SurVaxM Vaccine for Brain Cancer

Not currently recruiting at 15 trial locations

Overseen By Nina Butingan, MBS

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Pediatric Brain Tumor Consortium

Must not be taking: Cannabidiol, Medical marijuana, others

Disqualifiers: Active infection, Autoimmune disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 15

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a vaccine called SurVaxM for treating certain types of brain cancer. SurVaxM trains the immune system to target and destroy cancer cells carrying a specific protein, potentially controlling tumor growth. The trial consists of two phases: an initial phase with four vaccine doses over six weeks and a maintenance phase with doses every eight weeks for up to two years. This study may suit patients aged 1 to 21 with certain recurring or progressing brain tumors, such as medulloblastoma or glioblastoma, or newly diagnosed DIPG after radiation therapy. As a Phase 1 trial, this research focuses on understanding how SurVaxM works in people, offering participants the opportunity to be among the first to receive this innovative treatment.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot be on any other anti-cancer or investigational drug therapy, and certain medications like cannabidiol (CBD) or medical marijuana are not allowed. It's best to discuss your current medications with the study team to ensure eligibility.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that SurVaxM, a vaccine designed to help the immune system fight brain cancer, is safe for patients. An earlier study with individuals who have aggressive brain cancers like glioblastoma found that SurVaxM was well-tolerated, with most participants experiencing no severe side effects.

An ongoing study has also shown positive results, confirming that SurVaxM remains safe for many patients. The vaccine is administered with Montanide ISA 51, which boosts the immune response, and sargramostim, a drug that also supports the immune system. So far, these treatments have not raised significant safety concerns.

These findings are based on studies in other phases. While the results are promising, this trial aims to further confirm the vaccine's safety for participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about SurVaxM because it offers a novel approach to treating brain cancers like medulloblastoma (MB), high-grade gliomas (HGG), ependymomas, and diffuse intrinsic pontine glioma (DIPG). Unlike conventional treatments such as surgery, radiation, and chemotherapy, SurVaxM is a cancer vaccine that works by stimulating the immune system to target and destroy cancer cells. It uses a unique peptide called survivin, which is highly expressed in cancer cells but not in normal cells, making it a more targeted therapy. This approach aims to reduce the side effects typically associated with traditional therapies while potentially improving patient outcomes.

What evidence suggests that the SurVaxM vaccine might be an effective treatment for brain cancer?

Studies have shown that SurVaxM, an experimental vaccine, targets a protein called survivin, present in up to 95% of glioblastomas and other cancers but absent in normal cells. Research indicates that SurVaxM can help the immune system attack these cancer cells, potentially controlling tumor growth and preventing recurrence. Early trials demonstrated promising results for safety and immune system activation. In this trial, SurVaxM is combined with Montanide ISA 51 to enhance the immune response, and patients also receive sargramostim to further boost the immune system. These findings suggest that SurVaxM could be an effective treatment option for certain brain cancers. ^{1 2 4 6 7}

Who Is on the Research Team?

Clare Twist, MD

Principal Investigator

Roswell Park Comprehensive Cancer Center

Are You a Good Fit for This Trial?

This trial is for children and young adults aged 1 to 21 with specific brain tumors (like medulloblastoma, high-grade glioma, ependymoma) that are progressive or relapsed. They must have completed prior treatments without severe side effects, be HIV-negative or well-controlled on therapy, and have good organ function. Participants need to agree to use birth control and provide consent.

Inclusion Criteria

I have a high-grade brain tumor with clear signs on an MRI.

My hepatitis B virus load is undetectable with treatment.

I have a newly diagnosed brain tumor in the pons without needing a biopsy.

Exclusion Criteria

Patients living outside the US

I have had a condition where my lymphocytes grow abnormally.

I do not have an active infection needing treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Priming

Patients receive one dose of SurVaxM combined with Montanide ISA 51 and sargramostim every 2 weeks for 6 weeks (total of 4 doses)

8 weeks

4 visits (in-person)

Maintenance

Patients receive a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years

Up to 2 years

Every 8 weeks (in-person)

Follow-up

Patients are monitored for safety and effectiveness after treatment, with clinic visits every 3 months

Up to 3 years

Every 3 months (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• SurVaxM

Trial Overview

The study tests SurVaxM vaccine combined with Montanide ISA 51 in two phases: Priming (4 doses every 2 weeks) and Maintenance (doses every 8 weeks up to two years). It aims to train the immune system to attack tumor cells expressing survivin protein. Sargramostim is also given to boost the immune response.

