Clinical trial NCT04138589: Effect of Lumacaftor/Ivacaftor in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function
Official title: Real World Data of the Effect of Lumacaftor/Ivacaftor Therapy in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis in Children
Keywords: lumacaftor/ ivacaftor
tezacaftor/ ivacaftor
Multiple breath washout
Lung clearance index
PRAGMA CF score
Lead sponsor: University Medical Center Groningen
Sponsor class: OTHER
Collaborators: name: Vertex Pharmaceuticals Incorporated
name: Marien Hospital Wesel
First posted: 2019-10-24
Enrollment: 30
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients are included from the Beatrix Children's Hospital, UMCG, the Netherlands and the Marien Hospital Wesel, Germany

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 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.

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 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 .

Eligibility criteria: 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

Primary outcomes:
measure: Change in lung clearance index
timeFrame: 12 months
Secondary outcomes:
measure: Change in PRAGMA-CF score
timeFrame: 12 months
Other outcomes:
measure: percent predicted forced expiratory volume in 1 second
timeFrame: 12 months
measure: percent predicted forced vital capacity measured by spirometry
timeFrame: 12 months
measure: Residual volume measured by blodypethysmography
timeFrame: 12 months
measure: Residual volume/ total lung capacity measured by blodypethysmography
timeFrame: 12 months
measure: Mid upper arm circumference in SDS
timeFrame: 12 months
measure: Weight SDS
timeFrame: 12 months
measure: Sweat chloride level
timeFrame: 12 months
measure: CF questionnaire revised respiratory domain
timeFrame: 12 months
Locations:
city: Düsseldorf
country: Germany
facility: Children's Hospital Marien Hospital Wesel
city: Groningen
country: Netherlands
facility: Beatrix Children's Hospital, University Medical Center Groningen
Study officials:
References: