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Completed

NCT Number: NCT04138589

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

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 F508del. The effect of the medication on small airway disease is evaluated by measurement of multiple breath washout (MBW) with its outcome parameter lung clearance index (LCI) and the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF) cpmputed tomography (CT) score. In addition the relation between changes in LCI and PRAGMA-CF score is evaluated.

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Key information

Conditions

Cystic Fibrosis in Children

Age range

6 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's Hospital Marien Hospital Wesel, Düsseldorf, Germany

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About this study

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 and continuation of lumacaftor/ivacaftor or tezacaftor/ ivacaftor, the investigators of the CF center (Beatrix Children's Hospital, University Medical Center Groningen (UMCG), the Netherlands) developed an extensive protocol of testing before children aged 6-18 years start therapy and during the first year after start.

The protocol includes the following tests: growth parameters, sweat test, lung function testing (spirometry, MBW, body plethysmography), blood test panel (AST, ALT, alkalic phosphatase (AF), total and direct bilirubin, LDH), fecal elastase, high resolution (HR)CT and CF quality of life questionnaires. These tests are repeated at regular intervals.

Multi-center observational study. Duration 12 months after the start of lumacaftor/ ivacaftor or tezacaftor/ ivacaftor .

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • - Unable to perform acceptable, repeatable lung function tests

Treatment and study plan

Primary outcomes

  1. Change in lung clearance index

    Time frame: 12 months

    Change between t=0 and t=12

Secondary outcomes

  1. Change in PRAGMA-CF score

    Time frame: 12 months

    Change between t=0 and t=12

Other outcomes

  1. percent predicted forced expiratory volume in 1 second

    Time frame: 12 months

    t= 0 and t-=12

  2. percent predicted forced vital capacity measured by spirometry

    Time frame: 12 months

    Change between t=0 and t=12

  3. Residual volume measured by blodypethysmography

    Time frame: 12 months

    Change between t=0 and t=12

  4. Residual volume/ total lung capacity measured by blodypethysmography

    Time frame: 12 months

    Change between t=0 and t=12

  5. Mid upper arm circumference in SDS

    Time frame: 12 months

    Change between t=0 and t=12

  6. Weight SDS

    Time frame: 12 months

    Change between t=0 and t=12

  7. Sweat chloride level

    Time frame: 12 months

    Change between t=0 and t=12

  8. CF questionnaire revised respiratory domain

    Time frame: 12 months

    Change between t=0 and t=12, scale 0-100

Sponsors and collaborators

Lead sponsor

University Medical Center Groningen

Other

Collaborators

  • Marien Hospital Wesel
  • Vertex Pharmaceuticals Incorporated

Registry information

Official study title

Real World Data of the Effect of Lumacaftor/Ivacaftor Therapy in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function

Acronym: ROOTS

Important dates

Study start
2017
Primary completion
2022
Study completion
2022
First posted
Oct 24, 2019
Registry last updated
Nov 29, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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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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