Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05844449
• Other study ID #: VX22-121-106
• Secondary ID: 2022-503081-74-0
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: August 11, 2023
• Est. completion date: October 2030

Study information

• Verified date: April 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 180
• Est. completion date: October 2030
• Est. primary completion date: October 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

Key Inclusion Criteria:

• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Key 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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VNZ/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.

Locations

Country	Name	City	State
Australia	Women & Children's Hospital	North Adelaide
Australia	The Royal Childrens Hospital	Parkville
Australia	Queensland Children's Hospital	South Brisbane
France	CHU Lyon - Hopital Femme Mere-Enfant	Bron Cedex
France	Hopital Necker, Enfants Malades	Paris Cedex 15
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Kinderklinik III, Abt. fur Pneumologie	Essen
Germany	Medizinische Hochschule Hannover	Hannover
Netherlands	Erasmus Medical Center / Sophia Children's Hospital	Rotterdam
Sweden	Sahlgrenska Universitetssjukhuset	Göteborg
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Kinderspital Zuerich	Zürich
United Kingdom	Children and Young Adults Research Unit	Cardiff
United Kingdom	Great Ormond Street Hospital for Children	London
United States	The Emory Clinic / Children's Healthcare of Atlanta at Egleston	Atlanta	Georgia
United States	Children's Hospital of Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Vermont Lung Center	Colchester	Vermont
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	Cohen Children's Medical Center	Lake Success	New York
United States	University of Wisconsin Hospital and Clinics	Madison	Wisconsin
United States	Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Children's Hospital of Orange County	Orange	California
United States	Stanford University	Palo Alto	California
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine / St. Louis Children's Hospital	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  France,  Germany,  Netherlands,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)		From Day 1 up to Week 100
Secondary	All Cohorts: Absolute Change in Sweat Chloride (SwCl)		From Baseline Through Week 96
Secondary	Cohort 1: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)		From Baseline Through Week 100
Secondary	All Cohorts: Number of Pulmonary Exacerbation (PEx) Related Hospitalizations		From Baseline Through Week 100
Secondary	All Cohorts: Number of CF- Related Hospitalizations		From Baseline Through Week 100
Secondary	Cohort 1: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score		From Baseline Through Week 100
Secondary	Cohorts 1 and 2: Absolute Change in Body Mass Index (BMI)		From Baseline Through Week 100
Secondary	Cohorts 1 and 2: Absolute Change in BMI-for-age Z-score		From Baseline Through Week 100
Secondary	Cohort 3: Absolute Change in Weight-for-length		From Baseline Through Week 100
Secondary	Cohort 3: Absolute Change in Weight-for-length Z-score		From Baseline Through Week 100
Secondary	All Cohorts: Absolute Change in Weight		From Baseline Through Week 100
Secondary	All Cohorts: Change in Weight-for-age Z-score		From Baseline Through Week 100
Secondary	Cohorts 1 and 2: Absolute Change in Height		From Baseline Through Week 100
Secondary	Cohorts 1 and 2: Absolute Change in Height-for-age Z-score		From Baseline Through Week 100
Secondary	Cohort 3: Absolute Change in Length		From Baseline Through Week 100
Secondary	Cohort 3: Absolute Change in Length-for-age Z-score		From Baseline Through Week 100