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OpenTrials
Completed

NCT Number: NCT05274269

Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) 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

Medizinische Universität Innsbruck, Innsbruck, Austria

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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:

  • Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not have an exclusionary CFTR mutation
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • History of solid organ or hematological transplantation
  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

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

Tablet for oral administration.

Other names: VX-770, ivacaftor

Placebo (matched to ELX/TEZ/IVA)

Other

Placebo matched to ELX/TEZ/IVA for oral administration.

Placebo (matched to IVA)

Other

Placebo matched to IVA for oral administration.

Primary outcomes

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

    Time frame: From Baseline Through Week 24

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

Secondary outcomes

  1. Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline Through Week 24

    Sweat samples were collected using an approved collection device.

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

    Time frame: From Baseline Through Week 24

    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.

  3. Absolute Change in Body Mass Index (BMI)

    Time frame: From Baseline at Week 24

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

  4. Absolute Change in Weight

    Time frame: From Baseline at Week 24

  5. Number of Pulmonary Exacerbations (PEx)

    Time frame: From Baseline Through Week 24

    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.

  6. 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 28

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
Mar 10, 2022
Registry last updated
Aug 1, 2024

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

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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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