Recurrent Brain Tumor, ChildhoodNCT03955068
Strict Classic Ketogenic Diet as a Therapy for Recurrent or Progressive and Refractory Brain Tumors in Children
A study testing Strict Classic Ketogenic Diet in people with Recurrent Brain Tumor, Childhood and Ketogenic Diet, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
This is a pilot study to test the feasibility of implementing a strict classic ketogenic diet among a population of children with recurrent or progressive and refractory brain tumors. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Recurrent Brain Tumor, Childhood, Ketogenic Diet
- What is being tested?
- Strict Classic Ketogenic Diet
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2024to Jan 2027
- 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
- Age: Patients must be < 21 years of age inclusive at the time of enrollment on this protocol.
- Histologic Diagnosis: Histologically proven, primary brain tumor that is recurrent or progressive and refractory (nonresponsive) to standard, accepted therapy. A histopathologic diagnosis from either the initial presentation or at the time of recurrence is required for all subjects, except for diffuse intrinsic pontine glioma (DIPG).
- Measurable Disease: Patients must have radiographically documented measurable tumor as assessed by RANO or RAPNO criteria.
- Cranial and Spinal MRI: To document the degree of residual tumor, all patients must have an MRI of the brain with and without gadolinium performed within 2 weeks prior to study enrollment. For patients who undergo a tumor debulking surgery at the time of recurrence/progression, the following must be obtained:
- Cranial pre-operative MRI scan with and without contrast.
- Cranial post-operative MRI scan with and without contrast within 28 days following surgery. If possible, post-operative MRI should be performed within 48 hours of surgery, prior to the onset of edema or post-operative surgical enhancement, which can make measurements of residual tumor difficult.
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- Performance Level: Patients must have a performance status of > 50. Use Karnofsky score for patients > 16 years of age and Lansky score for patients ≤ 16 years of age.
- Life Expectancy: Patients must have a life expectancy of > 8 weeks.
- Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, biologic therapy, or radiotherapy prior to entering this study
- Growth factor(s): Must not have received within 1 week prior to entry onto this study.
- Other anti-cancer or experimental agents or therapies are not permitted. Homeopathic medicines are not permitted. Multivitamins are permitted.
- Organ Function Requirements: Adequate bone marrow, renal, liver, pulmonary, and central nervous system function
- All patients and/or their parents or legal guardians must sign a written informed consent.
- In the investigator's opinion, patients must have the ability to adhere to or tolerate the dietary protocol.
- Patients must be able to eat by mouth or must be willing to have a nasogastric tube (NGT) if gastrostomy tube is not already present.
Who cannot join
- Pregnancy
- Pregnant or breastfeeding. Female patients of childbearing potential must have negative pregnancy test result within the previous 14 days..
- Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation.
- Other Exclusionary Criteria: Disorder of B-oxidation, as measured by plasma aceyl-carnitine, Uncontrolled infection, History of nephrolithiasis, Diagnosis of diabetes mellitus that is being treated by medication.
- The following medications are excluded: Steroids: Therapeutic systemic doses of steroids must be stopped greater than 2 weeks prior to admission to the hospital to start the diet. A steroid washout of 1 week is acceptable for patients with DIPG. Concomitant chemotherapy drugs and homeopathic drugs are not permitted.
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas Southwestern Medical Center
Dallas, Texas, 75235
About this study
Official title: Pilot Study of a Strict Classic Ketogenic Diet as a Therapy for Recurrent or Progressive and Refractory Brain Tumors in Children
This is a pilot study to test the feasibility of implementing a strict classic ketogenic diet among a population of children with recurrent or progressive and refractory brain tumors. Eligible participants will be admitted to the neurosciences floor for 5 days to begin the ketogenic diet either orally, by gastrostomy tube or via nasogastric tube. During the inpatient stay, they will be extensively educated on the diet restrictions and rules. Participants will then continue on the diet at home returning about 17 times over the next 12 months. Daily logs will be kept tracking diet changes, bowel movements and urine ketones. Blood will be collected during the inpatient stay and at all visits for both standard clinical care and research testing.
