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

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ClinicalTrials.gov Identifier: NCT03328858
Recruitment Status : Recruiting
First Posted : November 1, 2017
Last Update Posted : March 2, 2018
Sponsor:
Information provided by (Responsible Party):
Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Brain Tumors Other: Ketogenic Diet Not Applicable

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  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Ketogenic Diet will be initiated as inpatient, once all necessary studies are obtained and reviewed. The "classic ketogenic diet" will be initiated, and once ketosis is reached and patient demonstrates tolerance to diet he/she will be discharged home. Follow-up visits will be necessary up to 1 year.
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: A Phase II Study of the Ketogenic Diet in Children With Malignant or Recurrent/Refractory Brain Tumor
Actual Study Start Date : November 1, 2017
Estimated Primary Completion Date : November 30, 2022
Estimated Study Completion Date : November 30, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Brain Tumors

Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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).

  2. Ketosis Levels [ Time Frame: one year ]
    To compare the betahydroxybutyrate level to decrease tumor size using MRI.

  3. 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.

  4. Correlation of ketosis levels and ketogenic diet [ Time Frame: one year ]
    To evaluate correlation between degree of ketosis (betahydroxybutyrate level) and ketogenic diet ratio.



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Ages Eligible for Study:   12 Months to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03328858


Contacts
Contact: Jennifer Caceres, MS, RDN 786-624-4854 jennifer.caceres@mch.com
Contact: Matthew Bittle, Supervsor 786-624-2838 matthew.bittle@nicklaushealth.org

Locations
United States, Florida
Nicklaus Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Jennifer Caceres, MS, RDN, LDN    786-624-4854    jennifer.caceres@mch.com   
Contact: Matthew Bittle, MBA/HSA, RRC    786-624-2838    matthew.bittle@nicklaushealth.org   
Principal Investigator: Jennifer Caceres, MS, RDN, LDN         
Sub-Investigator: Ziad Khatib, MD         
Sub-Investigator: Michael Barbato, MD         
Sub-Investigator: Maria Pilar Generao, ARNP, CNRN         
Sub-Investigator: Nolan Altman, MD         
Sub-Investigator: Sara Rivero-Conil, PsyD         
Sub-Investigator: Daria Salyakina, PhD         
Sub-Investigator: Weize Wang, Statistician         
Sponsors and Collaborators
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

Additional Information:
Publications:
Responsible Party: Nicklaus Children's Hospital f/k/a Miami Children's Hospital
ClinicalTrials.gov Identifier: NCT03328858     History of Changes
Other Study ID Numbers: MCH05817
First Posted: November 1, 2017    Key Record Dates
Last Update Posted: March 2, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nicklaus Children's Hospital f/k/a Miami Children's Hospital:
Ketogenic Diet, brain tumors

Additional relevant MeSH terms:
Brain Neoplasms
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases