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

March 6, 2024 updated by: Danone Nutricia SpA Società Benefit

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study 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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients requiring KD
Pediatric patients with any disease which require a ketogenic diet (KD), i.e. metabolic or genetic disorder or neurological (congenital and acquired) diseases
Other: ketogenic diet (as per clinical practice)
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
6 months,(12 months optional)
% 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
6 months,(12 months optional)
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
6 months,(12 months optional)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Danone Nutricia SpA Società Benefit

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 1, 2022

Primary Completion (Estimated)

May 31, 2025

Study Completion (Estimated)

May 31, 2026

Study Registration Dates

First Submitted

February 29, 2024

First Submitted That Met QC Criteria

March 6, 2024

First Posted (Actual)

March 13, 2024

Study Record Updates

Last Update Posted (Actual)

March 13, 2024

Last Update Submitted That Met QC Criteria

March 6, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KetoSTrENgTH

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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