Pediatric Brain TumorPhase 1NCT05521984
Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)
A Phase 1 study testing Dapagliflozin, Carmustine and Topotecan in people with Pediatric Brain Tumor and Pediatric Solid Tumor, run by Washington University School of Medicine at 1 site in United States.
Trial summary
This is a pilot phase Ib study of the safety of dapagliflozin (in addition to standard of care treatment) for the treatment of pediatric patients with recurrent brain tumors and relapsed/refractory solid tumors. The primary hypothesis is that dapagliflozin is well-tolerated and safe to use in this patient population. Read more about this study
- Who is it for?
- Ages 6 to 21 years
- Conditions the study is for
- Pediatric Brain Tumor, Pediatric Solid Tumor
- What is being tested?
- Dapagliflozin, Carmustine and Topotecan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Apr 2023to 30 Jun 2031
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 6 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 OR diagnosis of relapsed/refractory solid tumor with no curative option and has trialed past a second line of therapy.
- Measurable disease per the following:
- For patients with brain tumors: measurable disease pediatric Response Assessment in Neuro-Oncology Criteria (RANO) criteria
- For patients with solid tumors: measurable disease using response evaluation criteria in solid tumors (RECIST 1.1). Includes patients with diagnoses of relapsed or refractory sarcomas, neuroblastoma, and Wilms tumor. Other rare solid tumors can be discussed with study chair.
- Life expectancy > 12 weeks.
- Prior treatment with radiation alone, chemotherapy alone or combined radiation and chemotherapy is allowed.
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- Patient is between 6 and 29 years old (inclusive)
- Patient is capable of swallowing whole pills
- 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 m^2 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 and their partners must agree to use two forms of acceptable contraception (including one barrier method) prior to study entry and for the duration of study participation. Should a female patient or partner of a male 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. Legally authorized representatives may sign and give informed consent on behalf of study participants. All patients and/or their parents or legal guardians must sign an IRB approved written informed consent document.
Who cannot join
- Current or previous treatment with SGLT2i or thiazolidinedione.
- Current use of high dose dexamethasone (exceeding 4 mg/day). Seven days prior to start of dapagliflozin, patients receiving dexamethasone must be on a stable or decreasing dose (≤ 0.1 mg/kg/day or maximum 4 mg/day). Note that it is preferred that patients not be on dexamethasone during the study.
- A history of other malignancy with the exceptions of malignancies for which all treatment was completed at least 2 years before registration with no evidence of disease and locally treated skin squamous or basal cell carcinoma.
- Type 1 diabetes or current insulin treatment.
- History of stroke or transient ischemic attack (in the last 5 years).
- HbA1c > 8.5%. The rationale is that this is the level that would require addition of insulin. However, insulin use is excluded in this study due to the increased risk of ketoacidosis.
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- Currently receiving any other investigational agents.
- A history of allergic reactions attributed to compounds of similar chemical or biologic composition to dapagliflozin or other agents used in the study.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, peripheral arterial disease, ketoacidosis, severe kidney disease (estimated glomerular filtration rate eGFR < 30 mL/min/1.73m^2), symptomatic hypotension, and chronic/frequent urinary tract infections or yeast infections.
- Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days prior to first dose of dapagliflozin.
- Patients with HIV are eligible unless their CD4+ T-cell counts are < 350 cells/mcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, 63110
Andrew Cluster, M.D., 314-273-1451, acluster@wustl.edu
Andrew Cluster, M.D.
About this study
This is a pilot phase Ib study of the safety of dapagliflozin (in addition to standard of care treatment) for the treatment of pediatric patients with recurrent brain tumors and relapsed/refractory solid tumors. The primary hypothesis is that dapagliflozin is well-tolerated and safe to use in this patient population. The investigators also hypothesize that dapagliflozin will be efficacious as an adjunct to front-line chemotherapy assessed by decreased tumor markers mediated by its pleiotropic metabolic effects.
- Conditions
- Pediatric Brain Tumor, Pediatric Solid Tumor
- Being tested
- Dapagliflozin, Carmustine, Topotecan, Cyclophosphamide
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DapagliflozinDrug
Commercially available
CarmustineDrug
Standard of care
TopotecanDrug
Standard of care
CyclophosphamideDrug
Standard of care
| Study group | Role | What they receive |
|---|---|---|
| Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) | Experimental | * Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (carmustine).
