Status:

COMPLETED

Effect of Lumacaftor/Ivacaftor in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function

Lead Sponsor:

University Medical Center Groningen

Collaborating Sponsors:

Vertex Pharmaceuticals Incorporated

Marien Hospital Wesel

Conditions:

Cystic Fibrosis in Children

Eligibility:

All Genders

6-18 years

Brief Summary

To obtain prospective real world data of the effect of lumacaftor/ivacaftor or tezacaftor/ ivacaftor on small airway disease in children aged 6-18 years with cystic fibrosis (CF) homozygous for F508de...

Detailed Description

Multi-center observational study. Duration 12 months after the start of lumacaftor/ ivacaftor or tezacaftor/ ivacaftor . To collect these data and to assist in clinical decisions regarding initiation...

Eligibility Criteria

Inclusion

  • Children aged 6-18 years
  • CF, Homozygote F508del confirmed by DNA analysis
  • Considered for start of lumacaftor/ ivacaftor or tezacaftor/ivacaftor

Exclusion

  • \- Unable to perform acceptable, repeatable lung function tests

Key Trial Info

Start Date :

November 1 2017

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

July 19 2022

Estimated Enrollment :

30 Patients enrolled

Trial Details

Trial ID

NCT04138589

Start Date

November 1 2017

End Date

July 19 2022

Last Update

November 29 2023

Active Locations (2)

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Page 1 of 1 (2 locations)

1

Children's Hospital Marien Hospital Wesel

Düsseldorf, Germany

2

Beatrix Children's Hospital, University Medical Center Groningen

Groningen, Netherlands