|- candidate number||24052|
|- NTR Number||NTR5765|
|- ISRCTN||ISRCTN no longer applicable|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||29-feb-2016|
|- Secondary IDs||NL5526607816 |
|- Public Title||MyCyFAPP PERT model|
|- Scientific Title||MyCyFAPP Work Package 3: Development of the enzyme replacement predictive model|
|- ACRONYM||MyCyFAPP |
|- hypothesis||In the paediatric age, secondary malnutrition to Cystic Fibrosis (CF) has a negative impact in the patients clinical evolution for its repercussion, among others, on the digestive and absorptive functions and the appetite. Maintaining an
adequate nutritional status is an indispensable aspect of the CF treatment, since it directly affects the quality of life, the lung function and the survival. Pancreatic insufficient patients require supplementation with exogenous pancreatic enzyme therapy (PERT), with the aim to reduce the fecal losses of fat, protein and biliary acids and the deficit of fatsoluble
vitamins associated to this disease. It is crucial that the administration of pancreatic extracts is in the correct dose and, moreover, adapted to each moment and each meal.|
The main objective in the first approach is to obtain a mathematical predictive model
(MPM) adapted to each patients' gastrointestinal conditions that calculates the optimal amount of enzymatic supplements for the optimal fat digestion of any food or meal. So for each patient an individual correction factor (ICF)
will be obtained. Afterwards, in a second step, the objective is to validate the MPM by assessing how much the final fat in stools after applying the ICF deviates from the optimal value (<6g of fat/24h).
The MPM would be eventually implemented into a mobile APP, and together with an enzyme requirements database for different food products and meals digestion, will be able to, through a calculation algorithm, predict the amount of PERD required for a specific meal, and taking into account individual needs for correction of the dose. This will allow for
self-management of this essential therapy regardless of time and place.
|- Healt Condition(s) or Problem(s) studied||Cystic Fibrosis (CF)|
|- 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. Informed consent by parent or legal guardian; assent for children from age 12 years on
3. Having pancreatic insufficiency (stool elastase < 200 mcg/g stool) and using PERT
4. Age °› 12 months and < 18 years at Screening visit
5. Stable clinical status at least two weeks before signing the informed consent.
6. Patients°Į capacity and willingness to fulfill the meal test and the faeces collection during the weekend
|- 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. Severe cholestasis (direct bilirubin increase above 2 mg/dL with respect to the normal limit for age).
4. FEV1 <40% for age, gender, weight and height.
5. Severe hypoalbuminemia (albumin in blood <2.5g/mL).
6. Hospitalisation or intravenous antibiotics <2 weeks before signing the informed consent.
7. Changes in the usual treatment (prokinetics, antiacids, H2 blockers and antibiotics) < 2 weeks before signing the
8. Presence of alterations that, according to the investigator consideration, could jeopardise the safety of the patient.
9. Hypersensibility or adverse reactions to the enzymatic supplements.
|- mec approval received||no|
|- multicenter trial||yes|
|- Type||Single arm|
|- planned startdate ||13-mrt-2016|
|- planned closingdate||31-jul-2017|
|- Target number of participants||12|
|- Interventions||The interventions consist of following a test diet, the use of PERT (own capsules, test dose), the collection of feces that is marked by the intake of color capsules and keeping a nutritional diary. These interventions take place during 3 x 2 weekends.|
|- Primary outcome||The main study parameter is to assess, how much the final fat in stools deviates from the normal concentration (6g/24h) after applying the individual correction factor (ICF).|
|- Secondary outcome||NA|
|- Timepoints||The study consists of 3 parts of each 2 x 1 day.
- Follow a test diet with a fixed PERT dose (theoretical dose).
- Collection of feces that is marked by intake of color capsules.
- Keeping a nutritional diary.
- same as part 1 except the use of a PERT dosage that takes the individual correction factor (as obtained by part 1)
- same as part 2, so with a personal PERT dosage, but follow of a free diet
The 3 parts will take place during a period of 9 months.
|- Trial web site|
|- CONTACT FOR PUBLIC QUERIES||MD, PhD JM Hulst|
|- CONTACT for SCIENTIFIC QUERIES||MD, PhD JM Hulst|
|- Sponsor/Initiator ||Erasmus Medical Center, Sophia Children's Hospital|
(Source(s) of Monetary or Material Support)
|- Brief summary||This study is part of a large HORIZON 2020 project Innovative approach to self-management and social welfare of Cystic Fibrosis patients in Europe: development, validation and implementation of a telematics tool)|
|- Main changes (audit trail)|
|- RECORD||29-feb-2016 - 25-mei-2016|