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

Cystic Fibrosis Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03625466
• Other study ID #: VX16-809-121
• Secondary ID: 2017-003761-99
• Status: Completed
• Phase: Phase 2
• Start date: August 10, 2018
• Est. completion date: October 7, 2021

Study information

• Verified date: October 2022
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 51
• Est. completion date: October 7, 2021
• Est. primary completion date: October 9, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 5 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Disease Progression
• Fibrosis

Intervention

Drug:
• LUM/IVA
FDC tablets or granules for oral administration.
• LUM/IVA
FDC granules for oral administration.
• Placebo
Placebo matched to LUM/IVA for oral administration.

Locations

Country	Name	City	State
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin	Giessen
Germany	Hannover Medical School	Hannover
Germany	Heidelberg Cystic Fibrosis Center	Heidelberg
Germany	Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin	Lubeck

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	From Baseline at Week 48
Secondary	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.	From Baseline Through Week 48
Secondary	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.	From Baseline at Week 48
Secondary	Part 1: Absolute Change in Stature-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.	From Baseline at Week 48
Secondary	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.	From Baseline at Week 48