Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04183790
• Other study ID #: VX19-445-107
• Secondary ID: 2019-001827-11
• Status: Completed
• Phase: Phase 3
• Start date: February 17, 2020
• Est. completion date: February 24, 2024

Study information

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

Clinical Trial Summary

This study will evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 64
• Est. completion date: February 24, 2024
• Est. primary completion date: February 24, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Key Inclusion Criteria:

• Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study

Key Exclusion Criteria:

• History of study drug intolerance in parent study

Other protocol defined Inclusion/Exclusion criteria may apply

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
• IVA
Mono tablet for oral administration.

Locations

Country	Name	City	State
Australia	Queensland Children's Hospital	South Brisbane
Australia	The Children's Hospital at Westmead	Westmead
Canada	The Hospital for Sick Children	Toronto
Canada	British Columbia's Children's Hospital	Vancouver
Ireland	Children's Health Ireland at Crumlin	Dublin
Ireland	Children's Health Ireland at Temple Street	Dublin
United Kingdom	Birmingham Children's Hospital - NHS Foundation Trust	Birmingham
United Kingdom	Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital	London
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Clinical Research of Charlotte	Charlotte	North Carolina
United States	Ann & Robert Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Northwell Health- Long Island Jewish Medical Center	New Hyde Park	New York
United States	Children's Hospital of Orange County	Orange	California
United States	Oregon Health & Science University	Portland	Oregon
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Ireland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability as assessed by adverse events (AEs) and serious adverse events (SAEs)		From Baseline up to Week 196
Secondary	Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)		From Baseline up to Week 192
Secondary	Absolute change in sweat chloride (SwCl)		From Baseline up to Week 192
Secondary	Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score		From Baseline up to Week 192
Secondary	Absolute change in body mass index (BMI)		From Baseline up to Week 192
Secondary	Absolute change in BMI-for-age z-score		From Baseline up to Week 192
Secondary	Number of pulmonary exacerbations (PEx)		From Baseline up to Week 192
Secondary	Number of CF-related hospitalizations		From Baseline up to Week 192
Secondary	Absolute change in lung clearance index 2.5 (LCI 2.5)		From Baseline up to Week 192
Secondary	Absolute change in weight		From Baseline up to Week 192
Secondary	Absolute change in weight-for-age z-score		From Baseline up to Week 192
Secondary	Absolute change in height		From Baseline up to Week 192
Secondary	Absolute change in height-for-age z-score		From Baseline up to Week 192