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Recurrent MedulloblastomaPhase 1PNOC020 rMBNCT07492316

RNA-lipid Particle (RNA-LP) Vaccines for Recurrent/Progressive Medulloblastoma (MB)

A Phase 1 study testing Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs) in people with Recurrent Medulloblastoma, run by University of Florida at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in pediatric patients with recurrent/progressive Medulloblastoma (MB) Read more about this study

Who is it for?
Ages 4 to 39 years
Conditions the study is for
Recurrent Medulloblastoma
What is being tested?
Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
1 siteUnited States
Study dates
24 Apr 2026to 31 Mar 2031
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 39 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age > 3 and </= 39 years.
  • Histologically confirmed or suspected recurrent/progressive MB in first or second relapse.
  • Patients must have received radiation therapy as part of prior therapy.
  • Patient must have been enrolled on a screening consent and have had sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs).
  • Prior Therapy: Patients must have fully recovered from all acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately.
  • XRT/External Beam Irradiation, including Protons: ≥ 90 days after local XRT; ≥ 150 days after TBI, craniospinal XRT or if radiation to ≥ 50% of the pelvis.
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  • Other therapeutic clinical trials: ≥ 14 days after last dose of investigational agent, unless otherwise defined above.
  • Patients must not have received prior exposure to pp65-directed therapy or any RNA-LP therapy.
  • A diagnostic contrast-enhanced MRI of the brain and spine must be performed preoperatively, and diagnostic contrast-enhanced MRI of the area biopsied or resected must be performed postoperatively. Pre-op MRI must be performed within 28 days prior to study enrollment. Post-op MRI must be completed within 7 days after surgery.
  • Performance Score: Karnofsky ≥ 60 for participants > 16 years of age and Lansky ≥ 60 for participants < 16 years of age (See Appendix A) assessed within 2 weeks prior to enrollment. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Bone Marrow:
  • ANC (Absolute neutrophil count) ≥ 1,000/μl (unsupported) e. Platelets ≥ 100/μl (unsupported for at least 7 days) f. Hemoglobin > 8 g/dL (may be supported)
  • Renal: Creatinine clearance or radioisotope GFR ≥ 70mL/min/1.73 m2
  • Hepatic:
  • Bilirubin ≤ 3 times upper limit of institutional normal for age. e. SGPT (ALT) ≤ 5 times upper limit of institutional normal for age. f. SGOT (AST) ≤ 5 times upper limit of institutional normal for age.
  • Participants who are receiving systemically-administered steroids must be on a stable or decreasing dose for >1 week prior to enrollment. The patient steroid dose should be no more than a dexamethasone-equivalent of 2.8 mg/m2/day. Corticosteroid physiologic replacement therapy for management of pituitary/adrenal axis insufficiency and/or topical administration (e.g. inhaled or dermatologic) is allowed.
  • Willing to take an antiepileptic medication such as levetiracetam for the duration of RNA-LP vaccinations
  • A legal parent/guardian or patient must be able to understand and be willing to sign a written informed consent document
  • For women of childbearing potential (WOCBP), negative serum/urine pregnancy test at enrollment
  • 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.
  • Males of child-fathering 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.
  • Participants with post-surgical neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.
  • Patients must be enrolled on PNOC COMP prior to enrollment on PNOC020 if PNOC COMP is open to accrual at the enrolling institution.

Who cannot join

  • Diffuse intrinsic pontine glioma, brainstem diffuse midline glioma, or BRAFV600E+
  • Bulky disease, defined as:
  • Tumor with evidence of clinically significant uncal herniation, midline shift, tonsillar herniation, or brainstem infiltration, or that shows significant mass effect in either brain or spine
  • Tumor with extensive and diffuse multilobular involvement (>3 lobes)
  • Tumor with extracranial disease (with the exception of spinal metastases in Stratum 3)
  • Known HIV, Hepatitis B, or Hepatitis C seropositive.
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  • Uncontrolled seizure disorder
  • History of myocarditis
  • Receipt of any live vaccine within 30 days prior to enrollment
  • Known active infection or immunosuppressive disease.
  • Participants with significant renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), pulmonary, hepatic or other organ dysfunction.
  • Severe or unstable concurrent medical conditions.
  • Women must not be pregnant or breast-feeding.
  • Participants who are receiving any other investigational agents or who have been treated on any other therapeutic clinical protocols within 30 days prior to study entry.
  • Participants who are unwilling or unable to receive treatment and undergo follow-up evaluations.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • UF Health Shands Children's Hospital

