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

Vertex Pharmaceuticals Incorporated51 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Cystic Fibrosis

Interventions

LUM/IVADRUG

FDC tablets or granules for oral administration.

LUM/IVADRUG

FDC granules for oral administration.

PlaceboDRUG

Placebo matched to LUM/IVA for oral administration.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 5 Years

Medical Language ↔ Plain English

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.

Locations (5)

Charite Paediatric Pulmonology Department

Berlin, Germany

Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin

Giessen, Germany

Hannover Medical School

Hanover, Germany

Heidelberg Cystic Fibrosis Center

Heidelberg, Germany

Outcomes

Primary Outcomes

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

Time frame: From Baseline at Week 48

Secondary Outcomes

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

Time frame: From Baseline Through Week 48

Part 1: Absolute Change in Weight-for-age Z-score 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.

Time frame: From Baseline at Week 48

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

Time frame: From Baseline at Week 48

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

Data from ClinicalTrials.gov

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