Clinical trial NCT03292718: Clinical Trial to Assess Influence of MyCyFAPP Use on GI Related QOL in Children With Cystic Fibrosis
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Cystic Fibrosis in Children
Keywords: PERT
mobile application
Lead sponsor: Universitaire Ziekenhuizen KU Leuven
Sponsor class: OTHER
Collaborators: name: Hospital Universitario La Fe
name: European Commission
First posted: 2017-09-25
Enrollment: 200
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single group, open label, with intervention
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. 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:

   1. A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
   2. A documented genotype with two disease-causing mutations in the CFTR gene
2. Having pancreatic insufficiency (stool elastase \< 200 mcg/g stool) and using PERT
3. Age ≥ 24 months and \< 18 years at screening visit
4. 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:

1. Acute infection associated with decreased appetite or fever at time of run-in visit
2. Acute abdominal pain necessitating an intervention at time of run-in visit
3. Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator
4. Investigational drug use within 30 days prior to run-in visit
5. Started with CFTR modulator treatment less than 3 months before start of run-in visit
6. Inability to use the APP due to patient specific factors such as language or learning difficulties

Primary outcomes:
measure: change in Modified PedsQL GI
timeFrame: 3 months
Secondary outcomes:
measure: change in CFQ-R
timeFrame: 3 months and 6 months
measure: change in VAS
timeFrame: 3 months and 6 months
measure: change in Modified PedsQL GI
timeFrame: 6 months
measure: change in lung function
timeFrame: 3 and 6 months
Other outcomes:
Locations:
Study officials:
References: