Diabetes drug takes on childhood cancers in early safety trial

NCT ID NCT05521984

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Recruiting now This study
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Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
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Paused for now. It may or may not start again.
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Running, but no longer taking on new participants.
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The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
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Cancelled before anyone took part.

Expanded access (not trials)

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Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
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Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
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The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

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First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tests whether dapagliflozin, a diabetes medicine, is safe for children with brain or solid tumors that have come back or not responded to other treatments. About 20 children will receive the drug alongside standard care. The goal is to check for side effects, not to cure the disease.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2023

Expected to finish

Jun 2031

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. * 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. Exclusion Criteria: * 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. * 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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

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  1. The places running it

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  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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  3. A doctor treating you

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Contacts and locations

Locations

  • Washington University School of Medicine/St. Louis Children's Hospital

    RECRUITING

    St Louis, Missouri, 63110, United States

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