Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis

NCT ID: NCT03591861

Last Updated: 2022-12-12

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: NA
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-05-01
• Study Completion Date: 2022-11-01

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.

Conditions

Pediatric Brain Tumor

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

Ketogenic diet

Intervention Type: 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.

BCNU

Intervention Type: DRUG

-Standard of care

Interventions

Ketogenic diet

• 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.

Intervention Type: PROCEDURE

BCNU

-Standard of care

Intervention Type: DRUG

Other Intervention Names

Carmustine; BiCNU

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.

• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Washington University School of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Andrew Cluster, M.D.

Role: PRINCIPAL_INVESTIGATOR

Washington University School of Medicine

Locations

Washington University School of Medicine

St Louis, Missouri, United States

Countries

United States

Related Links

http://www.siteman.wustl.edu

Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

Other Identifiers

201806141

Identifier Type: -

Identifier Source: org_study_id