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Recurrent GlioblastomaPhase 1ASPIRENCT06389591

RNA-Lipid Particle (RNA-LP) Vaccines for Recurrent Adult Glioblastoma (GBM)

A Phase 1 study testing pp65 RNA loaded lipid particles, pp65 RNA-LPs (Drug Product 1 or DP1) and RNA loaded lipid particles, RNA-LPs (Drug Product 2 or DP2) in people with Recurrent Glioblastoma, 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

This is a Phase I study to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in adult patients with recurrent glioblastoma. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Recurrent Glioblastoma
What is being tested?
pp65 RNA loaded lipid particles, pp65 RNA-LPs (Drug Product 1 or DP1) and RNA loaded lipid particles, RNA-LPs (Drug Product 2 or DP2)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
1 siteUnited States
Study dates
2 Dec 2024to 1 Dec 2027
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 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age >/= 18years
  • Histopathologically proven GBM using the 2021 WHO Classification of Tumors of the CNS (WHO CNS5).
  • Unequivocal evidence of tumor progression as documented by brain MRI scan per RANO criteria.
  • Tumor must have a primary supratentorial component at the time of disease progression.
  • Patients must have received surgery and completed Fractionated Radiation therapy as frontline treatment for primary disease, either alone or with concurrent therapy (including temozolomide or another systemic chemotherapy agent). Patients must be at least 12 weeks post chemoradiation completion.
  • Patient must be at least 90 days from completion of prior radiation
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  • Any adverse events patient has experienced from prior therapy must have resolved to ≤ Gr. 1 according to CTCAE (NCI Common Terminology Criteria for Adverse Events) v5.0 prior to enrollment
  • Patient must be either weaned off steroids or weaned onto physiologic dosing at the time of enrollment.
  • Patient must be a candidate for surgery/biopsy as acceptable standard of care for sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs).
  • A diagnostic contrast-enhanced MRI of the brain must be performed preoperatively and postoperatively. Pre-op MRI must be performed within 28 days prior to study enrollment.
  • Performance Score: (KPS) ≥ 60. 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) ≥ 1,500µl (unsupported)
  • Platelets ≥ 100/µl (unsupported for at least 3 days)
  • Hemoglobin > 8 g/dL
  • Renal:
  • BUN ≤ 25 mg/dl
  • Creatinine ≤ 1.7 mg/dl
  • Hepatic
  • Bilirubin ≤ 2.0 mg/dl
  • ALT ≤ 5 times institutional upper limits of normal for age
  • AST ≤ 5 times institutional upper limits of normal for age
  • Patient must be able to give consent.
  • 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 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.

Who cannot join

  • Patients who received prior treatment with bevacizumab.
  • Known active infection (requiring treatment by antiviral or antibiotics) or immunosuppressive disease.
  • Patients with multifocal recurrent disease characterized by more than one enhancing lesion separated by noncontiguous T2/FLAIR signal abnormality. Patients with recurrence outside of the original tumor site are eligible if there is stability at the original site of disease.
  • Patients with uncontrolled seizure disorders
  • Any patients that have received any live vaccines within 30 days prior to enrollment
  • Tumors with primary localization to the brainstem or spinal cord
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  • Severe, active co-morbidity, defined as follows:
  • Unstable angina and/or congestive heart failure requiring hospitalization.
  • Unstable cardiac arrhythmias, abnormalities, or transmural myocardial infarction within the last 6 months.
  • Acute bacterial or fungal infection requiring intravenous treatment at study treatment.
  • Chronic Obstructive Pulmonary Disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy at study treatment
  • 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.
  • Patients with autoimmune disease requiring medical management with immunosuppressants.
  • Major medical illnesses or psychiatric impairments that, in the investigator's opinion, will prevent administration or completion of protocol therapy.
  • Pregnancy or women of childbearing potential and men who are sexually active and who are unwilling or unable to use an acceptable method of contraception for the entire study period; this exclusion is necessary because the treatment involved in this study may be significantly teratogenic.
  • Women of childbearing potential 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 projected first dose of study treatment.
  • 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

    Gainesville, Florida, 32608

Other trials in United States

About this study

Official title: A Phase I Study of RNA-Lipid Particle (RNA-LP) Vaccines for Recurrent Adult Glioblastoma (GBM)

This is a Phase I study to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in adult patients with recurrent glioblastoma.