Read the detailed description
Primary Objective is to determine the feasibility (safety and tolerability) of a strict classic ketogenic diet among a population of children with recurrent or progressive and refractory brain tumors.
Secondary Objectives are: (1) to determine the objective response rate (complete response + partial response) to a strict classic ketogenic diet among children with recurrent or progressive and refractory brain tumor; (2) to estimate the time interval to tumor progression (Progression-Free Survival), time to treatment failure (Event-Free Survival), and time to death (Overall Survival) after initiation of a strict classic ketogenic diet among a group of pediatric patients with recurrent or progressive and refractory brain tumors; and (3) to describe and characterize tolerability and toxicities from a strict classic ketogenic diet among children with recurrent or progressive and refractory brain tumors.
Exploratory Objective: Identify biomarker correlates, including MR spectroscopy and metabolites of tumor response to a strict classic ketogenic diet.
- Conditions
- Recurrent Brain Tumor, Childhood, Ketogenic Diet
- Being tested
- Strict Classic Ketogenic Diet
- Medical subject headings
- Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Strict Classic Ketogenic DietOther
Diet is administered from a 2:1 to 4:1 ratio; this means 2 to 4 parts of fat to 1 part of both protein (calculated based on RDA and whatever remaining portion of carbohydrates.
| Study group | Role | What they receive |
|---|---|---|
| Strict Classic Ketogenic Diet Arm | Other | The classic ketogenic diet is individually calculated for each patient based on age, weight, and nutritional needs. The diet is typically administered from a 2:1 to 4:1 ratio; this means 2 to 4 parts of fat to 1 part of both protein (calculated based on RDA and whatever remaining portion of carbohydrates. The basis of calculations is first on the amount of required protein needed to meet RDA to insure adequate growth. Fine tuning of ketogenic diet therapy is based on serum beta-hydroxybutyrate levels (target levels of 3.5-6.5 mmol/L), tolerance of the diet, and response to treatment. Receives: Other: Strict Classic Ketogenic Diet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility (safety and tolerability) of the ketogenic diet
Defined by the enrolled subject completing 28 days of ketogenic diet therapy without grade 3 - 5 adverse events. These will be assessed via NCI's CTCAE v5.0 toxicity criteria.
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2019
- Study startJul 2024 (estimated)
- Primary completionJan 2026 (estimated)
- Study completionJan 2027 (estimated)
Why it stopped: Lack of funding for this study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Brain Tumor, ChildhoodStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Daniel Bowers, University of Texas Southwestern Medical Center
- Investigators
- Daniel Bowers, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03955068
- Sponsor's protocol ID
- STU-2019-0853
- Status last verified
- May 2024
- First submitted
- 15 May 2019
- Last updated
- 22 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of a Strict Classic Ketogenic Diet as a Therapy for Recurrent or Progressive and Refractory Brain Tumors in Children
- ClinicalTrials.gov ID
- NCT03955068
- Acronym / sponsor ID
- STU-2019-0853
- Conditions
- Recurrent Brain Tumor, Childhood, Ketogenic Diet
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding for this study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- Jul 2024 (estimated)
- Primary completion
- Jan 2026 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 17 May 2019 (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.
This is a pilot study to test the feasibility of implementing a strict classic ketogenic diet among a population of children with recurrent or progressive and refractory brain tumors. Eligible participants will be admitted to the neurosciences floor for 5 days to begin the ketogenic diet either orally, by gastrostomy tube or via nasogastric tube. During the inpatient stay, they will be extensively educated on the diet restrictions and rules. Participants will then continue on the diet at home returning about 17 times over the next 12 months. Daily logs will be kept tracking diet changes, bowel movements and urine ketones. Blood will be collected during the inpatient stay and at all visits for both standard clinical care and research testing.
Detailed description
Primary Objective is to determine the feasibility (safety and tolerability) of a strict classic ketogenic diet among a population of children with recurrent or progressive and refractory brain tumors.
