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Completed

NCT Number: NCT04183790

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older

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

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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

Queensland Children's Hospital, South Brisbane, Australia

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Who can participate

Healthy volunteers accepted: No

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

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.

Treatment and study plan

ELX/TEZ/IVA

Drug

Fixed-dose combination (FDC) tablet for oral administration

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

IVA

Drug

Mono tablet for oral administration.

Other names: VX-770, ivacaftor

Primary outcomes

  1. Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: From Baseline up to Week 196

Secondary outcomes

  1. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    Time frame: From Baseline up to Week 192

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  2. Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline up to Week 192

    Sweat samples were collected using an approved collection device.

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

    Time frame: From Baseline up to Week 192

    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  4. Absolute Change in Body Mass Index (BMI)

    Time frame: From Baseline up to Week 192

    BMI was defined as weight in kg divided by squared height in meters (m^2).

  5. Absolute Change in BMI-for-age Z-score

    Time frame: From Baseline up to Week 192

    BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

  6. Number of Participants With Pulmonary Exacerbations (PEx) for 106/107

    Time frame: From Baseline up to Week 192

    Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.

  7. Number of CF-related Hospitalizations for 106/107

    Time frame: From Baseline up to Week 192

    The total number of CF related hospitalization (Planned + Unplanned) events across all participants were reported.

  8. Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)

    Time frame: From Baseline up to Week 192

    The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.

  9. Absolute Change in Weight

    Time frame: From Baseline up to Week 192

  10. Absolute Change in Weight-for-age Z-score

    Time frame: From Baseline up to Week 192

    The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

  11. Absolute Change in Height

    Time frame: From Baseline up to Week 192

  12. Absolute Change in Height-for-age Z-score

    Time frame: From Baseline up to Week 192

    The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.

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 ELX/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

Important dates

Study start
2020
Primary completion
2024
Study completion
2024
First posted
Dec 3, 2019
Registry last updated
May 18, 2025

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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