Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
- Conditions
- Cystic Fibrosis
- Interventions
- Drug: VNZ/TEZ/D-IVA
- Registration Number
- NCT05844449
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 174
Parts A and B:
• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)
Part B:
-Meets at least 1 of the following criteria:
- Completed study drug treatment in Part A
- Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A
Key
- Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
- History of solid organ, hematological transplantation, or cancer
- History of drug intolerance in the parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description VNZ/TEZ/D-IVA VNZ/TEZ/D-IVA Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.
- Primary Outcome Measures
Name Time Method Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) From Baseline up to Week 100 Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) From Baseline up to Week 196
- Secondary Outcome Measures
Name Time Method Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 96 Part B: Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 192 Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) From Baseline Through Week 100 Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) From Baseline Through Week 196 Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx) From Baseline Through Week 100 Part B: Number of Pulmonary Exacerbation (PEx) From Baseline Through Week 196 Part A (All Cohorts): Number of CF- Related Hospitalizations From Baseline Through Week 100 Part B: Number of CF- Related Hospitalizations From Baseline Through Week 196 Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score From Baseline Through Week 100 Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score From Baseline Through Week 196 Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI) From Baseline Through Week 100 Part B: Absolute Change in Body Mass Index (BMI) From Baseline Through Week 196 Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score From Baseline Through Week 100 Part B: Absolute Change in BMI-for-age Z-score From Baseline Through Week 196 Part A (Cohort 3): Absolute Change in Weight-for-length From Baseline Through Week 100 Part B: Absolute Change in Weight-for-length From Baseline Through Week 196 Part A (Cohort 3): Absolute Change in Weight-for-length Z-score From Baseline Through Week 100 Part B: Absolute Change in Weight-for-length Z-score From Baseline Through Week 196 Part A (All Cohorts): Absolute Change in Weight From Baseline Through Week 100 Part B: Absolute Change in Weight From Baseline Through Week 196 Part A (All Cohorts): Change in Weight-for-age Z-score From Baseline Through Week 100 Part B: Change in Weight-for-age Z-score From Baseline Through Week 196 Part A (Cohorts 1 and 2): Absolute Change in Height From Baseline Through Week 100 Part B: Absolute Change in Height From Baseline Through Week 196 Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score From Baseline Through Week 100 Part B: Absolute Change in Height-for-age Z-score From Baseline Through Week 196 Part A (Cohort 3): Absolute Change in Length From Baseline Through Week 100 Part B: Absolute Change in Length From Baseline Through Week 196 Part A (Cohort 3): Absolute Change in Length-for-age Z-score From Baseline Through Week 100 Part B: Absolute Change in Length-for-age Z-score From Baseline Through Week 196
Trial Locations
- Locations (32)
Inselspital - Universitaetsspital Bern
🇨🇭Bern, Switzerland
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Women & Children's Hospital
🇦🇺North Adelaide, Australia
Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
The Royal Childrens Hospital
🇦🇺Parkville, Australia
Children and Young Adults Research Unit
🇬🇧Cardiff, United Kingdom
Hopital Necker, Enfants Malades
🇫🇷Paris Cedex 15, France
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
The Emory Clinic / Children's Healthcare of Atlanta at Egleston
🇺🇸Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Vermont Lung Center
🇺🇸Colchester, Vermont, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Stanford University
🇺🇸Palo Alto, California, United States
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Cohen Children's Medical Center
🇺🇸Lake Success, New York, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
CHU Lyon - Hopital Femme Mere-Enfant
🇫🇷Bron Cedex, France
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Erasmus Medical Center / Sophia Children's Hospital
🇳🇱Rotterdam, Netherlands
Kinderspital Zuerich
🇨🇭Zürich, Switzerland