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
|Condition or disease||Intervention/treatment||Phase|
|Brain Tumors||Other: Ketogenic Diet||Not Applicable|
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|
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.
- 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
- 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).
- Ketosis Levels [ Time Frame: one year ]To compare the betahydroxybutyrate level to decrease tumor size using MRI.
- 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.
- Correlation of ketosis levels and ketogenic diet [ Time Frame: one year ]To evaluate correlation between degree of ketosis (betahydroxybutyrate level) and ketogenic diet ratio.
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
|Contact: Jennifer Caceres, MS, RDNfirstname.lastname@example.org|
|Contact: Matthew Bittle, Supervsoremail@example.com|
|United States, Florida|
|Nicklaus Children's Hospital||Recruiting|
|Miami, Florida, United States, 33155|
|Contact: Jennifer Caceres, MS, RDN, LDN 786-624-4854 firstname.lastname@example.org|
|Contact: Matthew Bittle, MBA/HSA, RRC 786-624-2838 email@example.com|
|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|
|Principal Investigator:||Jennifer Caceres, MS, RDN||Nicklaus Children's Hospital|
|Study Director:||Ziad Khatib, MD||Nicklaus Children's Hospital|