Therapeutic Targeting of Sex Differences in Pediatric Bra... | NCT03591861

NCT03591861

Protocol Section

Identification Module

Results Section

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Annotation Section

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Document Section

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Derived Section

Miscellaneous Info Module

Status Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Ketogenic Diet	Experimental	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	Procedure: Ketogenic diet Drug: BCNU

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Ketogenic diet	Procedure	Children under 2 years will immediately start on a full calorie classical 3:1 ketogenic diet. The 3:1 ratio indicates that the diet contains 3 g of fat for every 1 g of protein + carbohydrate, which results in about 87% of calories coming from fat. Children 2 years or older will skip breakfast and lunch on the day of admission but start a full calorie 3:1 ketogenic diet at dinner.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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	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	Through completion of enrollment of all participants (estimated to be 3 years)
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	-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	Up to 2 years

Secondary Outcomes

Measure	Description	Time Frame
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	All toxicities will be summarized by noting the count of participants who experience each toxicity The descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be utilized for all toxicity reporting.

Eligibility Module

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
- AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
- Creatinine ≤ IULN OR creatinine clearance ≥ 60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal
- Normal room air oxygenation must be documented. If room air oxygen saturation is less than 97%, a diffusion capacity of carbon monoxide (DLCO) of greater than 80%, must be demonstrated.
Karnofsky or Lansky performance score of ≥ 60
Patients of childbearing potential must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a female patient become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).

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
- Prolonged QT syndrome
- Liver insufficiency
- Renal insufficiency
- Pancreatic insufficiency
- Pulmonary insufficiency
- Hyper insulinism
Pregnant and/or breastfeeding. Female patients of childbearing potential must have a negative pregnancy test within 14 days of study entry.

ID	Term
D055423	Diet, Ketogenic
D002330	Carmustine

ID	Term
D050528	Diet, Carbohydrate-Restricted
D004035	Diet Therapy
D044623	Nutrition Therapy
D013812	Therapeutics