Pediatric Brain TumorNCT03591861
Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis
A study testing Ketogenic diet and BCNU in people with Pediatric Brain Tumor, run by Washington University School of Medicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Pediatric Brain Tumor
- What is being tested?
- Ketogenic diet and BCNU
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 1 May 2019to 1 Nov 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
Show all 16Show fewer
- 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).
Who cannot join
- 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
Show all 17Show fewer
- 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 locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
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
- Being tested
- Ketogenic diet, BCNU
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ketogenic dietProcedure or surgery
- 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.
BCNUDrug
Also called Carmustine, BiCNU
-Standard of care
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Procedure: Ketogenic diet; Drug: BCNU |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: Up to 2 years
Secondary outcomes (2)
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.
Measured: 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)
- PFS is defined as the duration of time from start of treatment to time of radiographic progression or death due to any cause, whichever occurs first.
- Progression will be defined by the Response Assessment in Neuro-Oncology (RANO) working group guideline.
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2018
- Study start1 May 2019
- Primary completion1 Nov 2022
- Study completion1 Nov 2022
Why it stopped: Low accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Brain TumorStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andrew Cluster, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03591861
- Sponsor's protocol ID
- 201806141
- Status last verified
- Dec 2022
- First submitted
- 20 Jun 2018
- Last updated
- 12 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis
- ClinicalTrials.gov ID
- NCT03591861
- Acronym / sponsor ID
- 201806141
- Conditions
- Pediatric Brain Tumor
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low accrual
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 1 May 2019 (actual)
- Primary completion
- 1 Nov 2022 (actual)
- Study completion
- 1 Nov 2022 (actual)
- First posted
- 19 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ketogenic Diet | Experimental | Procedure: Ketogenic diet, Drug: BCNU* 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 |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- 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
Key exclusion criteria (17 listed)
- Patient does not have any of the following conditions as they are contraindicated for ketogenic diet:
- Pregnant and/or breastfeeding. Female patients of childbearing potential must have a negative pregnancy test within 14 days of study entry.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Procedure or surgery | Ketogenic Diet |
| BCNUAlso: Carmustine, BiCNU-Standard of care | Drug | Ketogenic Diet |
Primary outcome
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
2 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
Time frame: 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)
Time frame: Up to 10 years
Results and safety
The study was stopped early (reason given: Low accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2022.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Pediatric Brain Tumor
Recruiting
Facial Expression Awareness Training to Understand and Recognize Emotions
Survivors of Pediatric Brain Tumors, Pediatric Cancer
Recruiting
High-Intensity Interval Exercise in Young Adult Survivors of Pediatric Brain Tumors: A Pilot Feasibility Study
Pediatric Brain Tumor
Not yet recruiting
Measuring Brain Changes Following Cognitive Intervention in Pediatric Patients With Brain Tumors
Brain Tumor, Pediatric
Recruiting
Irradiation in Paediatrics: Neurocognition, Neuroimaging and Evaluation of Memory and Attention
Brain Tumor, Pediatric
RecruitingPhase 3
Methylphenidate in Pediatric Brain Tumor Survivors With Cancer-related Fatigue
Brain Tumor, Pediatric, Cancer-related Fatigue
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew Cluster, M.D. principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Ketogenic diet and BCNU in people with Pediatric Brain Tumor, run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 12 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Low accrual). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2022.