Evaluating a new treatment for cystic fibrosis in young children

A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age

This study is testing a new combination treatment for cystic fibrosis in young children to see how well it works and if it's safe for them.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 210 (estimated)
• Ages: 1 Year to 11 Years
• Sex: All
• Sponsor: Vertex Pharmaceuticals Incorporated (Industry-sponsored)
• Locations: 38 sites (Orange, California and 37 other locations)
• Trial ID: NCT05422222 on ClinicalTrials.gov

What this trial studies

This study evaluates the pharmacokinetics, safety, tolerability, and efficacy of a combination therapy consisting of VX-121, tezacaftor, and deutivacaftor in children aged 1 to 11 years with cystic fibrosis. Participants must have at least one triple combination responsive mutation in the CFTR gene. The study aims to determine how well this treatment works and its safety profile in a pediatric population. It is a Phase 3 interventional trial, indicating a focus on confirming efficacy and monitoring adverse effects.

Who should consider this trial

Good fit: Ideal candidates are children aged 1 to 11 years with stable cystic fibrosis and at least one TCR mutation in the CFTR gene.

Not a fit: Patients with a history of solid organ or hematological transplantation, cancer, or severe hepatic impairment may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the health and quality of life for young patients with cystic fibrosis.

How similar studies have performed: Other studies have shown success with similar combination therapies in cystic fibrosis, indicating a promising approach.

Eligibility criteria

Key Inclusion Criteria:

* Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene

Key Exclusion Criteria:

* History of solid organ, hematological transplantation, or cancer
* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
* Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply.

Where this trial is running

Orange, California and 37 other locations

• Children's Hospital of Orange County — Orange, California, United States (Completed)
• Stanford University Clinical and Translational Research Unit — Palo Alto, California, United States (Completed)
• Children's Hospital of Colorado — Aurora, Colorado, United States (Not_yet_recruiting)
• The Emory Clinic / Children's Healthcare of Atlanta at Egleston — Atlanta, Georgia, United States (Completed)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Completed)
• Riley Hospital for Children at Indiana University Health — Indianapolis, Indiana, United States (Not_yet_recruiting)
• Boston Children's Hospital — Boston, Massachusetts, United States (Not_yet_recruiting)
• Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota — Minneapolis, Minnesota, United States (Completed)
• The Children's Mercy Hospital — Kansas City, Missouri, United States (Completed)
• Washington University School of Medicine / St. Louis Children's Hospital — St Louis, Missouri, United States (Completed)
• Cohen Children's Medical Center — Lake Success, New York, United States (Completed)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Completed)
• Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States (Completed)
• Nationwide Children's Hospital — Columbus, Ohio, United States (Completed)
• Oregon Health & Science University — Portland, Oregon, United States (Completed)
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (Completed)
• Texas Children's Hospital - Wallace Tower — Houston, Texas, United States (Completed)
• Vermont Lung Center — Colchester, Vermont, United States (Completed)
• American Family Childrens Hospital — Madison, Wisconsin, United States (Completed)
• The Kids Research Institute Australia — Nedlands, Australia (Not_yet_recruiting)
• Women's and Children's Hospital — North Adelaide, Australia (Not_yet_recruiting)
• The Royal Children's Hospital — Parkville, Australia (Not_yet_recruiting)
• Queensland Children's Hospital — South Brisbane, Australia (Not_yet_recruiting)
• The Hospital for Sick Children — Toronto, Canada (Not_yet_recruiting)
• British Columbia Children's Hospital — Vancouver, Canada (Not_yet_recruiting)
• CHU Lyon - Hopital Femme Mere-Enfant — Bron, France (Not_yet_recruiting)
• Hopital Necker, Enfants Malades — Paris, France (Not_yet_recruiting)
• Charité - Paediatric Pulmonology Department — Berlin, Germany (Not_yet_recruiting)
• Universitatsklinikum Essen — Essen, Germany (Not_yet_recruiting)
• Medizinische Hochschule Hannover — Hanover, Germany (Not_yet_recruiting)
• Erasmus Medical Center / Sophia Children's Hospital — Rotterdam, Netherlands (Not_yet_recruiting)
• Starship Children's Hospital — Grafton, New Zealand (Completed)
• Sahlgrenska Universitetssjukhuset — Gothenburg, Sweden (Completed)
• Karolinska University Hospital - Pulmonology — Stockholm, Sweden (Not_yet_recruiting)
• Inselspital - Universitaetsspital Bern — Bern, Switzerland (Recruiting)
• Kinderspital Zurich — Zurich, Switzerland (Not_yet_recruiting)
• Children and Young Adults Research Unit — Cardiff, United Kingdom (Not_yet_recruiting)
• Great Ormond Street Hospital for Children — London, United Kingdom (Not_yet_recruiting)

Study contacts

• Study coordinator: Medical Information

Conditions Cystic Fibrosis