Ketogenic Diet in Children With Malignant or Recurrent/Refractory Brain Tumor

January 31, 2020 updated by: Nicklaus Children's Hospital f/k/a Miami Children's Hospital

A Phase II Study of the Ketogenic Diet in Children With Malignant or Recurrent/Refractory Brain Tumor

The purpose of this study is to evaluate the effect of the ketogenic diet on tumor size and quality of life in pediatric patients with malignant or recurrent/refractory brain tumors.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

Brain tumors account for nearly 20% of all childhood malignancies. Of these, gliomas represent 50% of all brain tumors in children and young adults. Gliomas are classically divided into two subtypes - low-grade and high-grade. Low-grade gliomas (LGG) include pilocytic astrocytomas and diffuse astrocytomas, and high-grade gliomas (HGG) include anaplastic astrocytoma and glioblastoma multiforme. Although patients with grade I and II tumors have a good prognosis with 5-year overall survival rates of 80-90%, those cases that are recurrent, refractory, and/or unresectable remain a challenge. The prognosis of children and young adults with recurrent or refractory malignant brain tumors remains poor despite dramatic improvements in treatment over the past few decades, with only a minority achieving long-term survival if recurrence occurs following initial surgical resection and adjuvant chemotherapy.

For patients with HGG prognosis remains dismal despite aggressive treatment. In this subset of patients, the 5-year overall survival for anaplastic astrocytoma ranges from 20-40% and for glioblastoma 15-20%. Diffuse intrinsic brain stem gliomas (DIPG) have the worst overall prognosis with a nine-month mean overall survival and with most patients dying from the disease within 2 years. Thus, the development of new treatment protocols for children and young adults with both high grade gliomas and with recurrent or refractory low grade gliomas is crucial to improving the survival rates of these patients.

The Ketogenic Diet (KD) has been in clinical use for nearly a century, initially designed to mimic the effects of starvation. Over the last two decades metabolic studies have been gaining momentum as increasingly promising in disease modification of central nervous system disorders and tumors.

Tests in animals and studies in adult patients with brain tumors have shown that there are advantages to using the ketogenic diet. These include: improved response of the tumor to standard treatment (chemotherapy/radiation) and improvement in quality of life measures (alertness).

Study Type

Interventional

Enrollment (Actual)

2

Phase

  • Not Applicable

Contacts and Locations

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

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital

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

6 months to 33 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosis Medulloblastoma, high-grade glioma, low-grade glioma, and ependymoma are eligible. Other central nervous system tumors may be considered for treatment at discretion of investigator. Pathology is required unless diffuse intrinsic pontine glioma or optic pathway tumor. The patient should have failed first line therapy and be considered refractory, relapsed, or recurrent. Exceptions are Grade III and Grade IV gliomas including brain stem gliomas who can be enrolled at initial diagnosis.
  • Prior Therapy Patients should have already had first line standard of care therapy, unless their initial diagnosis is high grade glioma or DIPG in which case they are eligible. Patients with high grade glioma can be enrolled after the completion of initial radiation. Investigator discretion may also be used.
  • Organ Function Requirements A. Functioning gastrointestinal system, i.e. ability to chew and swallow or alternative means of nutrition therapy such as a gastrostomy or jejunostomy tube B. SGPT (ALT) < 3 x upper normal limit; Total Bilirubin ≤ 2 x upper normal limit C. No active pancreatitis D. No arrhythmia or prolonged QT.

Exclusion Criteria:

  • History of cardiac arrhythmia
  • Patient unable to chew/swallow and who do not consent to alternative methods of nutrition support, e.g. nasogastric or nasoduodenal tube, or gastrostomy or jejunostomy tube placement.

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

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Ketogenic Diet
Participants will be provided a consultation with a ketogenic dietitian, and test the tolerance to the diet in an inpatient mode
Other: Ketogenic Diet
Once tolerance to the diet has been assessed, participants will be placed in the ketogenic diet, followed-up every three months until the one year completion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Response Rate
Time Frame: one year
To evaluate the overall response rate of subjects with malignant or refractory brain tumors receiving dietary therapy with the ketogenic diet
one year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quality of Life Measures
Time Frame: one year
To evaluate the changes of quality of life of subjects with relapsed or refractory brain tumors receiving dietary therapy with the ketogenic diet using age-appropriate quality of life modules (Peds QL).
one year
Ketosis Levels
Time Frame: one year
To compare the betahydroxybutyrate level to decrease tumor size using MRI.
one year
Blood Glucose Levels
Time Frame: one year
To compare average blood glucose during ketogenic diet initiation compared to decrease in tumor size at time of first follow-up MRI.
one year
Correlation of ketosis levels and ketogenic diet
Time Frame: one year
To evaluate correlation between degree of ketosis (betahydroxybutyrate level) and ketogenic diet ratio.
one year

Collaborators and Investigators

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

Sponsor

Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Investigators

  • Principal Investigator: Jennifer Caceres, MS, RDN, Nicklaus Children's Hospital
  • Study Director: Ziad Khatib, MD, Nicklaus Children's Hospital

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

Helpful Links

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)

November 1, 2017

Primary Completion (Actual)

January 31, 2020

Study Completion (Actual)

January 31, 2020

Study Registration Dates

First Submitted

October 23, 2017

First Submitted That Met QC Criteria

October 27, 2017

First Posted (Actual)

November 1, 2017

Study Record Updates

Last Update Posted (Actual)

February 5, 2020

Last Update Submitted That Met QC Criteria

January 31, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MCH05817

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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