Experimental cell therapy targets deadly childhood brain cancer
NCT ID NCT03396575
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 participants) and was terminated early, so it is unclear how well it works, but researchers were checking for safety and immune responses.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- TTRNA-DC vaccines with GM-CSF and TTRNA-xALT T cells
- What this could lead to
- If it works, this could point toward a new treatment approach for DIPG, a rare and deadly childhood brain cancer.
- What could go wrong
- This was a very early, small Phase I trial that was terminated early, so results are limited. The treatment involves strong chemotherapy and cell therapy, which can cause serious side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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11 people
The number who actually took part.
- Started
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Jul 2018
- Finished
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Mar 2025
- Lead sponsor
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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
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3 to 30 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: Initial Screening * Radiologically confirmed DIPG or other diffuse intrinsic brain stem glioma (Grade III or IV). * Patient and/or parents/guardian willing to consent to biopsy for obtaining tumor material for confirmatory diagnosis and/or tumor RNA extraction and amplification. * Biopsy confirmation of any grade of glioma (for patients with classic DIPG on neuroimaging or at least grade III glioma in case of other diffuse intrinsic brain stem gliomas) * Karnofsky Performance Status (KPS) of \> 50% (KPS for \> 16 years of age) or Lansky performance Score (LPS) of ≥ 50 (LPS for ≤ 16 years of age) assessed within 2 weeks prior to registration; * Bone Marrow; * ANC (absolute neutrophil count) ≥ 1000/µl (unsupported) * Platelets ≥ 100,000/µl (unsupported) * Hemoglobin \> 8 g/dL (can be transfused) * Renal; * Serum creatinine ≤ upper limit of institutional normal * Hepatic; * Bilirubin ≤ 1.5 times upper limit of institutional normal for age * SGPT (ALT) ≤ 3 times upper limit of institutional normal for age * SGOT (AST) ≤ 3 times upper limit of institutional normal for age * Patients of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control while being treated on this study. * Signed informed consent according to institutional guidelines. Post Biopsy * Patients with post-surgical neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration; * Pathologic diagnosis of glioma on tumor biopsy. Exclusion Criteria: * Patients with severe dysphagia, obtundation, or tetraplegia (poor risks for anesthesia and biopsy procedure); * Absence of tumor on biopsy specimen; * Pregnant or need to breast feed during the study period (Negative serum pregnancy test required) * Known autoimmune or immunosuppressive disease or human immunodeficiency virus infection; * Patients with significant renal, cardiac, pulmonary, hepatic or other organ dysfunction; * Severe or unstable concurrent medical conditions; * Patients who require corticosteroids above physiologic doses (\>4 mg/day dexamethasone) after chemoradiotherapy; * Patients scheduled to receive any other concurrent anticancer or investigational drug therapy; * Prior allergic reaction to TMZ, GM-CSF, or Td; * Patients who are unwilling or unable to receive treatment and undergo follow-up evaluations at University of Florida; * Patient and/or parent/guardian demonstrating an inability to comply with the study and/or follow-up procedures.
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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.
Contacts and locations
Locations
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UF Health Shands Children's Hospital
Gainesville, Florida, 32610, United States
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