Read the detailed description

This is a first in human Phase I study of RNA-LP vaccines for recurrent adult glioblastoma. Participants will receive two study drug products. The first, pp65 RNA-LP, is a messenger RNA (mRNA) pp65 vaccine given for the first 3 vaccines to try to change how the tumor behaves. The second study drug RNA-LP, given as monthly vaccines 4-15, includes pp65 mRNA and tumor RNA from each patient's tumor tissue.

Patients will may receive up to three pp65 RNA-LP vaccines (DP1) before receiving full dose monthly RNA-LPs (RNA loaded lipid particles, RNA-LPs, DP2). All participants will receive the same number of vaccines, up to 15.

The immunotherapy with RNA lipid particle (RNA-LP) vaccines is the treatment portion of this study. During this study, we will make, test and give the RNA-LP vaccine therapy. As part of this study, participants will undergo up to 4 additional MRIs.

Being tested
pp65 RNA loaded lipid particles, pp65 RNA-LPs (Drug Product 1 or DP1), RNA loaded lipid particles, RNA-LPs (Drug Product 2 or DP2)
Keywords
brain tumor, malignant brain tumor, immunotherapy, adult
Medical subject headings
Glioblastoma, Brain Neoplasms

What participants receive

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

  • pp65 RNA loaded lipid particles, pp65 RNA-LPs (Drug Product 1 or DP1)Biological

    pp65 RNA loaded lipid particles or pp65 RNA-LPs administered intravenously

  • RNA loaded lipid particles, RNA-LPs (Drug Product 2 or DP2)Biological

    personalized tumor mRNA, pp65 fl LAMP mRNA and DOTAP liposomes or RNA loaded lipid particles, RNA-LPs administered intravenously

Study groups in NCT06389591
Study groupRoleWhat they receive
Arm 1: pp65 RNA-LPs (DP1) before biopsyExperimentalParticipants will receive pp65 RNA-LPs (DP1) starting before tumor biopsy/resection. All patients will receive three pp65 RNA-LP vaccines (DP1) before receiving full dose monthly RNA-LPs (RNA loaded lipid particles, RNA-LPs, DP2).
Receives: Biological: pp65 RNA loaded lipid particles, pp65 RNA-LPs (Drug Product 1 or DP1); Biological: RNA loaded lipid particles, RNA-LPs (Drug Product 2 or DP2)

What is being measured

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

  1. Percentage of vaccines meeting release criteria in the DLT window during the first three vaccines

    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, the investigators will conclude that RNA-LPs can be successfully manufactured.

    Measured: from the date of surgery until administration of third vaccine, up to 20 weeks

  2. Incidence of investigational treatment related toxicities

    AEs and SAEs must be reported begins at time of first investigational product is received and ends 30 days after last investigational product is given.

    Measured: from first vaccine to 30 days after last dose of vaccine administered, up to 17 months

Timeline

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

  1. First posted29 Apr 2024
  2. Study start2 Dec 2024
  3. Primary completion1 Dec 2026 (estimated)
  4. Study completion1 Dec 2027 (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
NCT06389591
Sponsor's protocol ID
202301957
Other IDs
R37CA251978, OCR44973
Acronym
ASPIRE
Status last verified
Oct 2026
First submitted
24 Apr 2024
Last updated
6 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Carrera-Justiz S, Aghaee M, Qdaisat S, Weidert F, Garcia GA, Fagman L, Zhang D, Stover B, Moor RSF, Xie C, Goldenberg E, von Roemeling C, Doonan B, Chardon-Robles J, Elliott L, Sawyer WG, Deleyrolle LP, Sahay B, Foster TP, Seligson ND, Rahman M, Ghiaseddin A, Castillo P, Lee JH, Silver NL, Doty A, Ligon JA, Milner RJ, Mitchell D, Mendez-Gomez H, Moore H, Sayour EJ. Systemic mRNA vaccines elicit rapid immune activation in canine brain tumors. J Immunother Cancer. 2025 Nov 11;13(11):e011817. doi: 10.1136/jitc-2025-011817.

    PubMed 41218853

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing pp65 RNA loaded lipid particles, pp65 RNA-LPs (Drug Product 1 or DP1) and RNA loaded lipid particles, RNA-LPs (Drug Product 2 or DP2) in people with Recurrent Glioblastoma, 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 26 inclusion and 19 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 6 Oct 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?

Percentage of vaccines meeting release criteria in the DLT window during the first three vaccines (time frame: from the date of surgery until administration of third vaccine, up to 20 weeks). The study lists 2 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 NCT06389591, which also lists 1 linked publication.

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 6 Oct 2026.

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