A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
A Pilot Study of Safety, Tolerability, and Immunological Effects of SurVaxM in Pediatric Patients With Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
3 other identifiers
interventional
35
2 countries
14
Brief Summary
Patients will receive a vaccine called SurVaxM on this study. While vaccines are usually thought of as ways to prevent diseases, vaccines can also be used to treat cancer. SurVaxM is designed to tell the body's immune system to look for tumor cells that express a protein called survivin and destroy them. The survivin protein can be found on up to 95% of glioblastomas and other types of cancer but is not found in normal cells. If the body's immune system knows to destroy cells that express survivin, it may help to control tumor growth and recurrence. SurVaxM will be mixed with Montanide ISA 51 before it is given. Montanide ISA 51 is an ingredient that helps create a stronger immune response in people, which helps the vaccine work better. This study has two phases: Priming and Maintenance. During the Priming Phase, patients will get one dose of SurVaxM combined with Montanide ISA 51 through a subcutaneous injection (a shot under the skin) at the start of the study and every 2 weeks for 6 weeks (for a total of 4 doses). At the same time that patients get the SurVaxM/Montanide ISA 51 injection, they will also get a second subcutaneous injection of a medicine called sargramostim. Sargramostim is given close to the SurVaxM//Montanide ISA 51 injection and works to stimulate the immune system to help the SurVaxM/Montanide ISA 51 work more effectively. If a patient completes the Priming Phase without severe side effects and his or her disease stays the same or improves, he or she can continue to the Maintenance Phase. During the 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 a patient finishes the study treatment, the doctor and study team will continue to follow his/her condition and watch for side effects up to 3 years following the last dose of SurVaxM/Montanide ISA 51. Patients will be seen in clinic every 3 months during the follow-up period.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Jul 2022
Longer than P75 for phase_1
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 20, 2021
CompletedFirst Posted
Study publicly available on registry
July 27, 2021
CompletedStudy Start
First participant enrolled
July 1, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 28, 2030
March 5, 2026
August 1, 2025
4.8 years
July 20, 2021
March 3, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Percentage of Subjects with Regimen-Limiting Toxicity (RLT)
The RLT rate during the first 8 weeks (14 days after the 4th priming dose of vaccine) will be used as the primary endpoint for safety monitoring and for determining when enrollment can begin in the next sub-cohort.
The first 8 weeks of protocol therapy (6 weeks for 4 priming doses +14 days follow-up from the 4th priming dose)
Percentage of Subjects with Pseudoprogression Related Regimen-Limiting Toxicity
A separate RLT assessment window for risk of pseudoprogression will encompass the 6 weeks for administration of 4 priming doses + 8 weeks of follow-up after administration of the 4th priming dose. Pseudoprogression RLTs during this interval will be included in the primary safety monitoring rules and analysis.
The first 14 weeks of protocol therapy (6 weeks for 4 priming doses + 8 weeks follow-up from the 4th priming dose)
Secondary Outcomes (1)
Differences in MR permeability and MR perfusion parameters for patients with true progression vs. pseudoprogression
Up to 3 years from treatment initiation.
Other Outcomes (3)
Best Response Rate
Up to 118 weeks of treatment
Progression Free Survival
Up to 3 years from treatment initiation
Overall Survival
Up to 3 years from treatment initiation
Study Arms (3)
SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥10 and ≤21 years
EXPERIMENTAL500 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 for patients with relapsed or progressive MB, HGG or ependymoma ages ≥1 and <10 years
EXPERIMENTAL500 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 for patients with non-relapsed DIPG post radiation-therapy ages ≥1 and ≤21 years
EXPERIMENTAL500 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.
Interventions
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.
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.
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.
Eligibility Criteria
You may qualify if:
- DIAGNOSIS: Patients with a histologically confirmed diagnosis of a primary CNS tumor that is progressive or recurrent defined as radiographic progression in any known residual tumor, or the appearance of one or more new lesions, leptomeningeal disease, or new cerebrospinal fluid (CSF) positivity for malignant cells, after most recent treatment modality. At the time of diagnosis or recurrence, all tumors must have histologic verification of one of the following:
- Medulloblastoma
- Glioblastoma multiforme (GBM)
- Anaplastic astrocytoma
- High-grade astrocytoma, NOS
- Anaplastic oligodendroglioma
- Anaplastic ependymoma (WHO Grade III)
- Ependymoma (WHO Grade II)
- Diffuse Intrinsic Pontine Gliomas (DIPG) Patients:
- Patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPGs), defined as tumors with a pontine epicenter and diffuse involvement of 2/3 or more of the pons, are eligible without histologic confirmation and will proceed directly to enrollment without screening.
