KETO-TUMOR: a Study on Brain Tumors and Central Obesity

Single-arm, Open-label, Single-center, Non-profit Interventional Clinical Trial on the Effects of the Ketogenic Diet in Patients With Brain Tumors and Central Obesity

Hypothalamic-chiasmatic tumours account for 5-10% of CNS tumours in children and can compromise hypothalamic function, causing alterations in energy balance and weight gain. In inoperable cases, chemotherapy and radiotherapy are used; the latter, although the gold standard, is associated with significant neurocognitive and endocrine-metabolic side effects, proportional to the hypothalamic damage.

The ketogenic diet, used for decades in the treatment of drug-resistant childhood epilepsy, induces the use of ketone bodies as a source of energy for the brain and is effective in controlling seizures. Among the different variants, the modified Atkins diet was chosen in this study to promote better patient adherence.

This study aims to evaluate the effectiveness of the ketogenic diet (KD) in treating central obesity secondary to hypothalamic-chiasmatic tumours (gliomas, craniopharyngiomas, germ cell tumours, etc.), which often lead to excessive weight gain. This is refractory to drug therapy and lifestyle changes, such as low-calorie diets and exercise.

Study Overview

• Status: Recruiting
• Conditions: Hypothalamic Neoplasms; Obese Patients
• Intervention / Treatment: Behavioral: Dietary intervention

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Iacopo Sardi; Phone Number: 0555662631; Email: iacopo.sardi@meyer.it

Study Locations

• Italy
  • Florence
    • Florence, Florence, Italy, 50139
      • Recruiting
      • Meyer Children's Hospital IRCCS
      • Contact: Iacopo Sardi; Phone Number: 0555662631

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Diagnosis of hypothalamic-chiasmatic tumour according to the WHO 2021 classification
2. Diagnosis of hypothalamic obesity: after 5 years of age, BMI >97th percentile in the WHO 2007 curves
3. Males and females aged between 7 and 30 years
4. Performance status: Lansky score > 40 for patients aged < 18 years and Karnofsky score > 40 for patients aged between 18 and 30 years
5. Signature of informed consent to participate in the study
6. Signature of consent by the minor patient to participate in the study (7-13 years and 14-17 years).

Exclusion Criteria:

1. Deficiencies of:

• Primary carnitine
• Carnitine palmitoyltransferase 2 (CPT 2)
• Carnitine acylcarnitine translocase (CACT)
• Beta-oxidation
• Medium-chain acyl-CoA dehydrogenase (MCAD)
• long-chain acyl-CoA dehydrogenase (LCAD)
• short-chain acyl-CoA dehydrogenase (SCAD)
• porphyria
• pyruvate carboxylase
• long-chain 3-hydroxyacyl-CoA dehydrogenase.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Keto-tumor	Behavioral: Dietary intervention; Atkins Modified Diet (MAD)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percentage of patients who experience weight loss of at least 5% over 24 months	From enrollment to the end of the follow up period at 24 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Percentage of patients showing weight reduction, improvement in lipid and carbohydrate parameters	From enrollment to the end of the follow up period at 24 months
Percentage of patients with ketosis	From enrollement to the end of the follow up period at 24 months
Percentage of patients with serious adverse events	From enrollement to the end of the follow up period at 24 months
Adherence rate to the proposed dietary intervention, considering the number of patients who discontinue the diet and those who continue it	From enrollment to the end of the follow up at 24 months
Percentage of patients showing reduction of lean body mass in 24 months	From enrollment to the end of the follow up period at 24 months

Collaborators and Investigators

• Sponsor: Meyer Children's Hospital IRCCS

Study record dates

Study Major Dates

• Study Start (Actual): May 8, 2025
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2028

Study Registration Dates

• First Submitted: February 2, 2026
• First Submitted That Met QC Criteria: February 2, 2026
• First Posted (Actual): February 9, 2026

Study Record Updates

• Last Update Posted (Actual): February 9, 2026
• Last Update Submitted That Met QC Criteria: February 2, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms by Site; Neoplasms; Nervous System Neoplasms; Central Nervous System Neoplasms; Hypothalamic Diseases; Supratentorial Neoplasms; Brain Neoplasms; Hypothalamic Neoplasms; Therapeutics; Nutrition Therapy; Diet Therapy
• Other Study ID Numbers: KETO-TUMOR

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