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Completed

NCT Number: NCT03537651

A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation

This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.

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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

Perth Children's Hospital, Nedlands, Australia

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115
  • Eligible CFTR Mutation

Exclusion criteria

  • Pregnant and nursing females
  • History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator
  • Ongoing participation in another study with investigational drug

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

TEZ/IVA

Drug

Fixed-dose combination tablet for oral administration.

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

IVA

Drug

Tablet for oral administration.

Other names: VX-770, ivacaftor

Primary outcomes

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

    Time frame: Day 1 up to Week 100

Secondary outcomes

  1. Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)

    Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

    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.

  2. Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS

    Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

    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.

  3. Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group)

    Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

    Sweat samples were collected using an approved collection device.

  4. Part A: Absolute Change in SwCl for 113B/116 FAS

    Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

    Sweat samples were collected using an approved collection device.

  5. Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)

    Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

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

  6. Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS

    Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

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

  7. Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)

    Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

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

  8. Part A: Absolute Change in BMI for 113B/116 FAS

    Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

    BMI was defined as weight in kg divided by m^2.

  9. Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 192

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation

Important dates

Study start
2018
Primary completion
2020
Study completion
2023
First posted
May 25, 2018
Registry last updated
Apr 19, 2024

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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