NCT04138589

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Nov 2017

Longer than P75 for all trials

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2017

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

September 18, 2019

Completed
1 month until next milestone

First Posted

Study publicly available on registry

October 24, 2019

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 19, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 19, 2022

Completed
Last Updated

November 29, 2023

Status Verified

November 1, 2023

Enrollment Period

4.7 years

First QC Date

September 18, 2019

Last Update Submit

November 28, 2023

Conditions

Cystic Fibrosis in Children

Keywords

lumacaftor/ ivacaftortezacaftor/ ivacaftorMultiple breath washoutLung clearance indexPRAGMA CF score

Outcome Measures

Primary Outcomes (1)

  • Change in lung clearance index

    Change between t=0 and t=12

    12 months

Secondary Outcomes (1)

  • Change in PRAGMA-CF score

    12 months

Other Outcomes (8)

  • percent predicted forced expiratory volume in 1 second

    12 months

  • percent predicted forced vital capacity measured by spirometry

    12 months

  • Residual volume measured by blodypethysmography

    12 months

  • +5 more other outcomes

Study Arms (1)

CF patients aged 6-18 years homozygeous for delta F508

CF patients aged 6-18 years homozygeous for delta F508 starting with lumacaftor/ ivacaftor or tezacaftor/ ivacaftor

Eligibility Criteria

Age6 Years - 18 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

Patients are included from the Beatrix Children's Hospital, UMCG, the Netherlands and the Marien Hospital Wesel, Germany

You may qualify if:

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

You may not qualify if:

  • \- Unable to perform acceptable, repeatable lung function tests

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Children's Hospital Marien Hospital Wesel

Düsseldorf, Germany

Location

Beatrix Children's Hospital, University Medical Center Groningen

Groningen, Netherlands

Location

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

September 18, 2019

First Posted

October 24, 2019

Study Start

November 1, 2017

Primary Completion

July 19, 2022

Study Completion

July 19, 2022

Last Updated

November 29, 2023

Record last verified: 2023-11

Data Sharing

IPD Sharing
Will not share

No plans to share IPD

Locations

DE(1)NL(1)