    Gainesville, Florida, 32608

Other trials in United States

About this study

Official title: A Phase I/II Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM), and Recurrent/Progressive Medulloblastoma (MB)

The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in pediatric patients with recurrent/progressive Medulloblastoma (MB)

Read the detailed description

This is a first in human Phase I study of RNA-LP vaccines for pediatric patients with recurrent/progressive Medulloblastoma (MB). The phase I portion of the study will involve a dose-escalation study to identify the maximally tolerated dose (MTD).

This clinical trial will consist of three parts: Surgery, Salvage Therapy (Radiation and/or chemotherapy), and Immunotherapy. Potentially eligible participants will be enrolled on a screening consent for the sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs). Pediatric participants will have tumor material sent to the University of Florida (UF) from qualified PNOC sites. Following surgical resection with confirmatory pathologic diagnosis, patients will be enrolled in the trial after informed consent has been obtained.

The RNA-LP vaccination will begin within 4 weeks following salvage therapy and after review of post-radiation MRI (for baseline). After radiation patients will receive three RNA-LP vaccines every 2 weeks before beginning 12 cycles of adjuvant monthly RNA- LP vaccines for a total of 15 vaccines.

Participants may receive RNA-LP vaccines for up to 14 months.

Participants will be followed until death due to any cause. MRI and clinical evaluation for assessment of disease progression will be conducted every 3 months for the first-year post-immunotherapy.

Being tested
Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)
Medical subject headings
Medulloblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)Biological

    RNA-LP vaccines will be administered intravenous. Three RNA-LP vaccines will be administered every 2 weeks followed by 12 cycles of adjuvant monthly RNA-LP vaccines for a total of 15 vaccines.

Study groups in NCT07492316
Study groupRoleWhat they receive
recurrent/progressive Medulloblastoma (rMB)ExperimentalNot described
Receives: Biological: Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Manufacturing feasibility

    Manufacturing feasibility will be determined based on the percentage of vaccines that are successfully manufactured in the DLT window during the first three vaccines. If two-thirds of vaccines are successfully manufactured with QA/QC clearance, we will conclude that RNA-LPs can be successfully manufactured.

    Measured: from the date of surgery until administration of third vaccine, up to 15 weeks

  2. Safety of RNA-LP vaccine

    Number of patients with DLTs at MTD.

    DLTs will be monitored for two weeks after the 3rd vaccine before continuing with the next dose escalation. If there are no DLTs, only patients receiving at least 3 vaccines without toxicity will be considered as safe for MTD assessment. Patients receiving less than 3 vaccines will be replaced for safety MTD assessments. Toxicity encountered before the 3rd vaccine will be considered a DLT. If 2 or more DLTs are observed at any dose level, the dose level is determined to be unsafe, and no additional patients will be treated at that level and there will be no escalation beyond that level.

    Measured: First vaccine through 14 days after administration of the 3rd vaccine

  3. Determination of Maximum Tolerated Dose

    A traditional 3+3 phase 1 design will be employed during this study, where dose escalations are planned in groups of three patients. No intra-patient escalation will be allowed, and dose escalation will not be considered until toxicity information is available from at least 3 evaluable patients at the current dose level.

    Measured: up to 60 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Mar 2026
  2. Study start24 Apr 2026
  3. Primary completion1 Sep 2030 (estimated)
  4. Study completion31 Mar 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07492316
Sponsor's protocol ID
IRB202001710-rMB
Other IDs
CC#200813
Acronym
PNOC020 rMB
Status last verified
Sep 2026
First submitted
19 Mar 2026
Last updated
29 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs) in people with Recurrent Medulloblastoma, run by University of Florida at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 23 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Manufacturing feasibility (time frame: from the date of surgery until administration of third vaccine, up to 15 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07492316.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.

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