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Completed

NCT Number: NCT04058366

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).

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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

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Prince Charles Hospital, Chermside, 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 (VX18-445-104); 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

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: From Baseline up to Week 100

Secondary outcomes

  1. Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    Time frame: From Baseline at Week 96

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

  2. Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)

    Time frame: From Baseline at Week 96

    Sweat samples were collected using an approved collection device.

  3. Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI)

    Time frame: From Baseline at Week 96

    BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).

  4. Part A: Absolute Change From Parent Study Baseline in BMI Z-score

    Time frame: From Baseline at Week 96

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

  5. Part A: Absolute Change From Parent Study Baseline in Body Weight

    Time frame: From Baseline at Week 96

  6. Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

    Time frame: From Baseline at Week 96

    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.

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 VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Important dates

Study start
2019
Primary completion
2022
Study completion
2022
First posted
Aug 15, 2019
Registry last updated
Jan 16, 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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