- TUMOR TISSUE- Patients must provide tumor tissue (3 unstained slides or paraffin block) to determine their survivin expression status.
- Demonstration of survivin expression of at least 1% on tumor tissue by immunohistochemistry is required and must be performed in the central laboratory at Roswell Park Comprehensive Cancer Center (RPCCC) to confirm eligibility.
- Age: Patients must be ≥ 1 year of age and ≤ 21 years of age at the time of screening.
- Screening Consent: Participant is willing to sign a screening consent. The screening consent is to be obtained according to institutional guidelines. Assent, when appropriate, will be obtained according to institutional guidelines.
- Potential Eligibility for Study Enrollment: Patients screened for this trial should be expected to meet the criteria for treatment.
- +69 more criteria
You may not qualify if:
- BREAST FEEDING WOMEN - Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with SurVaxM breastfeeding should be discontinued if the mother is treated with SurVaxM. Female patients who are breastfeeding are not eligible for this study unless they agree not to breastfeed.
- Excluded Diagnoses:
- Patients with spinal cord primary tumors
- Patients with relapsed or progressive DIPG
- Patients with metastatic disseminated DIPG are not eligible. MRI of spine must be performed if disseminated disease is suspected by the treating physician.
- Patients with midline high grade gliomas including those with H3 K27M-altered diffuse midline glioma (DMG) centered outside of the pons
- Patients with Grade II myxopapillary ependymoma
- Patients with WHO Grade I or II gliomas are not eligible unless tumor is defined as DIPG as per above.
- Patients with bone-only metastatic lesions that do not have otherwise evaluable CNS disease.
- Bulky Disease
- Patients with bulky tumor on imaging are ineligible. Bulky tumor is defined as any of the following:
- Tumor with evidence of clinically significant tonsillar herniation
- Tumor with evidence of clinically significant uncal herniation causing midbrain compression or midline shift greater than 5 mm
- Tumor with a diameter \>4cm in one dimension on T2/FLAIR
- Tumor that in the opinion of the site investigator, shows significantly rapid progression of mass effect in either the brain or spinal cord such that the priming phase of vaccination (i.e., 6 weeks) cannot be completed before clinical deterioration is likely to occur.
- +19 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Children's Oncology Grouplead
- Roswell Park Cancer Institutecollaborator
- National Cancer Institute (NCI)collaborator
- American Lebanese Syrian Associated Charitiescollaborator
- Pediatric Brain Tumor Consortiumcollaborator
Study Sites (14)
Children's Hospital Los Angeles
Los Angeles, California, 90026, United States
Lucile Packard Children's Hospital at Stanford University Medical Center
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's National
Washington D.C., District of Columbia, 20010, United States
University of Florida
Gainesville, Florida, 32608, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263, United States
Memorial Sloan Kettering
New York, New York, 10065, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Clare Twist, MD
Roswell Park Comprehensive Cancer Center
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 20, 2021
First Posted
July 27, 2021
Study Start
July 1, 2022
Primary Completion (Estimated)
April 30, 2027
Study Completion (Estimated)
February 28, 2030
Last Updated
March 5, 2026
Record last verified: 2025-08
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, ICF, CSR
- Time Frame
- 6 months after publication and end timeframe may be up to 10 years.
The Pediatric Brain Tumor Consortium (PBTC) ensures that high dimensional molecular data generated from PBTC samples will be submitted to the NIH Database of Genotypes and Phenotypes (dbGaP) or other NIH-designated data repository. PBTC is required to make data available to other investigators for use in research projects. An investigator requesting data from published PBTC studies will submit a proposal describing the studies from which data are requested, the requested data, and a list of investigators in the project and their affiliated institutions, along with the investigator's CV to the PBTC Steering Committee for review. Once approved, the requesting investigator will be asked to complete a Data Transfer Agreement before the release of deidentified data. Requests for data are typically only considered once the primary study manuscript has been published though exceptions may be made in the event of long delays in the primary study data publication.