Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- 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
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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).
排除标准
- •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.
研究组 & 干预措施
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
干预措施: Ketogenic diet (Procedure)
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
干预措施: BCNU (Drug)
结局指标
主要结局
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
时间窗: 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
时间窗: 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
次要结局
- 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)