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

Cystic Fibrosis in Children Clinical Trial

— ROOTS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04138589
• Other study ID #: 201900032
• Status: Completed
• Start date: November 1, 2017
• Est. completion date: July 19, 2022

Study information

• Verified date: November 2023
• Source: University Medical Center Groningen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 .

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: July 19, 2022
• Est. primary completion date: July 19, 2022
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Cystic Fibrosis in Children
• Fibrosis

Locations

Country	Name	City	State
Germany	Children's Hospital Marien Hospital Wesel	Düsseldorf
Netherlands	Beatrix Children's Hospital, University Medical Center Groningen	Groningen

Sponsors (3)

Lead Sponsor	Collaborator
University Medical Center Groningen	Marien Hospital Wesel, Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Germany,  Netherlands, 

Outcome

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