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Active, Not Recruiting

NCT Number: NCT05331183

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Medizinische Universität Innsbruck - Heilstättenschule Universitätsklinik, Innsbruck, Austria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

Part A: Completed study drug treatment in parent study 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

Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A

Key Exclusion Criteria:

History of study drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

ELX/TEZ/IVA

Drug

Fixed-dose combination (FDC) tablets for oral administration.

Other names: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

IVA

Drug

Tablets for oral administration.

Other names: VX-770, ivacaftor

Primary outcomes

  1. Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 196

Secondary outcomes

  1. Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)

    Time frame: From Baseline up to Week 96

  2. Part A: Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline up to Week 96

  3. Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    Time frame: From Baseline up to Week 96

  4. Part A: Absolute Change in Body Mass Index (BMI)

    Time frame: From Baseline up to Week 96

  5. Part A: Absolute Change in Weight

    Time frame: From Baseline up to Week 96

  6. Part A: Number of Pulmonary Exacerbations (PEx)

    Time frame: From Baseline up to Week 96

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

Important dates

Study start
2022
Primary completion
2027
Study completion
2027
First posted
Apr 15, 2022
Registry last updated
Jul 21, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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