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 Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Commercially available
Standard of care
Standard of care
Standard of care
Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, United States
RECRUITINGNumber and type of adverse events experienced by participants
-Adverse events will be graded by CTCAE (version 5.0).
Time frame: From start of treatment through 30 days after last day of dapagliflozin treatment (estimated to be 4 months)
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
* 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.
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
* 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.
Time frame: From pre-therapy to post-12 weeks of therapy
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