Clinical Trial to Assess Influence of MyCyFAPP Use on GI Related QOL in Children With Cystic Fibrosis

Study Overview
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Brief Summary
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Interventional trial to study the influence of the use of MyCyFAPP (mobile application) on the gastro-intestinal related quality of life. This mobile APP has been developed during previous workpackages of the Horizon2020 Project and contains several modules: mathematical prediction model to calculate the needed dose for pancreatic enzyme replacement therapy educational games and other educational material communication with doctor/dietician through professional webtool diary to register symptoms and data on nutrition. The app will be introduced and used during 6 months. Primary outcome parameter will be change in modified PedsQL GI after 3 months. PedsQL GI is an existing questionnaire that evaluates gastro-intestinal related quality of life in children. We validated it for use in cystic fibrosis in a previous observational study.

Official Title
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Innovative Approach for Self-management and Social Welfare of Cystic Fibrosis Patients in Europe: Development, Validation and Implementation of a Telematics Tool. WP6.2: Impact Assessment Through a European Multicentre Clinical Trial: Validation of MyCyFAPP as a Portable System for Self-management in Children With CF

Conditions
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Cystic Fibrosis in Children

Intervention / Treatment
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* Device: MyCyFAPP


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria: A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT) A documented genotype with two disease-causing mutations in the CFTR gene Having pancreatic insufficiency (stool elastase < 200 mcg/g stool) and using PERT Age ≥ 24 months and < 18 years at screening visit Informed consent by parent or legal guardian; assent for children from age 12 years on 6. Inclusion visit coincides with scheduled routine clinic visit 7. Ability and willingness to comply with APP use and evaluations at time of routine clinic visits as judged by the site investigator 8. Availability of wifi connection at home so that connection to the internet is feasible at home at least weekly. Exclusion Criteria: Acute infection associated with decreased appetite or fever at time of run-in visit Acute abdominal pain necessitating an intervention at time of run-in visit Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator Investigational drug use within 30 days prior to run-in visit Started with CFTR modulator treatment less than 3 months before start of run-in visit Inability to use the APP due to patient specific factors such as language or learning difficulties

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Single group, open label, with intervention
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: use of MyCyFAPP<br>use of MyCyFAPP during 6 months | Device: MyCyFAPP<br>* use of the MycyFAPP with all its features during 6 months<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| change in Modified PedsQL GI | Modified PedsQL GI will be assessed at month 0 and month 3 by applying questionnaires to children and their parents | 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| change in CFQ-R | CFQ-R will be assessed by questionnaires | 3 months and 6 months | 
| change in VAS | VAS will be assessed by questionnaires | 3 months and 6 months | 
| change in Modified PedsQL GI | Modified PedsQL GI will be assessed at month 0 and month 6 by applying questionnaires to children and their parents | 6 months | 
| change in lung function | spirometry will be performed | 3 and 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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mobile application, PERT