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NCT05153317 Clinical Trial

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

Cystic Fibrosis Clinical Trial

Official title:
A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT05153317
Other study ID # VX20-445-112
Secondary ID 2020-002239-31
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date January 17, 2022
Est. completion date April 2026

Study information
Verified date March 2024
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 71
Est. completion date April 2026
Est. primary completion date April 2026
Accepts healthy volunteers No
Gender All
Age group 2 Years and older
Eligibility key Inclusion Criteria: - Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study Key Exclusion Criteria: - History of study drug intolerance in the parent study - History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study Other protocol defined Inclusion/Exclusion criteria may apply

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination granules or tablets for oral administration.
• IVA
Granules or tablets for oral administration.

Locations
Country Name City State
Australia Telethon Kids Institute Nedlands
Australia The Royal Children's Hospital Parkville
Australia Queensland Children's Hospital South Brisbane
Canada The Hospital for Sick Children Toronto
Canada British Columbia Children's Hospital Vancouver
Germany Charite Paediatric Pulmonology Department Berlin
Germany Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie Essen
United Kingdom Alder Hey Children's NHS Foundation Trust Liverpool
United Kingdom Royal Brompton Hospital London
United States Children's Hospital of Colorado Aurora Colorado
United States Boston Children's Hospital Boston Massachusetts
United States NC TraCS Institute - CTRC University of North Carolina at Chapel Hill Chapel Hill North Carolina
United States Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States Riley Hospital for Children at Indiana University Health Indianapolis Indiana
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 Stanford University Palo Alto California
United States UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
United States Oregon Health & Science University Portland Oregon
United States Washington University School of Medicine / St. Louis Children's Hospital Saint Louis Missouri
United States Seattle Children's Hospital Seattle Washington
United States Banner University of Arizona Medical Center Tucson Arizona

Sponsors (1)
Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Germany,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Day 1 up to Week 196
Secondary Absolute Change in Sweat Chloride (SwCl) From Baseline up to Week 192
Secondary Absolute Change in Lung Clearance Index (LCI) 2.5 From Baseline up to Week 192
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