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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04138589
Other study ID # 201900032
Secondary ID
Status Completed
Phase
First received
Last updated
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
Health authority
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
Accepts healthy volunteers
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

Study Design


Related Conditions & MeSH terms


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
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