Immune cocktail takes on tough brain cancer in kids
NCT ID NCT06514898
First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 1 time
Summary
This early-phase trial is testing whether a combination of immune-based treatments is safe and feasible for children and young adults (ages 4–30) with a recurrent form of medulloblastoma, a type of brain cancer. The treatment includes personalized dendritic cell vaccines, immune cells grown in the lab, stem cells, and an immune checkpoint blocker. Only 12 participants will be enrolled, and the main goal is to see if the therapy can be given without causing severe side effects.
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, TTRNA-xALT (adoptive T cells), and PD-1 blockade (immune checkpoint inhibitor)
- What this could lead to
- If this approach proves safe and feasible, it could point toward a new treatment option for children and young adults with recurrent medulloblastoma that is hard to treat.
- What could go wrong
- This is a very early (Phase 1) pilot study with only 12 participants, so it is designed mainly to test safety and feasibility, not effectiveness. The combination therapy is complex and may cause serious side effects, and it is not yet known if it will help control the cancer.
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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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2025
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- 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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4 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: 1. Children and young adults ages 4-30 years with suspected recurrence/progression of Group 3 or 4 (non-SHH/non-WNT) MB since completion of definitive focal +/- craniospinal irradiation who are a candidate for surgical resection or biopsy. Of the 6 evaluable subjects, a minimum of 3 slots must be reserved for patients with confirmed Group 4 MB. Patients who are unable to receive radiation therapy due to genetic disorders that put them at significant risk for radiation-induced secondary malignancies (i.e. Gorlin's syndrome or NF1 mutation) are eligible for enrollment at first disease recurrence/progression. 2. Patients must currently be prescribed and approved to receive pembrolizumab therapy (patients who have progressed on anti-PD-1 targeting therapy but are otherwise eligible may be enrolled to receive combination with immunotherapy. Patients who have been previously treated with anti-PD-1 targeting therapy alone or in combination with other agents and discontinued for reasons other than toxicity may be enrolled). 3. Must be a candidate for surgery/biopsy Or tumor tissue obtained clinically, has been previously stored in a qualified site in a manner suitable for tumor RNA extraction and amplification and sample is made available to the PI. 4. Karnofsky or Lansky Performance Status (KPS) ≥ 60% (KPS for \> 16 years of age) or Lansky performance Score (LPS) of ≥ 60 (LPS for \< 16 years of age) 5. Adequate bone marrow and organ function as defined below: * ANC ≥ 1,000/mcL (unsupported) * Platelets ≥ 100,000/mcL (unsupported for at least 3 days) * Hemoglobin ≥ 9 g/dL (may be supported) * Serum creatinine ≤ 1.5 x IULN OR Creatinine clearance by Cockcroft-Gault ≥ 60 mL/min for patients with serum creatinine \> 1.5 x IULN * Serum total bilirubin ≤ 1.5 x IULN for age OR Direct bilirubin ≤ IULN for patients with total bilirubin \> 1.5 x IULN for age * AST (SGOT) and ALT (SGPT) ≤ 3 x IULN for age * Cardiac shortening fraction ≥27% or LVEF ≥50% by echocardiogram * Adequate pulmonary function defined as baseline pulse oximetry of ≥92% on room air 6. For females of childbearing potential, negative serum pregnancy test at enrollment 7. For women of childbearing potential (WOCBP) must be willing to use acceptable contraceptive methods to avoid pregnancy throughout the study and for at least 24 weeks after the last dose of study drug. or For males with female partners of childbearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 24 weeks following the last dose of study drug. 8. Signed informed consent by patient and/or legally authorized representative Exclusion Criteria: this study: 1. Prior discontinuation of PD-1 inhibitor treatment due to toxicity. 2. Corticosteroids equivalent to ≥ 4mg dexamethasone daily. 3. Known HIV, Hepatitis B, or Hepatitis C seropositive. 4. Known active infection or immunosuppressive disease. 5. Known autoimmune disease requiring medical management with immunosuppressant. 6. Pregnancy or lactation, due to possible adverse effects on the developing fetus or infant. 7. Treatment with another investigational drug or other intervention within 30 days prior to projected first dose of study treatment (Priming phase with TTRNA-DC). 8. Known severe, active co-morbidity, defined as follows: * Unstable angina and/or congestive heart failure requiring hospitalization. * Transmural myocardial infarction within the last 6 months. * Acute bacterial or fungal infection requiring intravenous antibiotics at time of enrollment. * Chronic Obstructive Pulmonary Disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy. * Hepatic insufficiency resulting in clinical jaundice and/or coagulation defects. * Acquired Immune Deficiency Syndrome (AIDS) based upon current CDC definition. The need to exclude patients with AIDS from this protocol is necessary because the treatments involved in this protocol may be significantly immunosuppressive. * Major medical illnesses or psychiatric impairments that, in the investigator's opinion, will prevent administration or completion of protocol therapy.
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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
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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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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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Children's National Hospital
NOT_YET_RECRUITINGWashington D.C., District of Columbia, 20010, United States
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University of Florida Health
RECRUITINGGainesville, Florida, 32608, United States