Secondary Objectives are: (1) to determine the objective response rate (complete response + partial response) to a strict classic ketogenic diet among children with recurrent or progressive and refractory brain tumor; (2) to estimate the time interval to tumor progression (Progression-Free Survival), time to treatment failure (Event-Free Survival), and time to death (Overall Survival) after initiation of a strict classic ketogenic diet among a group of pediatric patients with recurrent or progressive and refractory brain tumors; and (3) to describe and characterize tolerability and toxicities from a strict classic ketogenic diet among children with recurrent or progressive and refractory brain tumors.
Exploratory Objective: Identify biomarker correlates, including MR spectroscopy and metabolites of tumor response to a strict classic ketogenic diet.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Strict Classic Ketogenic Diet Arm | Other | Other: Strict Classic Ketogenic DietThe classic ketogenic diet is individually calculated for each patient based on age, weight, and nutritional needs. The diet is typically administered from a 2:1 to 4:1 ratio; this means 2 to 4 parts of fat to 1 part of both protein (calculated based on RDA and whatever remaining portion of carbohydrates. The basis of calculations is first on the amount of required protein needed to meet RDA to insure adequate growth. Fine tuning of ketogenic diet therapy is based on serum beta-hydroxybutyrate levels (target levels of 3.5-6.5 mmol/L), tolerance of the diet, and response to treatment. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- Age: Patients must be < 21 years of age inclusive at the time of enrollment on this protocol.
- Histologic Diagnosis: Histologically proven, primary brain tumor that is recurrent or progressive and refractory (nonresponsive) to standard, accepted therapy. A histopathologic diagnosis from either the initial presentation or at the time of recurrence is required for all subjects, except for diffuse intrinsic pontine glioma (DIPG).
- Measurable Disease: Patients must have radiographically documented measurable tumor as assessed by RANO or RAPNO criteria.
- Cranial and Spinal MRI: To document the degree of residual tumor, all patients must have an MRI of the brain with and without gadolinium performed within 2 weeks prior to study enrollment. For patients who undergo a tumor debulking surgery at the time of recurrence/progression, the following must be obtained:
- Cranial pre-operative MRI scan with and without contrast.
Key exclusion criteria (5 listed)
- Pregnancy
- Pregnant or breastfeeding. Female patients of childbearing potential must have negative pregnancy test result within the previous 14 days..
- Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation.
- Other Exclusionary Criteria: Disorder of B-oxidation, as measured by plasma aceyl-carnitine, Uncontrolled infection, History of nephrolithiasis, Diagnosis of diabetes mellitus that is being treated by medication.
- The following medications are excluded: Steroids: Therapeutic systemic doses of steroids must be stopped greater than 2 weeks prior to admission to the hospital to start the diet. A steroid washout of 1 week is acceptable for patients with DIPG. Concomitant chemotherapy drugs and homeopathic drugs are not permitted.
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 |
|---|---|---|
| Strict Classic Ketogenic DietDiet is administered from a 2:1 to 4:1 ratio; this means 2 to 4 parts of fat to 1 part of both protein (calculated based on RDA and whatever remaining portion of carbohydrates. | Other | Strict Classic Ketogenic Diet Arm |
Primary outcome
Feasibility (safety and tolerability) of the ketogenic diet
Time frame: 28 days
Defined by the enrolled subject completing 28 days of ketogenic diet therapy without grade 3 - 5 adverse events. These will be assessed via NCI's CTCAE v5.0 toxicity criteria.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2024.
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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.
Daniel Bowers, MD principal investigator
University of Texas Southwestern Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Strict Classic Ketogenic Diet in people with Recurrent Brain Tumor, Childhood and Ketogenic Diet, run by University of Texas Southwestern Medical Center 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 15 inclusion and 5 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: withdrawn, as of the sponsor's last update on 22 May 2024. 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 withdrawn before enrolling participants, so there are no results.
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 (safety and tolerability) of the ketogenic diet (time frame: 28 days). 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 22 May 2024.