To validate and assess the impact of the use of the MyCyFAPP system for self-management in children with CF, more specifically the effect on patients* quality of life, nutritional status, gastro-intestinal symptoms, general wellbeing and economic…
ID
Source
Brief title
Condition
- Chromosomal abnormalities, gene alterations and gene variants
- Exocrine pancreas conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
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 outcome
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 (<-0.5): change
in H, W, BMI z-score at M3 and M6 compared to baseline
* Change in Lipase units used per day and per meal at M3 and M6 compared to M0
* Extra health care visits compared to baseline as a surrogate for economic
savings
* Change in food pattern (% of energy of proteins/carbohydrates/fat, % intake
of fruit and vegetables) at M6 compared to M0. Data will be
available from the food record in the APP
* Change in malabsorption of fat between M0 and M1, based on results of stool
collections in a subgroup of patients (substudy).
Change in abdominal symptoms as measured by the CF Abd-Score at M3 and
M6 compared to baseline (M0)
B. Exploratory outcomes considering the user acceptance of the app and the game
APP.
Background summary
Nutritional status has an enormous impact on the progression of the pulmonary
disease in CF, and particular attention has to be devoted to the occurrence of
potential imbalance between energy losses (due to pancreatic insufficiency),
increased energy needs (due to pulmonary disease) and actual energy intake.
Malnutrition and stunting can only be avoided by accurate pancreatic enzyme
replacement therapy (PERT) and close nutritional follow up and support.
Despite the use of high doses of PERT, many children still have abdominal
pain, diarrhea and other gastrointestinal problems and growth may be
suboptimal. The developed APP contains a prediction model for optimal PERT
dosing for each meal, taking into account the individual needs of the patient.
This formula is based on the results of the previous laboratory studies in
which the optimal dose of PERT was assessed per food item.
Study objective
To validate and assess the impact of the use of the MyCyFAPP system for
self-management in children with CF, more specifically the effect on patients*
quality of life, nutritional status, gastro-intestinal symptoms, general
wellbeing and economic save. The validation is a crucial stage before
implementing the APP in clinical practice.
Objective substudy: evaluation of the developed prediction model for PERT
dosing in clinical practice with use of free diet.
Study design
An open label prospective European multicentre interventional study among
paediatric CF patients in six reference paediatric CF units of the European
Union: Valencia (Hospital Universitari i Politècnic La Fe Valencia* Spain),
Madrid (Hospital Universitario Ramón y Cajal Madrid* Spain), Rotterdam (Erasmus
University Medical Center * The Netherlands), Milan (Università degli Studi di
Milano, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico * Italy),
Lisbon (Associação para a Investigação e Desenvolvimento da Faculdade de
Medicina Lisboa * Portugal) and Leuven (University Hospital Leuven * Belgium).
After a screening visit and obtaining informed consent, 3 study visits will
take place (M0, M3 en M6) in a period of 6 months (mostly coinciding with
regular outpatient visits) during which the modified 4 questionanires will be
obtained and also optional questionnaires about user acceptance of the APP.
Height, weight, BMI and lung function will be obtained during the study visits
and the nutritional intake will be evaluated (via APP). The intervention (use
of the APP on smartphone and/or tablet) will last 6 months. The link of the
self-management APP and the professional webtool will allow for online
interaction between clinical teams and patients.
Substudy: when participating in the substudy, fat absorption will be determined
at M0 and M1 by assessing amount of fat in collected stools in comparison with
intake (via nutritional diary in APP).
Intervention
The study intervention consists of use of the MyCyFAPP with its different
features during 6 months.
Study burden and risks
The burden of the study for participating patients consists of the use of the
app by a smartphone or tablet during 6 months with 3 visits, which will co
inside with he regular poli clinic visits. Extra time needed for the study
visit will be 40 minutes for filling in 4 questionnaires (PedsQL-GI, CFQr, CF
Abd Score and VAS) and optional, 3x a questionnaire about user acceptance of
the app. Patients/parents need to follow a training on the use of the app and
tablet. During the 6 months patients will be asked to use the app and the
suggested amount of creon daily for one month, the other 5 months at least for
2x a week and fill in a food diary 2x during 3 days at the beginning and the
end of the study.If patient takes part of the sub study ( which is optional)
they will be asked to collect feces 2x during 48hrs.
Participating in this study can increase knowledge on the optimal use of creon
which will lead to less abdominal pain or diarrhea and in the long term a
positive effect on growth and quality of life. It will also increase knowledge
on CF and healthy food. disadvantage of participating is the time it will take
and the advised amount of creon could lead to more abdominal pain.
Wytemaweg 80
Rotterdam 3015 CN
NL
Wytemaweg 80
Rotterdam 3015 CN
NL
Listed location countries
Age
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 < 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
5. Inclusion visit coincides with scheduled routine clinic visit
6. Ability and willingness to comply with APP use and evaluations at time of routine clinic visits as judged by the site investigator
7. 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 APP due to patient specific factors such as language or educational issues
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | clinical trials.gov, nummer volgt |
| CCMO | NL63421.078.17 |