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Completed

NCT Number: NCT03625466

A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).

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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

2 year–5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Charite Paediatric Pulmonology Department, Berlin, Germany

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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:

  • Subjects with confirmed diagnosis of CF.
  • Homozygous for F508del (F/F).
  • Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit.

Key Exclusion Criteria:

  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
  • Solid organ or hematological transplantation.
  • History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study.

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

LUM/IVA

Drug

FDC tablets or granules for oral administration.

Other names: Lumacaftor/Ivacaftor, VX-809/VX-770

Placebo

Drug

Placebo matched to LUM/IVA for oral administration.

Primary outcomes

  1. Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48

    Time frame: From Baseline at Week 48

    MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = <50% of lobe involved and 2 = >=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.

Secondary outcomes

  1. Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48

    Time frame: From Baseline Through Week 48

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

  2. Part 1: Absolute Change in Weight-for-age Z-score at Week 48

    Time frame: From Baseline at Week 48

    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.

  3. Part 1: Absolute Change in Stature-for-age Z-score at Week 48

    Time frame: From Baseline at Week 48

    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.

  4. Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48

    Time frame: From Baseline at Week 48

    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.

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

An Exploratory Phase 2, 2-part, Randomized, Double-blind, Placebo-controlled Study With a Long-term, Open-label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

Important dates

Study start
2018
Primary completion
2020
Study completion
2021
First posted
Aug 10, 2018
Registry last updated
Nov 2, 2022

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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