Effectiveness and Impact on the Quality of Life of Ketogenic Diet in Pediatric Patients
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 18
SponsorDanone Nutricia SpA Società Benefit
About this trial
The goal of this observational study is to learn about the clinical and nutritional effectiveness of ketogenic diet (KD) in pediatric patients with genetic, neurological or metabolic conditions requiring KD.
The main question\[s\] it aims to answer are:
* does KD support adequate growth? * does KD improve clinical symptoms? * how does KD impact quality of life? Participants will be followed up as per clinical practice
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Pediatric patients (aged < 18 years) with drug-resistant epilepsy (fail to achieve (and maintain) seizure freedom with adequate trials of two or more antiseizure medications) or genetic, metabolic, neurological (congenital and acquired) diseases treated with ketogenic diet
Pediatric patients (aged < 18 years) with metabolic, genetic or neurological (congenital and acquired) diseases (not necessarily associated with drug-resistant epilepsy) treated with ketogenic diet; this includes the new KD indications or the administration of KD in the ICU for status epilepticus.
Disqualifiers
Patients affected by beta-oxidation cycle disorders, systemic primary carnitine deficiency, primary dyslipidemia, pyruvate carboxylase deficiency, porphyria, mitochondrial disease, defects in ketone body metabolism (ketogenesis or ketolysis), defect in gluconeogenesis.
Children with type 1 diabetes
Parents (or caregivers) unable to guarantee adherence to the
Trial population
Pediatri patients (0-18 years) affected by any disease which require a ketogenic diet (KD), i.e. metabolic or genetic disorder or neurological (congenital and acquired) diseases
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
ketogenic diet (as per clinical practice)
Other interventionPatients recruited receive a ketogenic diet. The prescription, the type of KD protocol and the route of administration will depend, essentially, on the patient's ability and willingness to feed spontaneously and independently, as well as on compliance with the therapies set and the severity of the clinical conditions.
Treatment groups
100 Participants
are divided into 1 treatment groupGroup A: Patients requiring KD1 intervention
Trial outcomes
Primary outcomes
1
Nutritional adequacy
evaluate the nutritional adequacy of the diet after 6 months of treatment with ketogenic diet (KD) in terms of adequate growth defined by weight-for-age Z Score (WAZ) and length-for-age Z score (LAZ).
Time frame
6 monthsSecondary outcomes
1
Nutritional adequacy
Evaluate the nutritional adequacy of the diet after 6 and 12 months of treatment with ketogenic diet (KD) in terms of correct growth defined by: Weight-for-age Z-score (WAZ), length/height-for-age z-score (LAZ), BMI z-score Blood levels of selected parametres (eg cholesterol, HDL, LDL) within normal ranges Body composition (optional)
Time frame
6 months,(12 months optional)2
% of responders to KD
Evaluate the effectiveness of the ketogenic diet (KD) on the clinical outcomes (% of responders): * Epilepsy: at least 50% seizure reduction after 6 months of KD treatment (vs 6 months before trial start( (daily diary records) * ASD: reduction of at least 2-8 in CARS score after 6 months of KD treatment * Chronic migraine: at least 50% episodes reduction after 6 months of KD treatment (vs 6 months before trial start( (daily diary records); reduction \>=50% of PedMiDas score after 6 months of KD treatment (vs baseline) Brain tumors: % survival; % of subjects with progression free survival
Time frame
6 months,(12 months optional)3
Health related quality of life improvement through parent questionnaire
Evaluate the effectiveness of the ketogenic diet (KD) on the patient's quality of life using a non-validated questionnaire administered to parents with qualitative open and closed questions. Improvement will be considered as an improvement in the score of disease burden related questions baseline vs end of observation
Time frame
6 months,(12 months optional)Sponsors and contacts
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Source ClinicalTrials.gov