Nervous System Diseases

All Conditions

Neoplasms

Behaviors and Mental Disorders

Nutritional and Metabolic Diseases

Rare Diseases

Migraine Disorders

Brain Neoplasms

Autistic Disorder

Autism Spectrum Disorder

Epilepsy

Drug Resistant Epilepsy

Metabolic Diseases

Brain Diseases

Central Nervous System Diseases

Child Development Disorders, Pervasive

Developmental Disabilities

Neurodevelopmental Disorders

Mental Disorders

Psychotic Disorders

Quality of Life

Chronic Graft Versus Host Disease

Pediatric patients with any disease which require a ketogenic diet (KD), i.e. metabolic or genetic disorder or neurological (congenital and acquired) diseases

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.

ketogenic diet (as per clinical practice)

Antonella Diamanti, MD

Sara Olivotto, MD

Roberto Previtali, MD

Valentina De Giorgis, MD

Carlo Alberto Quaranta, MD

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

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.

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

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

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).

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)

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

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

Danone Nutricia SpA Società Benefit

Skin and Connective Tissue Diseases

Symptoms and General Pathology

Ganglion Cysts

Synovial Cyst

Cysts

Headache

Headache Disorders

Headache Disorders, Primary

Mucinoses

Connective Tissue Diseases

Anti-Arrhythmia Agents

Channel Blockers

Central Nervous System Depressants

All Drugs and Chemicals

Anti-Inflammatory Agents

Antirheumatic Agents

Analgesics

Hypoglycemic Agents

Anticonvulsants

Lidocaine

Ibuprofen

Topiramate

Anti-Inflammatory Agents, Non-Steroidal

Analgesics, Non-Narcotic

Cyclooxygenase Inhibitors

Enzyme Inhibitors

Besides orally topiramate plus ibuprofen as standardized drug treatment, patients will also receive SGBs plus standardized drug therapy; SGB will be performed once a week for 4 consecutive times in an individual series. The interval of each separate SGB procedure is 1 week. Every time patients with bilateral headache will be administered SGB alternately on each side at an interval of 40 min, and patients with unilateral headache will be administered SGB on the ipsilateral side;

The patients will only receive Topiramate plus ibuprofen as standardized drug treatment according to the guidelines for the treatment of CM.

All SGB procedures will be performed by the same experienced physicians. Patients will be positioned in a supine position with the neck slightly hyperextended and receive a SGB procedure using the B-ultrasound visualization technique (Aurora A5 Ultrasound, Risco Tech Co., Ltd.). 1% lidocaine 5mL (Shanxi Jinxin Shuanghe Pharm Co., Ltd.) will be injected below the prevertebral fascia on the surface of longus colli muscle

Stellate Ganglion Block (SGB)

Topiramate(Topiramate, Xi'an Yangsen Pharmaceutical Co., Ltd) will be started at a low dose and then increased gradually until optimally effective, side effects become intolerable, or the maximum recommended dose is reached. During the 4-week up-titration phase, the intention will increase dose weekly in 25 mg increments with the aim of reaching 50-100 mg/day. Ibuprofen((Fenbid, Tianjin Shike Pharmaceutical Co., Ltd)will be chosen as acute treatment, but no more than 10 times per month to avoid medication overuse headache.

standardized drug treatment

Fang Luo, M.D

Lu Liu

To assess the 6-months effects and safety of stellate ganglion block(SGB) for Chronic Migraine (CM) patients who failure to undergo preventive therapy and are seeking a more suitable non-pharmacological therapy.

The investigators aim to investigate whether the stellate ganglion block(SGB) is better than traditional medication treatment and provides medical evidence for the clinical application and promotion of SGB to provide a minimally invasive, safe, and effective treatment for patients with chronic migraine(CM) who failed to receive standardized drug treatment.

Ultrasound-guided Stellate Ganglion Block for Treatment of Chronic Migraine in Adult

A Prospective, Randomized, Controlled, Blinded-endpoint Study to Evaluate the Efficacy and Safety of Stellate Ganglion Block for CM Treatment

The primary outcome is the change from baseline in mean monthly (defined as 4 weeks) migraine days across 6-months follow-up period, which will be assessed by a blinded independent central review (BICR). Baseline is defined as the number of migraine days during the last 28 days prior to the randomization date. A migraine day is defined as a calendar day with symptoms of a migraine attack lasting at least 30 min.

Proportion of patients achieved≥50% reduction

Total effective rate = cure rate + markedly effective rate + effective rate

The Numeric Rating Scale(NRS) scoring method typically uses a score range of 0-10, with 0 indicating no pain or symptoms and 10 indicating the most severe pain or symptoms.

Patients satisfaction (PS) evaluated by PS scores, which typically uses a score range of 0-10, with 0 indicating no patient satisfaction and 10 indicating the most patient satisfaction.

The six item headache impact test (HIT-6) is a self-reported questionnaire measuring the "impact that headaches have on the ability to function on the job, at school, at home, and in social situations.

MIDAS is a reliable and validated scale measuring migraine-related disability regarding the need for medical care.. Higher MIDAS scores indicate more days with disability

PSQI is a self-administered assessment of sleep disturbances that may affect sleep quality, including sleep apnoea, with a higher score indicating more severe sleep disturbances.

Proportion of participants with adverse events (AEs) and serious adverse events

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Effectiveness and Impact on the Quality of Life of Ketogenic Diet in Pediatric Patients	NCT06309251	Drug Resistant ... Autism Spectrum... Chronic Migrain... Brain Tumor, Pe...	ketogenic diet ...	- 18 Years	Danone Nutricia SpA Società Benefit
Ultrasound-guided Stellate Ganglion Block for Treatment of Chronic Migraine in Adult	NCT06322407	Chronic Migrain...	Stellate Gangli... standardized dr...	18 Years - 65 Years	Beijing Tiantan Hospital

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