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NCT Number: NCT03559062

A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

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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–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hunter Medical Research Institute (HMRI), New Lambton Heights, 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:

  • Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
  • Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
  • Participants with a screening LCI2.5 result ≥7.5.
  • Participants who are able to swallow tablets.

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension.
  • Colonization with organisms associated with a more rapid decline in pulmonary status.
  • Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

TEZ/IVA

Drug

Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.

Other names: VX-661/VX-770, tezacaftor/ivacaftor fixed dose combination

IVA

Drug

Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.

Other names: VX-770, ivacaftor

Placebo

Drug

Placebo matched to TEZ/IVA FDC

Primary outcomes

  1. Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8

    Time frame: From baseline through Week 8

    LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

Secondary outcomes

  1. Absolute Change in Sweat Chloride At Week 8

    Time frame: From baseline at Week 8

    Sweat samples were collected using an approved collection device.

  2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8

    Time frame: From baseline through Week 8

    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. Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit

    Time frame: From first dose of study drug up to safety follow-up visit (up to Week 12)

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Important dates

Study start
2018
Primary completion
2018
Study completion
2018
First posted
Jun 15, 2018
Registry last updated
Feb 11, 2020

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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