Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis
Overview
- Phase
- Not Applicable
- Intervention
- Ketogenic diet
- Conditions
- Pediatric Brain Tumor
- Sponsor
- Washington University School of Medicine
- Enrollment
- 2
- Locations
- 1
- Primary Endpoint
- Feasibility of combining a ketogenic diet with chemotherapy in children with relapsed brain tumors as measured by the number of patients who can recruited with 3 years
- Status
- Terminated
- Last Updated
- 3 years ago
Overview
Brief Summary
The investigators will develop the concept of a sex-specific therapeutic intervention for gliomas that is based upon dietary carbohydrate restriction. The investigators will integrate metabolomics tools and FDG-PET imaging to validate the ketogenic diet on a sex-specific basis.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of a recurrent primary brain tumor with no curative therapy available.
- •Measurable disease using pediatric Response Assessment in Neuro-Oncology Criteria (RANO) criteria.
- •Life expectancy \> 12 weeks
- •Prior treatment with radiation alone, chemotherapy alone or combined radiation and chemotherapy is allowed.
- •Patient is \< 21 years of age
- •Normal bone marrow and organ function as defined below:
- •Leukocytes ≥ 3,000/mcL
- •Absolute neutrophil count ≥ 1,500/mcl
- •Platelets ≥ 100,000/mcl
- •Total bilirubin ≤ 1.5 x IULN
Exclusion Criteria
- •Patient does not have any of the following conditions as they are contraindicated for ketogenic diet:
- •Primary and secondary carnitine deficiency
- •Carnitine palmitoyltransferase I or II deficiency
- •Carnitine translocase deficiency
- •Mitochondrial β-oxidation defects
- •Pyruvate carboxylase deficiency
- •Glycogen storage diseases
- •Ketolysis defects
- •Ketogenesis defects
- •Porphyria
Arms & Interventions
Ketogenic Diet
* Caregivers (and participating children) attend intro ketogenic diet class (4 - 30 min lectures) * Clinic visit with neurologist, nurse, and dietitian prior to hospital admission and then once every 3 months * Laboratory studies prior to hospital admission and then at each follow-up visit * Hospital admission (3-5 day) to start the ketogenic diet * Standard of care chemotherapy with BCNU for up to 2 years * Ketogenic diet can continue for up to 2 years
Intervention: Ketogenic diet
Ketogenic Diet
* Caregivers (and participating children) attend intro ketogenic diet class (4 - 30 min lectures) * Clinic visit with neurologist, nurse, and dietitian prior to hospital admission and then once every 3 months * Laboratory studies prior to hospital admission and then at each follow-up visit * Hospital admission (3-5 day) to start the ketogenic diet * Standard of care chemotherapy with BCNU for up to 2 years * Ketogenic diet can continue for up to 2 years
Intervention: BCNU
Outcomes
Primary Outcomes
Feasibility of combining a ketogenic diet with chemotherapy in children with relapsed brain tumors as measured by the number of patients who can recruited with 3 years
Time Frame: Through completion of enrollment of all participants (estimated to be 3 years)
* The study will be defined as being feasible if all 15 patients can be recruited within 3 years * Please note that feasibility of the study is dependent on both primary outcome measures
Feasibility of combining a ketogenic diet with chemotherapy in children with relapsed brain tumors as measured by if at least 80% of the patients comply with the intervention
Time Frame: Up to 2 years
-The study will be defined as being feasible if at least 80% of the patients comply with the intervention as defined as achieving 80% of the targeted level of ketosis as assessed from laboratory measures and 80% of the planned BCNU doses --Please note that feasibility of the study is dependent on both primary outcome measures
Secondary Outcomes
- Tolerability of combining a ketogenic diet with chemotherapy in male children with relapsed brain tumors versus female children with relapsed brain tumors as measured by toxicity(Up to 2 years)
- Tumor response of male children with relapsed brain tumors to a ketogenic diet combined with chemotherapy versus female children with relapsed brain tumors as measured by progression-free survival (PFS)(Up to 10 years)