How Is the Trial Designed?

Group I: SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥10 and ≤21 years Experimental Treatment 1 Intervention

500 mcg (1 mL) SurVaxM emulsion with Montanide ISA 51. Sargramostim dose is 3.33 mcg/kg/dose for patients \< 30 kg, and 100 mcg for patients ≥ 30 kg. Priming Phase: patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At each SurVaxM/Montanide ISA 51 injection, patients will also get an injection of sargramostim. Maintenance Phase: the patient will get a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years. After finishing study treatment, patients will be followed for up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be followed in clinic every 3 months during the follow-up.

Group II: SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥1 and <10 years Experimental Treatment 1 Intervention

500 mcg (1 mL) SurVaxM emulsion with Montanide ISA 51. Sargramostim dose is 3.33 mcg/kg/dose for patients \< 30 kg, and 100 mcg for patients ≥ 30 kg. Priming Phase: patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At each SurVaxM/Montanide ISA 51 injection, patients will also get an injection of sargramostim. Maintenance Phase: the patient will get a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years. After finishing study treatment, patients will be followed for up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be followed in clinic every 3 months during the follow-up.

Group III: SurVaxM for patients with non-relapsed DIPG post radiation-therapy ages ≥1 and ≤21 years Experimental Treatment 1 Intervention

500 mcg (1 mL) SurVaxM emulsion with Montanide ISA 51. Sargramostim dose is 3.33 mcg/kg/dose for patients \< 30 kg, and 100 mcg for patients ≥ 30 kg. Priming Phase: patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At each SurVaxM/Montanide ISA 51 injection, patients will also get an injection of sargramostim. Maintenance Phase: the patient will get a SurVaxM/Montanide ISA 51 dose along with a sargramostim dose about every 8 weeks for up to two years. After finishing study treatment, patients will be followed for up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be followed in clinic every 3 months during the follow-up.

SurVaxM is already approved in United States for the following indications:

Approved in United States as SurVaxM for:

• None approved yet; under investigation for glioblastoma, malignant gliomas, neuroendocrine tumors, multiple myeloma, medulloblastoma, high-grade glioma, ependymoma, and diffuse intrinsic pontine glioma

Who Is Running the Clinical Trial?

Pediatric Brain Tumor Consortium

Lead Sponsor

Trials

38

Recruited

1,600+

American Lebanese Syrian Associated Charities (ALSAC)

Collaborator

Trials

9

Recruited

670+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

American Lebanese Syrian Associated Charities

Collaborator

Trials

9

Recruited

670+

Roswell Park Cancer Institute

Collaborator

Trials

427

Recruited

40,500+

Published Research Related to This Trial

DCVax-Brain, a personalized treatment using dendritic cells to target brain tumors, shows promise in inducing antitumor immunity with low toxicity in patients with glioblastoma multiforme (GBM).

Recent trials indicate that DC vaccine therapy correlates with clinical improvements in GBM patients, although some reports suggest possible selection bias in the findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

DCVax-Brain and DC vaccines in the treatment of GBM. Wheeler, CJ., Black, KL.[2019]

SurVaxM, a peptide vaccine targeting the survivin protein in glioblastoma cells, was found to be safe and well tolerated in a phase IIa trial with 64 patients, showing no serious adverse events related to the vaccine.

The combination of SurVaxM with adjuvant temozolomide (TMZ) resulted in a high progression-free survival rate of 95.2% at 6 months and a median overall survival of 25.9 months, indicating promising efficacy in treating newly diagnosed glioblastoma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase IIa Study of SurVaxM Plus Adjuvant Temozolomide for Newly Diagnosed Glioblastoma. Ahluwalia, MS., Reardon, DA., Abad, AP., et al.[2023]

DCVax-L is a personalized cancer vaccine that uses a patient's own dendritic cells and tumor lysate to stimulate an immune response against glioblastoma multiforme (GBM).

