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

NCT03292718 | Unknown

• 1 other identifier: s60787
• Study Type: interventional
• Target: 200
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Cystic Fibrosis in Children

Keywords

PERT; mobile application

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• change in CFQ-R

  3 months and 6 months

• change in VAS

  3 months and 6 months

• change in Modified PedsQL GI

  6 months

• change in lung function

  3 and 6 months

Study Arms (1)

use of MyCyFAPP

EXPERIMENTAL

use of MyCyFAPP during 6 months

Device: MyCyFAPP

Interventions

MyCyFAPP DEVICE

use of the MycyFAPP with all its features during 6 months

use of MyCyFAPP

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Universitaire Ziekenhuizen KU Leuven: lead
• Hospital Universitario La Fe: collaborator
• European Commission: collaborator

Related Links

• Related Info

Central Study Contacts

Mieke Boon, MD PhD

CONTACT

+3216343820; mieke.boon@uzleuven.be

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Mieke Boon

Model Details: Single group, open label, with intervention

Study Record Dates

• First Submitted: September 20, 2017
• First Posted: September 25, 2017
• Study Start: October 1, 2017
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: September 25, 2017

Record last verified: 2017-09