Actively Recruiting
Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)
Led by Washington University School of Medicine · Updated on 2026-04-02
20
Participants Needed
1
Research Sites
13 weeks
Total Duration
On this page
Sponsors
W
Washington University School of Medicine
Lead Sponsor
C
Children's Discovery Institute
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying the safety of dapagliflozin, added to standard care treatment, for children and young adults aged 6 to 21 with recurrent brain tumors or relapsed/refractory solid tumors. This pilot phase Ib study aims to find out if dapagliflozin is well-tolerated and safe in this group. The study also explores whether dapagliflozin can support chemotherapy by reducing tumor markers through its metabolic effects. Participants will take dapagliflozin orally once daily alongside their standard chemotherapy, which may include carmustine, topotecan, and cyclophosphamide. Dosing starts at 5 mg per day, with some patients increasing to 10 mg after two weeks under endocrinologist guidance. Treatment lasts 12 weeks, after which dapagliflozin is stopped. Chemotherapy cycles last 21 days for some participants. Throughout the study and up to 30 days after treatment ends, researchers will monitor adverse events and assess blood glucose, ketones, HbA1c, fructosamine, c-peptide, and glucagon levels. Tumor response will be evaluated using imaging before and after 12 weeks. Participants will be assessed regularly to track safety and treatment effects during the approximately 4-month study period.
CONDITIONS
Brief Title
Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of recurrent primary brain tumor with no curative therapy available OR relapsed/refractory solid tumor with no curative option after second-line therapy
- Measurable disease by pediatric RANO criteria for brain tumors or RECIST 1.1 criteria for solid tumors including sarcomas, neuroblastoma, Wilms tumor, and other rare solid tumors
- Life expectancy greater than 12 weeks
- Prior radiation, chemotherapy, or combined treatment allowed
- Age between 6 and 21 years inclusive
- Able to swallow whole pills
- Normal bone marrow and organ function: leukocytes ≥ 3,000/mcL, absolute neutrophil count ≥ 1,500/mcL, platelets ≥ 100,000/mcL, total bilirubin ≤ 1.5 x IULN, AST/ALT ≤ 3.0 x IULN, creatinine ≤ IULN or creatinine clearance ≥ 60 mL/min/1.73 m² if above normal
- Normal oxygenation on room air or DLCO > 80% if saturation < 97%
- Karnofsky or Lansky performance score ≥ 60
- Patients of childbearing potential and partners must agree to use two forms of contraception during study
- Ability to understand and sign IRB approved informed consent (or legal guardian consent)
You will not qualify if you...
- Current or previous treatment with SGLT2 inhibitors or thiazolidinediones
- Current use of high-dose dexamethasone exceeding 4 mg/day; stable or decreasing dose ≤ 0.1 mg/kg/day or max 4 mg/day required 7 days before dapagliflozin start
- History of other malignancy unless treated > 2 years ago with no disease or locally treated skin cancers
- Type 1 diabetes or current insulin treatment
- History of stroke or transient ischemic attack within past 5 years
- HbA1c greater than 8.5%
- Currently receiving other investigational agents
- History of allergic reactions to dapagliflozin or similar compounds
- Uncontrolled illnesses including active infection, symptomatic heart failure, unstable angina, arrhythmia, peripheral arterial disease, ketoacidosis, severe kidney disease (eGFR < 30), symptomatic hypotension, frequent urinary or yeast infections
- Pregnant or breastfeeding; negative pregnancy test required within 14 days before first dose
- HIV patients with CD4+ T-cell counts below 350 or recent AIDS-defining infections; concurrent effective ART recommended
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 weeks
Participants take dapagliflozin by mouth once daily alongside standard chemotherapy treatment for brain or solid tumors. Treatment lasts for 12 weeks with dose adjustments depending on age and treatment arm.
Weekly visits for up to 12 weeks
Duration - Approximately 1 month
Participants are monitored for adverse events and tumor response for 30 days after the last day of dapagliflozin treatment.
1 to 2 visits
Trial Site Locations
Total: 1 location
1
Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, United States, 63110
Actively Recruiting
Research Team
A
Andrew Cluster, M.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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