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

NCT06309251 | Recruiting DMC

• 1 other identifier: KetoSTrENgTH
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 3

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

Conditions

Drug Resistant Epilepsy; Autism Spectrum Disorder; Chronic Migraine; Brain Tumor, Pediatric

Outcome Measures

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).

  6 months

Secondary Outcomes (3)

• Nutritional adequacy

  6 months,(12 months optional)

• % of responders to KD

  6 months,(12 months optional)

• Health related quality of life improvement through parent questionnaire

  6 months,(12 months optional)

Study Arms (1)

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)

Interventions

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.

Patients requiring KD

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Danone Nutricia SpA Società Benefit: lead

Study Sites (3)

ASST Fatebenefratelli Sacco (PO "V. Buzzi")

Milan, MI, 20154, Italy

RECRUITING

Fondazione "Istituto Neurologico Nazionale C. Mondino"

Pavia, PV, 27100, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù

Roma, RM, 00165, Italy

RECRUITING

Related Publications (4)

• 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.

  PMID: 30647441 BACKGROUND

• 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.

  PMID: 17591624 BACKGROUND

• 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.

  PMID: 11739822 BACKGROUND

• 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.

  PMID: 29881797 BACKGROUND

MeSH Terms

Conditions

Drug Resistant Epilepsy; Autism Spectrum Disorder; Brain Neoplasms

Interventions

Diet, Ketogenic

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms

Intervention Hierarchy (Ancestors)

Diet, Carbohydrate-Restricted; Diet Therapy; Nutrition Therapy; Therapeutics; Diet; Nutritional Physiological Phenomena; Diet, Food, and Nutrition; Physiological Phenomena

Central Study Contacts

Antonella Diamanti, MD

CONTACT

+39 06 6859 2189; antonella.diamanti@opbg.net

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 29, 2024
• First Posted: March 13, 2024
• Study Start: March 1, 2022
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2026
• Last Updated: September 3, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

IT (3)