ALPINE 2: AZLI for children with CF and PA lung infection – Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization (Gilead Pharmaceuticals). Opening soon. Commercial

VX15-770-126 ARRIVAL Extension Study: 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 (Vertex Pharmaceuticals). Ongoing. Commercial

VX15-770-124 ARRIVAL Study: 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 (Vertex Pharmaceuticals). Completed. Commercial

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 (Vertex Pharmaceuticals). Completed. Commercial

LCI –SEARCH: Lung Clearance Index (LCI) as a Sensitive measure of Early Airway Change in Cystic Fibrosis (Use of lung clearance index to improve detection of changes in lung function in cystic fibrosis). Non-commercial. Completed

Caregiver Burden in Cystic Fibrosis: Exploring the caregiver burden of Cystic Fibrosis in the United States, Germany, United Kingdom, and Ireland (Vertex Pharmaceuticals). Completed. Commercial

• VX24-445-130 EPIC Study. Faecal Elastase Study in babies less than 12 months old. Commercial. Commencing soon

• STOP 360: Standardised Treatment of Pulmonary Exacerbations (Co PI). Non-commercial. Commencing soon

• ENHANCE: Establishing Natural History in an Advanced New CF Care Era. Non-commercial. Commencing soon

• Skyline VX20-121-104 A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis(Vertex Pharmaceuticals). Commercial Ongoing

• Skyline VX20-121-103 Randomized, double-blind controlled study evaluating the efficacy and safety of VX-121 combination therapy in subjects with cystic fibrosis. (Vertex Pharmaceuticals). Commercial Completed

• The Right Intake of Vitamins Evaluation (THRIVE) Registry Non-commercial Ongoing

• Adolescent Management of CF PhD: PhD Supervision (University of Manchester) Non-commercial Ongoing

• NIHR Bioresource: Rare Disease blood sample collection for genetic analysis and storage within national network. Non-commercial (2022). Ongoing

• KAFPATH-CF Effects of Kaftrio on pathogen infection and the wider lung microbiota in cystic fibrosis. Non – commercial Ongoing

• CFSPID Qualitative Study: Supervising PhD study at University of Manchester (2022 – 2024). Non-commercial Ongoing

• Cystic Fibrosis Registry (CF Trust). Non-commercial Ongoing

• CF STORM A randomised open label trial to assess change in respiratory function for people with cystic fibrosis (pwCF) established on triple combination therapy (Kaftrio™) after rationalisation of nebulised mucoactive therapies. Non – commercial Ongoing

• VX-CFD-004 / MAGNIFY Study Quality of Life for Cystic Fibrosis Patients Treated With Orkambi® or Symkevi® and for Primary Caregivers in the United Kingdom. Commercial Completed

• TrIFIC (Targeting Immunotherapy for Fungal Infection in CF): Non-commercial Completed

• CF START (Co PI). The cystic fibrosis (CF) anti-staphylococcal antibiotic prophylaxis trial (CF START); a randomised registry trial to assess the safety and efficacy of flucloxacillin as a long-term prophylaxis agent for infants with CF. Non – commercial Ongoing

• VX17-445-105. 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 Homozygous or Heterozygous for the F508del Mutation. (Vertex Pharmaceuticals). Commercial Ongoing

• VX17-445-102. 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 F508del mutation and minimal function mutation. (Vertex Pharmaceuticals). Commercial Completed

• ALPINE 2: AZLI for children with CF and PA lung infection - Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization (Gilead Pharmaceuticals). Commercial Completed

• VX15-770-126. ARRIVAL Extension Study. 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 (Vertex Pharmaceuticals). Commercial. Completed

• Experiences of Living with Cystic Fibrosis; The impact on children, young people, adults and their families. Non-commercial. Completed

• 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 (Vertex Pharmaceuticals). Commercial. Completed

• 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 (Vertex Pharmaceuticals). Commercial. Completed

• LCI –SEARCH. Lung Clearance Index (LCI) as a Sensitive measure of Early Airway Change in Cystic Fibrosis (Use of lung clearance index to improve detection of changes in lung function in cystic fibrosis). Non-commercial. Completed

• Caregiver Burden in Cystic Fibrosis in the United States, Germany, United Kingdom, and Ireland (Vertex Pharmaceuticals). Commercial. Completed

• Observational Registry of Cystic Fibrosis Patients in Europe (VOICE) (Vertex Pharmaceuticals). Commercial. Completed