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

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04138589

Acronym

ROOTS

Enrollment

Registered

2019-10-24

Start date

2017-11-01

Completion date

2022-07-19

Last updated

2023-11-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis in Children

Keywords

lumacaftor/ ivacaftor, tezacaftor/ ivacaftor, Multiple breath washout, Lung clearance index, PRAGMA CF score

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 .

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

6 Years to 18 Years

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

Design outcomes

Primary

Measure	Time frame	Description
Change in lung clearance index	12 months	Change between t=0 and t=12

Secondary

Measure	Time frame	Description
Change in PRAGMA-CF score	12 months	Change between t=0 and t=12

Other

Measure	Time frame	Description
Residual volume measured by blodypethysmography	12 months	Change between t=0 and t=12
Residual volume/ total lung capacity measured by blodypethysmography	12 months	Change between t=0 and t=12
Mid upper arm circumference in SDS	12 months	Change between t=0 and t=12
percent predicted forced expiratory volume in 1 second	12 months	t= 0 and t-=12
Sweat chloride level	12 months	Change between t=0 and t=12
CF questionnaire revised respiratory domain	12 months	Change between t=0 and t=12, scale 0-100
Weight SDS	12 months	Change between t=0 and t=12
percent predicted forced vital capacity measured by spirometry	12 months	Change between t=0 and t=12

Countries

Germany, Netherlands

Outcome results