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NCT Number: NCT06309251

Effectiveness and Impact on the Quality of Life of Ketogenic Diet in Pediatric Patients

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

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Key information

Conditions

Drug Resistant Epilepsy Autism Spectrum Disorder Brain Diseases Brain Neoplasms Brain Tumor, Pediatric Central Nervous System Diseases Central Nervous System Neoplasms Child Development Disorders, Pervasive Chronic Migraine Epilepsy Mental Disorders Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodevelopmental Disorders

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

ASST Fatebenefratelli Sacco (PO "V. Buzzi"), Milan, MI, Italy

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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

Treatment and study plan

ketogenic diet (as per clinical practice)

Other

Patients 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.

Primary outcomes

  1. Nutritional adequacy

    Time frame: 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 outcomes

  1. Nutritional adequacy

    Time frame: 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)

  2. % of responders to KD

    Time frame: 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

  3. Health related quality of life improvement through parent questionnaire

    Time frame: 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

Study contacts

Contact information is provided by the study sponsor or research team.

Antonella Diamanti, MD

CONTACT

[email protected]

+39 06 6859 2189

Sponsors and collaborators

Lead sponsor

Danone Nutricia SpA Società Benefit

Industry

Registry information

Official study title

Clinical and Nutritional Effectiveness and Impact on the Quality of Life of the Ketogenic Diet in Pediatric Patients With Neurological, Genetic or Metabolic Disorders: a Multicenter Prospective Observational Study

Important dates

Study start
2022
Primary completion
2025
Study completion
2026
First posted
Mar 13, 2024
Registry last updated
Sep 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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