cystic fibrosis pancreas insufficieny
Conditions
Interventions
The study intervention consists of use of the MyCyFAPP with its different
features during 6 months.
Sponsors
Erasmus MC, Universitair Medisch Centrum Rotterdam
Eligibility
Age
2 Years to 17 Years
Inclusion 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: a) A documented sweat chloride * 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT) b) A documented genotype with two disease-causing mutations in the CFTR gene 2. Having pancreatic insufficiency (stool elastase
Exclusion criteria
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 APP due to patient specific factors such as language or educational issues
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome will be change of modified PedsQL GI questionnaire after 3 months (M3) of APP use. PedsQL GI is the Pediatric Quality of Life score, Gastrointestinal domain Symptoms Module. The questionnaire was validated in childeren with CF in the first phase of the study. Age appropriate questionnaires will be used. | — |
Secondary
| Measure | Time frame |
|---|---|
| A. Impact assessment of APP on health parameters: * Change in modified PedsQL GI score from baseline (M0) to M6 visit * Change in health-related quality of life as measured by the CFQ-R (age appropriate version) at M3 and M6 compared to baseline (M0) * Change in health-related quality of life as measured by the VAS at M3 and M6 compared to baseline (M0) * Change in FEV1, FVC, FEV1/FVC and FEF25-75 z-scores (lung function) at M3 and M6 compared to baseline. * Subgroup analysis in patients with low baseline BMI z-scores ( | — |
Countries
Netherlands
Outcome results
None listed