Cystic Fibrosis in ChildrenMyCyFAPPNCT03292718
Clinical Trial to Assess Influence of MyCyFAPP Use on GI Related QOL in Children With Cystic Fibrosis
A study testing MyCyFAPP in people with Cystic Fibrosis in Children, run by Universitaire Ziekenhuizen KU Leuven.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Cystic Fibrosis in Children
- What is being tested?
- MyCyFAPP
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- No sites listed yet
- Study dates
- Oct 2017to Dec 2018
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet.
About this study
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
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:
Read more
- 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
- Being tested
- MyCyFAPP
- Keywords
- PERT, mobile application
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MyCyFAPPDevice
use of the MycyFAPP with all its features during 6 months
| Study group | Role | What they receive |
|---|---|---|
| use of MyCyFAPP | Experimental | use of MyCyFAPP during 6 months Receives: Device: MyCyFAPP |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 3 months
Secondary outcomes (4)
change in CFQ-R
CFQ-R will be assessed by questionnaires
Measured: 3 months and 6 months
change in VAS
VAS will be assessed by questionnaires
Measured: 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
Measured: 6 months
change in lung function
spirometry will be performed
Measured: 3 and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2017
- Study startOct 2017 (estimated)
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis in ChildrenStudies of the same condition, open and under way.
- More trials run by Universitaire Ziekenhuizen KU LeuvenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single group, open label, with intervention
- Masking
- None (open label)
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universitaire Ziekenhuizen KU Leuven
- Sponsor type
- University, hospital or other
- Collaborators
- Hospital Universitario La Fe, European Commission
- Responsible party
- Principal investigator, Mieke Boon, MD PhD, Universitaire Ziekenhuizen KU Leuven
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03292718
- Sponsor's protocol ID
- s60787
- Acronym
- MyCyFAPP
- Status last verified
- Sep 2017
- First submitted
- 20 Sep 2017
- Last updated
- 25 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03292718
- Acronym / sponsor ID
- MyCyFAPP / s60787
- Conditions
- Cystic Fibrosis in Children
- Keywords
- PERT, mobile application
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Universitaire Ziekenhuizen KU LeuvenUniversity, hospital or other
- Collaborators
- Hospital Universitario La Fe, European Commission
- Study start
- Oct 2017 (estimated)
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 25 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single group, open label, with intervention
| Arm | Type | Receives |
|---|---|---|
| use of MyCyFAPP | Experimental | Device: MyCyFAPPuse of MyCyFAPP during 6 months |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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:
- 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.
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MyCyFAPPuse of the MycyFAPP with all its features during 6 months | Device | use of MyCyFAPP |
Primary outcome
change in Modified PedsQL GI
Time frame: 3 months
Modified PedsQL GI will be assessed at month 0 and month 3 by applying questionnaires to children and their parents
4 secondary outcomes
change in CFQ-R
Time frame: 3 months and 6 months
change in VAS
Time frame: 3 months and 6 months
change in Modified PedsQL GI
Time frame: 6 months
change in lung function
Time frame: 3 and 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A study testing MyCyFAPP in people with Cystic Fibrosis in Children, run by Universitaire Ziekenhuizen KU Leuven.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 25 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mieke Boon, MD PhD (+3216343820, mieke.boon@uzleuven.be), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
change in Modified PedsQL GI (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2017.