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

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

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.

Study Overview

• Status: Unknown
• Conditions: Cystic Fibrosis in Children
• Intervention / Treatment: Device: MyCyFAPP

• Study Type: Interventional
• Enrollment (Anticipated): 200
• Phase: Not Applicable

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: use of MyCyFAPP; use of MyCyFAPP during 6 months	Device: MyCyFAPP; use of the MycyFAPP with all its features during 6 months

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Universitaire Ziekenhuizen KU Leuven
• Collaborators: European Commission; Hospital Universitario La Fe

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): October 1, 2017
• Primary Completion (ANTICIPATED): December 1, 2018
• Study Completion (ANTICIPATED): December 1, 2018

Study Registration Dates

• First Submitted: September 20, 2017
• First Submitted That Met QC Criteria: September 20, 2017
• First Posted (ACTUAL): September 25, 2017

Study Record Updates

• Last Update Posted (ACTUAL): September 25, 2017
• Last Update Submitted That Met QC Criteria: September 20, 2017
• Last Verified: September 1, 2017

More Information

Terms related to this study

• Keywords: mobile application; PERT
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: s60787

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No