* Dapagliflozin 5 mg by mouth once daily on days 1-84 (duration of study)
* All patients will stop taking dapagliflozin after 12 weeks of treatment. Receives: Drug: Dapagliflozin; Drug: Carmustine |
| Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29) | Experimental | * Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (carmustine).
* Dapagliflozin will be initiated at 5 mg by mouth once daily, days 1-4 (2 weeks)
* Dapagliflozin will be escalated to 10 mg by mouth once daily for the remaining 10 weeks (after consultation with study endocrinologist)
* All patients will stop taking dapagliflozin after 12 weeks of treatment. Receives: Drug: Dapagliflozin; Drug: Carmustine |
| Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) | Experimental | * Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (topotecan + cyclophosphamide).
* Dapagliflozin 5 mg by mouth once daily on days 1-84 (duration of study)
* All patients will stop taking dapagliflozin after 12 weeks of treatment.
* Each cycle is 21 days. Receives: Drug: Dapagliflozin; Drug: Topotecan; Drug: Cyclophosphamide |
| Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29) | Experimental | * Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (topotecan + cyclophosphamide).
* Dapagliflozin will be initiated at 5 mg by mouth once daily, days 1-4 (2 weeks)
* Dapagliflozin will be escalated to 10 mg by mouth once daily for the remaining 10 weeks (after consultation with study endocrinologist)
* All patients will stop taking dapagliflozin after 12 weeks of treatment.
* Each cycle is 21 days. Receives: Drug: Dapagliflozin; Drug: Topotecan; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and type of adverse events experienced by participants
-Adverse events will be graded by CTCAE (version 5.0).
Measured: From start of treatment through 30 days after last day of dapagliflozin treatment (estimated to be 4 months)
Secondary outcomes (8)
Change in blood glucose
Measured: From baseline through end of treatment (estimated to be 3 months)
Change in ketones
Measured: From baseline through end of treatment (estimated to be 3 months)
Change in HbA1c
Measured: From baseline through end of treatment (estimated to be 3 months)
Changes in fructosamine
Measured: From baseline through end of treatment (estimated to be 3 months)
Changes in c-peptide
Measured: From baseline through end of treatment (estimated to be 3 months)
Changes in glucagon
Measured: From baseline through end of treatment (estimated to be 3 months)
Imaging-guided tumor response assessment
- Tumor response will be evaluated using the updated response assessment criteria for high-grade gliomas: Response Assessment in Neuro-Oncology (RANO) working group guideline for brain tumor patients.
- Tumor response will be evaluated using RECIST 1.1 for solid tumor patients.
Measured: From pre-therapy to post-12 weeks of therapy
Tumor response rate as measured by number of participants with complete response or partial response
- Tumor response will be evaluated using the updated response assessment criteria for high-grade gliomas: Response Assessment in Neuro-Oncology (RANO) working group guideline for brain tumor patients.
- Tumor response will be evaluated using RECIST 1.1 for solid tumor patients.
Measured: From pre-therapy to post-12 weeks of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2022
- Study start3 Apr 2023
- Primary completion31 Mar 2031 (estimated)
- Study completion30 Jun 2031 (estimated)
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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Discovery Institute
- Responsible party
- Sponsor
- Investigators
- Andrew Cluster, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05521984
- Sponsor's protocol ID
- 202210013
- Status last verified
- Jun 2026
- First submitted
- 26 Aug 2022
- Last updated
- 10 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)
- ClinicalTrials.gov ID
- NCT05521984
- Acronym / sponsor ID
- 202210013
- Conditions
- Pediatric Brain Tumor, Pediatric Solid Tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Children's Discovery Institute
- Study start
- 3 Apr 2023 (actual)
- Primary completion
- 31 Mar 2031 (estimated)
- Study completion
- 30 Jun 2031 (estimated)
- First posted
- 30 Aug 2022 (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 phase Ib study of the safety of dapagliflozin (in addition to standard of care treatment) for the treatment of pediatric patients with recurrent brain tumors and relapsed/refractory solid tumors. The primary hypothesis is that dapagliflozin is well-tolerated and safe to use in this patient population. The investigators also hypothesize that dapagliflozin will be efficacious as an adjunct to front-line chemotherapy assessed by decreased tumor markers mediated by its pleiotropic metabolic effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) | Experimental | Drug: Dapagliflozin, Drug: Carmustine* Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (carmustine). * Dapagliflozin 5 mg by mouth once daily on days 1-84 (duration of study) * All patients will stop taking dapagliflozin after 12 weeks of treatment. |
| Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29) | Experimental | Drug: Dapagliflozin, Drug: Carmustine* Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (carmustine). * Dapagliflozin will be initiated at 5 mg by mouth once daily, days 1-4 (2 weeks) * Dapagliflozin will be escalated to 10 mg by mouth once daily for the remaining 10 weeks (after consultation with study endocrinologist) * All patients will stop taking dapagliflozin after 12 weeks of treatment. |
| Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) | Experimental | Drug: Dapagliflozin, Drug: Topotecan, Drug: Cyclophosphamide* Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (topotecan + cyclophosphamide). * Dapagliflozin 5 mg by mouth once daily on days 1-84 (duration of study) * All patients will stop taking dapagliflozin after 12 weeks of treatment. * Each cycle is 21 days. |
| Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29) | Experimental | Drug: Dapagliflozin, Drug: Topotecan, Drug: Cyclophosphamide* Dapagliflozin will be initiated by mouth once daily at the same time as standard of care chemotherapy (topotecan + cyclophosphamide). * Dapagliflozin will be initiated at 5 mg by mouth once daily, days 1-4 (2 weeks) * Dapagliflozin will be escalated to 10 mg by mouth once daily for the remaining 10 weeks (after consultation with study endocrinologist) * All patients will stop taking dapagliflozin after 12 weeks of treatment. * Each cycle is 21 days. |
Who may qualify
- Ages
- 6 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Diagnosis of a recurrent primary brain tumor with no curative therapy available OR diagnosis of relapsed/refractory solid tumor with no curative option and has trialed past a second line of therapy.
- Measurable disease per the following:
- Life expectancy > 12 weeks.
- Prior treatment with radiation alone, chemotherapy alone or combined radiation and chemotherapy is allowed.
- Patient is between 6 and 29 years old (inclusive)
Key exclusion criteria (11 listed)
- Current or previous treatment with SGLT2i or thiazolidinedione.
- Current use of high dose dexamethasone (exceeding 4 mg/day). Seven days prior to start of dapagliflozin, patients receiving dexamethasone must be on a stable or decreasing dose (≤ 0.1 mg/kg/day or maximum 4 mg/day). Note that it is preferred that patients not be on dexamethasone during the study.
- A history of other malignancy with the exceptions of malignancies for which all treatment was completed at least 2 years before registration with no evidence of disease and locally treated skin squamous or basal cell carcinoma.
- Type 1 diabetes or current insulin treatment.
- History of stroke or transient ischemic attack (in the last 5 years).
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 |
|---|---|---|
| DapagliflozinCommercially available | Drug | Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29), Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10), Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29), Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) |
| CarmustineStandard of care | Drug | Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29), Brain Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) |
| TopotecanStandard of care | Drug | Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29), Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) |
| CyclophosphamideStandard of care | Drug | Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 11-29), Solid Tumor Cancer: Dapagliflozin + Standard of Care Chemotherapy (Ages 6-10) |
Primary outcome
Number and type of adverse events experienced by participants
Time frame: From start of treatment through 30 days after last day of dapagliflozin treatment (estimated to be 4 months)
-Adverse events will be graded by CTCAE (version 5.0).
8 secondary outcomes
Change in blood glucose
Time frame: From baseline through end of treatment (estimated to be 3 months)
Change in ketones
Time frame: From baseline through end of treatment (estimated to be 3 months)
Change in HbA1c
Time frame: From baseline through end of treatment (estimated to be 3 months)
Changes in fructosamine
Time frame: From baseline through end of treatment (estimated to be 3 months)
Changes in c-peptide
Time frame: From baseline through end of treatment (estimated to be 3 months)
Changes in glucagon
Time frame: From baseline through end of treatment (estimated to be 3 months)
Imaging-guided tumor response assessment
Time frame: From pre-therapy to post-12 weeks of therapy
Tumor response rate as measured by number of participants with complete response or partial response
Time frame: From pre-therapy to post-12 weeks of therapy
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2026.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
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 Phase 1 study testing Dapagliflozin, Carmustine and Topotecan in people with Pediatric Brain Tumor and Pediatric Solid 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 19 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 10 Jun 2026. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Andrew Cluster, M.D. (314-273-1451, acluster@wustl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number and type of adverse events experienced by participants (time frame: From start of treatment through 30 days after last day of dapagliflozin treatment (estimated to be 4 months)). 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 10 Jun 2026; AzTrials last refreshed its copy on 26 Oct 2026.