kingdom and IrelandNon-Interventional StudyVertexIndustry Sponsored
The POOL studyInflammatory and Microbiological Implications of Pooling of Bronchoalveolar Lavage samples in Preschool Children with Cystic FibrosisNon-Interventional Study
PALM StudyPALM 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 JulyNon-Interventional Study
Rhinovirus pilot studyThe Role of Human Rhinovirus Infections in Young Children with Cystic Fibrosis - A Pilot StudyNon-Interventional Study
BLOC-WORCBaseline lung clearance index of children with or without CFNon-interventional study
CUBS (CF Urinary biomarker study)CF Urinary biomarker studyNon-interventional study