Preliminary results from clinical trials, including ongoing Phase III studies, suggest that DCVax-L may offer a promising treatment option for patients with both newly diagnosed and recurrent GBM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dendritic cell immunotherapy for solid tumors: evaluation of the DCVax® platform in the treatment of glioblastoma multiforme. Hdeib, A., Sloan, AE.[2019]

The SurVaxM vaccine, targeting the survivin protein, was well tolerated in recurrent malignant glioma patients, with mostly mild side effects and no serious adverse events, indicating a favorable safety profile.

Immunologically, the vaccine successfully elicited immune responses in six out of eight evaluable patients, and showed promising clinical outcomes, with some patients maintaining stable disease for over 6 months and a median overall survival of 86.6 weeks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical study of a survivin long peptide vaccine (SurVaxM) in patients with recurrent malignant glioma. Fenstermaker, RA., Ciesielski, MJ., Qiu, J., et al.[2023]

Immunotherapy, particularly using active specific vaccination like the survivin-targeted peptide vaccine (SurVaxM), shows promise as a potential adjunctive treatment for malignant gliomas, especially given the limitations of conventional therapies.

Despite the optimism surrounding immunotherapy for gliomas, significant challenges remain in translating laboratory findings into effective Phase I and II clinical trials, highlighting the complexity of developing these treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Challenges in the development of a survivin vaccine (SurVaxM) for malignant glioma. Fenstermaker, RA., Ciesielski, MJ.[2022]

Vaccination strategies targeting cancer-associated antigens have shown promise in preclinical brain tumor models, demonstrating the immune system's ability to safely eliminate established tumors in the central nervous system.

Early phase clinical trials, particularly in glioblastoma patients, have confirmed the safety and immunogenicity of these vaccines, with initial results suggesting potential clinical benefits, although further studies are needed to establish definitive outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vaccination strategies for neuro-oncology. Sampson, JH., Mitchell, DA.[2018]

The DCVax-L trial indicated a potential survival benefit for glioblastoma patients receiving dendritic cell vaccination alongside standard care, showing improved overall survival in both newly diagnosed (19.3 months vs. 16.5 months) and recurrent settings (13.2 months vs. 7.8 months).

However, the trial's design and methodology raised significant concerns, including changes to endpoints and the use of external controls, which may have biased the results and limited the ability to draw reliable conclusions about the efficacy of the treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous tumor lysate-loaded dendritic cell vaccination in glioblastoma: What happened to the evidence? Olivier, T., Migliorini, D.[2023]

Citations

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Roswell Park Announces Positive Progress in Clinical ...

The clinical trial is designed to evaluate the safety, efficacy and overall survival benefit of SurVaxM in patients with newly diagnosed ...

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NCT04978727 | A Pilot Study of SurVaxM in Children ...

A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma.

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physicianresources.roswellpark.org

physicianresources.roswellpark.org/news/roswell-park-announces-positive-progress-in-clinical-trial-of-novel-immunotherapy-survaxm

Roswell Park Announces Positive Progress in Clinical Trial ...

The clinical trial is designed to evaluate the safety, efficacy and overall survival benefit of SurVaxM in patients with newly diagnosed ...

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PBTC-060 Version Date: 11/13/2024 1 ABSTRACT

This is a multicenter pilot study of SurVaxM (SVN53-67/M57-KLH) for children and young adults with progressive or relapsed medulloblastoma, high ...

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cancer.gov

cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2021-07694

Testing the Safety, Tolerability and Immunological Effects ...

This trial is studying a vaccine called SurVaxM combined with Montanide ISA 51 to see if it is safe and tolerated without severe side effects in children.

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cincinnatichildrens.org

cincinnatichildrens.org/service/c/clinical-trials/studies/PBTC-060

PBTC-060: A Pilot Study of SurVaxM in Children ...

This is a multicenter pilot study of SurVaxM (SVN53-67/M57-KLH) for children and young adults with progressive or relapsed medulloblastoma, high grade glioma, ...

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cancernetwork.com

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Phase 2b Trial for Cancer Vaccine in Newly Diagnosed ...

"SurVaxM's excellent safety profile and tolerability to date also help optimize quality of life for patients with this highly aggressive cancer, ...