Clinical trial · Interventional
Ketogenic Diet in Children With Malignant or Recurrent/Refractory Brain Tumor
A Phase II Study of the Ketogenic Diet in Children With Malignant or Recurrent/Refractory Brain Tumor
NCT03328858CI-TRIAL-00043132terminatedN/AClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): PI no longer available, not able to find replacement. 2 patients, lost to follow-up.
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Brain Tumors | Brain Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Ketogenic Diet | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- OTHER
- label
- Ketogenic Diet
- description
- Participants will be provided a consultation with a ketogenic dietitian, and test the tolerance to the diet in an inpatient mode
- interventionNames
- Other: Ketogenic Diet
Primary outcomes (1)
- measure
- Overall Response Rate
- timeFrame
- one year
- description
- To evaluate the overall response rate of subjects with malignant or refractory brain tumors receiving dietary therapy with the ketogenic diet
Secondary outcomes (4)
- measure
- Quality of Life Measures
- timeFrame
- one year
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 12 Months
- Maximum age
- 35 Years
Show eligibility criteria text
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.
References
Publications (6)
- BACKGROUNDGasior M, Rogawski MA, Hartman AL. Neuroprotective and disease-modifying effects of the ketogenic diet. Behav Pharmacol. 2006 Sep;17(5-6):431-9. doi: 10.1097/00008877-200609000-00009. PMID 16940764
- BACKGROUNDNeal EG, Chaffe H, Schwartz RH, Lawson MS, Edwards N, Fitzsimmons G, Whitney A, Cross JH. A randomized trial of classical and medium-chain triglyceride ketogenic diets in the treatment of childhood epilepsy. Epilepsia. 2009 May;50(5):1109-17. doi: 10.1111/j.1528-1167.2008.01870.x. Epub 2008 Nov 19. PMID 19054400
- BACKGROUNDKossoff EH. More fat and fewer seizures: dietary therapies for epilepsy. Lancet Neurol. 2004 Jul;3(7):415-20. doi: 10.1016/S1474-4422(04)00807-5. PMID 15207798
- BACKGROUNDLussier DM, Woolf EC, Johnson JL, Brooks KS, Blattman JN, Scheck AC. Enhanced immunity in a mouse model of malignant glioma is mediated by a therapeutic ketogenic diet. BMC Cancer. 2016 May 13;16:310. doi: 10.1186/s12885-016-2337-7. PMID 27178315
- BACKGROUNDVidone M, Clima R, Santorsola M, Calabrese C, Girolimetti G, Kurelac I, Amato LB, Iommarini L, Trevisan E, Leone M, Soffietti R, Morra I, Faccani G, Attimonelli M, Porcelli AM, Gasparre G. A comprehensive characterization of mitochondrial DNA mutations in glioblastoma multiforme. Int J Biochem Cell Biol. 2015 Jun;63:46-54. doi: 10.1016/j.biocel.2015.01.027. Epub 2015 Feb 7. PMID 25668474
- BACKGROUNDNebeling LC, Miraldi F, Shurin SB, Lerner E. Effects of a ketogenic diet on tumor metabolism and nutritional status in pediatric oncology patients: two case reports. J Am Coll Nutr. 1995 Apr;14(2):202-8. doi: 10.1080/07315724.1995.10718495. PMID 7790697