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Drug Resistant EpilepsyNCT06309251

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

An observational study of Drug Resistant Epilepsy and Autism Spectrum Disorder, run by Danone Nutricia SpA Società Benefit at 3 sites in Italy.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages Up to 18 years
What is being tested?
ketogenic diet (as per clinical practice)
How many people
100 participants
Where
3 sitesItaly
Study dates
1 Mar 2022to 31 Dec 2026
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

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

Who may be able to join

  • 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.

Who cannot join

  • 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 locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Antonella Diamanti, MD contact

+39 06 6859 2189

antonella.diamanti@opbg.net

Italy3 sites, 3 recruiting

  • ASST Fatebenefratelli Sacco (PO "V. Buzzi")

    Milan, MI, 20154

    Sara Olivotto, MD, sara.olivotto@asst-fbf-sacco.it

    Roberto Previtali, MD, roberto.previtali@unimi.it

    Recruiting

  • Fondazione "Istituto Neurologico Nazionale C. Mondino"

    Pavia, PV, 27100

    Valentina De Giorgis, MD, valentina.degiorgis@mondino.it

    Carlo Alberto Quaranta, MD, carloalberto.quaranta01@universitadipavia.it

    Recruiting

  • Ospedale Pediatrico Bambino Gesù

    Roma, RM, 00165

    Antonella Diamanti, MD, antonella.diamanti@opbg.net

    Recruiting

Other trials in Italy

About this study

Official 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

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
Read the 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.

Being tested
ketogenic diet (as per clinical practice)
Medical subject headings
Drug Resistant Epilepsy, Autism Spectrum Disorder, Brain Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

Study groups in NCT06309251
Study groupRoleWhat they receive
Patients requiring KDPediatric patients with any disease which require a ketogenic diet (KD), i.e. metabolic or genetic disorder or neurological (congenital and acquired) diseases
Receives: Other: ketogenic diet (as per clinical practice)

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: 6 months

Secondary outcomes (3)

  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)

    Measured: 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

    Measured: 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

    Measured: 6 months,(12 months optional)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Mar 2024
  2. Study start1 Mar 2022
  3. Primary completion31 Dec 2025 (estimated)
  4. Study completion31 Dec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
100 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06309251
Sponsor's protocol ID
KetoSTrENgTH
Status last verified
Aug 2025
First submitted
29 Feb 2024
Last updated
3 Sep 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Bertoli S, Foppiani A, De Amicis R, Leone A, Mastella C, Bassano M, Giaquinto E, Baranello G, Battezzati A. Anthropometric measurement standardization for a multicenter nutrition survey in children with spinal muscular atrophy. Eur J Clin Nutr. 2019 Dec;73(12):1646-1648. doi: 10.1038/s41430-019-0392-2. Epub 2019 Jan 15.

    PubMed 30647441

  • Cole TJ, Flegal KM, Nicholls D, Jackson AA. Body mass index cut offs to define thinness in children and adolescents: international survey. BMJ. 2007 Jul 28;335(7612):194. doi: 10.1136/bmj.39238.399444.55. Epub 2007 Jun 25.

    PubMed 17591624

  • Hershey AD, Powers SW, Vockell AL, LeCates S, Kabbouche MA, Maynard MK. PedMIDAS: development of a questionnaire to assess disability of migraines in children. Neurology. 2001 Dec 11;57(11):2034-9. doi: 10.1212/wnl.57.11.2034.

    PubMed 11739822

  • Kossoff EH, Zupec-Kania BA, Auvin S, Ballaban-Gil KR, Christina Bergqvist AG, Blackford R, Buchhalter JR, Caraballo RH, Cross JH, Dahlin MG, Donner EJ, Guzel O, Jehle RS, Klepper J, Kang HC, Lambrechts DA, Liu YMC, Nathan JK, Nordli DR Jr, Pfeifer HH, Rho JM, Scheffer IE, Sharma S, Stafstrom CE, Thiele EA, Turner Z, Vaccarezza MM, van der Louw EJTM, Veggiotti P, Wheless JW, Wirrell EC; Charlie Foundation; Matthew's Friends; Practice Committee of the Child Neurology Society. Optimal clinical management of children receiving dietary therapies for epilepsy: Updated recommendations of the International Ketogenic Diet Study Group. Epilepsia Open. 2018 May 21;3(2):175-192. doi: 10.1002/epi4.12225. eCollection 2018 Jun.

    PubMed 29881797

Questions about this trial

What is this clinical trial studying?

An observational study of Drug Resistant Epilepsy and Autism Spectrum Disorder, run by Danone Nutricia SpA Società Benefit at 3 sites in Italy.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 3 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Antonella Diamanti, MD (+39 06 6859 2189, antonella.diamanti@opbg.net), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Nutritional adequacy (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06309251, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2025.

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