Enrolling by invitation NCT05844449
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: VNZ/TEZ/D-IVA.
- Who it may be relevant to
- Registry conditions: Cystic Fibrosis. Basic parameters: from 1 year · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, France, Germany +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older
Overview
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).
Interventions
- Drug VNZ/TEZ/D-IVA
Fixed-dose combination tablets or granules for oral administration.
Primary outcome measures
- Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) [Time frame: From Baseline up to Week 100]
- Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) [Time frame: From Baseline up to Week 196]
Secondary outcome measures (12)
- Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl) [Time frame: From Baseline Through Week 96]
- Part B: Absolute Change in Sweat Chloride (SwCl) [Time frame: From Baseline Through Week 192]
- Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) [Time frame: From Baseline Through Week 100]
- Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) [Time frame: From Baseline Through Week 196]
- Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx) [Time frame: From Baseline Through Week 100]
- Part B: Number of Pulmonary Exacerbation (PEx) [Time frame: From Baseline Through Week 196]
- Part A (All Cohorts): Number of CF- Related Hospitalizations [Time frame: From Baseline Through Week 100]
- Part B: Number of CF- Related Hospitalizations [Time frame: From Baseline Through Week 196]
- Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [Time frame: From Baseline Through Week 100]
- Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [Time frame: From Baseline Through Week 196]
- Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI) [Time frame: From Baseline Through Week 100]
- Part B: Absolute Change in Body Mass Index (BMI) [Time frame: From Baseline Through Week 196]
Eligibility criteria
Inclusion criteria
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
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 18 centers
- Children's Hospital of Orange County — Orange
- Stanford University - Palo Alto - Pulmonology — Palo Alto
- Children's Hospital of Colorado - Pulmonology — Aurora
- The Emory Clinic - Clifton Road - Pulmonology — Atlanta
- Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology — Chicago
- JW Riley Hospital for Children - Pulmonology — Indianapolis
- Boston Children's Hospital — Boston
- Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota — Minneapolis
- … and 10 more centers
Australia · 4 centers
- The Kids Research Institute Australia — Nedlands
- Women's & Children's Hospital — North Adelaide
- The Royal Children's Hospital Melbourne — Parkville
- Children's Health Queensland Hospital and Health Service — South Brisbane
Germany · 3 centers
- Charite Paediatric Pulmonology Department — Berlin
- Universitatsklinikum Essen — Essen
- Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonat — Hanover
Canada · 2 centers
- Hospital for Sick Children - Pulmonology — Toronto
- British Columbia Children's Hospital — Vancouver
France · 2 centers
- Hopital Femme Mere-Enfant — Bron
- Hopital Necker Enfants Malades - Pulmonology — Paris
Sweden · 2 centers
- Sahlgrenska Universitetssjukhuset - Göteborg CF-center — Gothenburg
- Karolinska University Hospital - Pulmonology — Stockholm
Switzerland · 2 centers
- Inselspital Bern — Bern
- Kinderspital Zurich - Abteilung Pneumologie — Zurich
United Kingdom · 2 centers
- Noah's Ark Children's Hospital for Wales — Cardiff
- Great Ormond Street Hospital for Children — London
Netherlands · 1 center
- Sophia Children's Hospital — Rotterdam
New Zealand · 1 center
- Starship Child Health — Grafton
Identifiers
NCT: NCT05844449 · VX22-121-106 · 2022-503081-74-00