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
Status: Recruiting
Location: See all (38) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 11
Healthy Volunteers: f
View:
• Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
Locations
United States
California
Children's Hospital of Orange County
RECRUITING
Orange
Stanford University Clinical and Translational Research Unit
RECRUITING
Palo Alto
Colorado
Children's Hospital of Colorado
RECRUITING
Aurora
Georgia
The Emory Clinic / Children's Healthcare of Atlanta at Egleston
RECRUITING
Atlanta
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Indiana
Riley Hospital for Children at Indiana University Health
RECRUITING
Indianapolis
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Minnesota
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
RECRUITING
Minneapolis
Missouri
The Children's Mercy Hospital
RECRUITING
Kansas City
Washington University School of Medicine / St. Louis Children's Hospital
RECRUITING
St Louis
New York
Cohen Children's Medical Center
COMPLETED
Lake Success
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
RECRUITING
Cleveland
Nationwide Children's Hospital
RECRUITING
Columbus
Oregon
Oregon Health & Science University
COMPLETED
Portland
Pennsylvania
UPMC Children's Hospital of Pittsburgh
RECRUITING
Pittsburgh
Texas
Texas Children's Hospital - Wallace Tower
RECRUITING
Houston
Vermont
Vermont Lung Center
RECRUITING
Colchester
Wisconsin
American Family Childrens Hospital
ACTIVE_NOT_RECRUITING
Madison
Other Locations
Australia
The Kids Research Institute Australia
RECRUITING
Nedlands
Women's and Children's Hospital
RECRUITING
North Adelaide
The Royal Children's Hospital
RECRUITING
Parkville
Queensland Children's Hospital
RECRUITING
South Brisbane
Canada
The Hospital for Sick Children
RECRUITING
Toronto
British Columbia Children's Hospital
RECRUITING
Vancouver
France
CHU Lyon - Hopital Femme Mere-Enfant
RECRUITING
Bron
Hopital Necker, Enfants Malades
RECRUITING
Paris
Germany
Charité Universitätsmedizin BerlinX
RECRUITING
Berlin
Kinderklinik III, Abt. fur Pneumologie
RECRUITING
Essen
Medizinische Hochschule Hannover
RECRUITING
Hanover
Netherlands
Erasmus Medical Center / Sophia Children's Hospital
RECRUITING
Rotterdam
New Zealand
Starship Children's Hospital
RECRUITING
Grafton
Sweden
Sahlgrenska Universitetssjukhuset
COMPLETED
Gothenburg
Karolinska Universitetssjukhuset, Huddinge
RECRUITING
Stockholm
Switzerland
Inselspital - Universitaetsspital Bern
COMPLETED
Bern
Kinderspital Zurich
RECRUITING
Zurich
United Kingdom
Children and Young Adults Research Unit
RECRUITING
Cardiff
Great Ormond Street Hospital for Children
RECRUITING
London
Contact Information
Primary
Medical Information
medicalinfo@vrtx.com
617-341-6777
Time Frame
Start Date: 2022-06-21
Estimated Completion Date: 2030-06-30
Participants
Target number of participants: 210
Treatments
Experimental: Part A: VX-121/TEZ/D-IVA
Participants will receive VX-121/TEZ/D-IVA in the morning.
Experimental: Part B: VX-121/TEZ/D-IVA
Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
Related Therapeutic Areas
Sponsors
Leads: Vertex Pharmaceuticals Incorporated