GVAX Plus Checkpoint Blockade in Neuroblastoma
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|ClinicalTrials.gov Identifier: NCT04239040|
Recruitment Status : Recruiting
First Posted : January 23, 2020
Last Update Posted : October 12, 2021
This research clinical trial is studying the creation and administration of GVAX, an irradiated GM-CSF secreting, autologous neuroblastoma cell vaccine (GVAX) in combination with nivolumab and ipilimumab as a possible treatment for neuroblastoma.
The names of the study drugs involved in this study are:
- GVAX Vaccine, an immunotherapy developed from surgically removed tumor tissue
|Condition or disease||Intervention/treatment||Phase|
|Neuroblastoma Pediatric Solid Tumor||Procedure: Tissue collection Biological: GVAX vaccine Drug: Nivolumab Drug: Ipilimumab||Phase 1|
This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied.
The U.S. Food and Drug Administration (FDA) has not approved the GVAX vaccine as a treatment for any disease.
The U.S. Food and Drug Administration (FDA) has not approved nivolumab or ipilimumab for your specific disease but it has been approved for other uses.
The research study procedures include: screening for eligibility and study treatment including evaluations and follow up visits.
This phase 1 study will be conducted in 2 parts
- In the first part, participants will, as part of standard of care procedure to remove cancerous tissue, undergo neuroblastoma cell collection from a portion of resected tumor.
- Those cells will then be used to create the vaccine which will be stored for potential use during the second part of this research study.
- In the second part, participants who did not respond to standard therapy will receive the GVAX vaccine along with nivolumab and ipilimumab.
The study treatment will continue for up to 24 months and participants will be followed for 2 years after last study treatment (if received at least one vaccination).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||26 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1 Study of Combination Nivolumab and Ipilimumab With Irradiated GM-CSF Secreting Autologous Neuroblastoma Cell Vaccine (GVAX) for Relapsed or Refractory Neuroblastoma|
|Actual Study Start Date :||January 29, 2020|
|Estimated Primary Completion Date :||January 1, 2027|
|Estimated Study Completion Date :||January 1, 2027|
Experimental: Relapsed or Refractory High Risk Neuroblastoma
Tissue Collection of Cancerous cells during primary or clinically indicated surgical resection.
Manufacture and cryopreservation of vaccine. Treatment with vaccine, nivolumab and ipilimumab.
Procedure: Tissue collection
Collection of cancerous tissue to create GVAX vaccine or irradiated GMCSF-secreting autologous neuroblastoma cell vaccine at time of clinically indicated surgical procedure
Biological: GVAX vaccine
Vaccine injected weekly over initial 21 day cycle, biweekly for cycles 2-4 of 21 day cycle duration and cycles 5 and subsequent of 28 day cycle duration until vaccine supply is exhausted.
Other Name: GMCSF-secreting autologous neuroblastoma cell vaccine
Intravenous infusion of nivolumab every 3 weeks, for cycles 1-4. Cycles 1-4 are 21 days
Intravenous infusion of nivolumab biweekly for cycle 5 and subsequent of 28 day cycle duration. Subsequent 28 day cycles will last up to 2 years.
Other Name: Opdivo
Intravenous infusion of ipilimumab every 3 weeks, for cycles 1-4. Cycles 1-4 are 21 days
Other Name: Yervoy
- The number of participants with grade 4 toxicities [ Time Frame: Up to 2 years ]To assess safety, the number of grade 4 toxicities associated with vaccine and nivolumab/ipilimumab using the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5
- Number of Participants to receive 6 vaccinations [ Time Frame: Up to 2 years ]Feasibility will be assessed by the ability to make vaccine from available biopsy material in quantities sufficient for six vaccinations, for a given patient. A success rate (number of patients for whom quantity sufficient for six vaccinations exist / total number of eligible patients) under 65% may imply that the method needs further development or that the amount of tissue available in this population limits the feasibility of this strategy. Total number of vaccine doses administered per patient will also be counted
- Progression-free survival (PFS) [ Time Frame: time from receipt of first GVAX vaccine dose to the earlier of progression, relapse or death due to disease up to 48 months ]Kaplan-Meier plots, 1-year PFS estimate will be calculated, along with the standard errors
- Overall best response [ Time Frame: Start of the treatment until disease progression/recurrence up to 48 months ]Each patient will be categorized as a responder (≥PR) or a non-responder (<PR) by INRC criteria
- Overall Survival [ Time Frame: Time from receipt of the first GVAX vaccine dose to death due to any cause up to 48 months ]Kaplan-Meier plots, 1-year OS estimates will be calculated, along with the standard errors
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04239040
|Contact: Natalie B Collins, MD, PHDemail@example.com|
|United States, Massachusetts|
|Boston Children's Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Natalie B. Collins, MD firstname.lastname@example.org|
|Principal Investigator: Natalie B Collins, MD|
|Dana Farber Cancer Institite||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Natalie B Collins, MD 617-632-3027 email@example.com|
|Principal Investigator: Natalie B. Collins, MD|
|Principal Investigator:||Natalie B Collins, MD, PHD||Dana-Farber Cancer Institute|