Effectiveness and Impact on the Quality of Life of Ketogenic Diet in Pediatric Patients
- Conditions
- Autism Spectrum DisorderChronic MigraineBrain Tumor, PediatricDrug Resistant Epilepsy
- Interventions
- Other: ketogenic diet (as per clinical practice)
- Registration Number
- NCT06309251
- Lead Sponsor
- Danone Nutricia SpA Società Benefit
- Brief Summary
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
- Detailed Description
Scientific and clinical interest in KD is growing with a progressive increase in its therapeutic indications, ie recent studies show beneficial effects of KD in other diseases like migraine, autism spectrum disorder and brain tumors.
However, so far, the majority of results on KD effects have been collected in patients with drug-resistant epilepsy proving its effectiveness on clinical outcome (e.g. reducing epileptic seizures), frequently leaving out the nutritional assessment and the impact on quality of life. Nonetheless, these factors are indeed essential to ensure safety and efficacy of KD treatment, especially when dealing with children and in perspective of the progressive expansion of the target population that can benefit from this nutritional therapy.
The present multicenter study aims to analyse these poorly explored areas, by evaluating, in a cohort of pediatrics patient with indication to KD therapy, not only the KD effectiveness on the course of the underlying disease, but also the impact on nutritional parameters (e.g. growth, lipid profile, vitamin intake, bone mineral density, ..) and on quality of life.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- 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.
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients requiring KD ketogenic diet (as per clinical practice) Pediatric patients with any disease which require a ketogenic diet (KD), i.e. metabolic or genetic disorder or neurological (congenital and acquired) diseases
- Primary Outcome Measures
Name Time Method Nutritional adequacy 6 months 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).
- Secondary Outcome Measures
Name Time Method Nutritional adequacy 6 months,(12 months optional) 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)Health related quality of life improvement through parent questionnaire 6 months,(12 months optional) 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
% of responders to KD 6 months,(12 months optional) 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
Trial Locations
- Locations (3)
ASST Fatebenefratelli Sacco (PO "V. Buzzi")
🇮🇹Milan, MI, Italy
Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, RM, Italy
Fondazione "Istituto Neurologico Nazionale C. Mondino"
🇮🇹Pavia, PV, Italy