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Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages N/A - 21 Years
Gender All
More Inclusion & Exclusion 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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03591861
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

N/A
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Washington University School of Medicine
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Andrew Cluster, M.D.
Principal Investigator Affiliation Washington University School of Medicine
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Pediatric Brain Tumor
Study Website: View Trial Website
Arms & Interventions

Arms

Experimental: 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

Interventions

Procedure: - 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.

Drug: - BCNU

-Standard of care

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Washington University School of Medicine, Saint Louis, Missouri

Status

Recruiting

Address

Washington University School of Medicine

Saint Louis, Missouri, 63110

Site Contact

Andrew Cluster, M.D.

acluster@wustl.edu

314-273-1451