Clinical Trial: Dendritic Cell Vaccine Therapy With In Situ Maturation in Pediatric Brain Tumors

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: A Phase I Study of Dendritic Cell Vaccine Therapy With In Situ Maturation for Pediatric Brain Tumors

Brief Summary: DC vaccine manufactured and partially matured using our standard operating procedures, developed in collaboration with the HGG Immuno Group, then administered through imiquimod treated skin will be safe and feasible in children with refractory brain tumors. This will result in anti-tumor immunity that will prolong survival of subjects treated and results will be consistent with the outcomes found for subjects treated by HGG Immuno Group investigators. Study treatment will correlate with laboratory evidence of immune activation. Correlative studies will also reveal targets in the immune system which can be exploited to improve response for patients on successor trials.

Detailed Summary:
Sponsor: Edward Ziga

Current Primary Outcome:

• Rate of Toxicity in Study Participants Receiving Protocol Therapy [ Time Frame: Up to 28 Weeks ]
  Rate of treatment-limiting toxicities (TLT) and/or adverse events in study participants receiving protocol therapy.
• Rate of Feasibility of Protocol Therapy in Study Participants [ Time Frame: Up to 4 Weeks ]
  Rate of feasibility of protocol therapy in study participants. Feasibility refers to clinical feasibility - whether or not the patient can have enough monocytes removed to manufacture Dendritic Cells.

Original Primary Outcome: Number of Subjects Experiencing Adverse Events [ Time Frame: 36 months ]

Current Secondary Outcome:

• Rate of Prolonged Survival or Prolonged Progression-Free Survival in Study Participants [ Time Frame: Up to 24 Months ]
  Rate of prolonged survival or prolonged progression-free survival in study participants. Overall Survival is defined as the time elapsed from the start of treatment until death. For surviving patients, follow-up will be censored at the date of last contact. Progression-Free Survival (PFS) is defined as the time elapsed from the start of treatment to the date of documented progression or death, whichever comes first.
• Rate of Measurable Immune Response in Subjects Receiving Protocol Therapy. [ Time Frame: Up to 24 months ]
  Rate of measurable immune response in subjects receiving protocol therapy demonstrated by measurement levels of Myeloid Derived Suppressor Cells before and after treatment.
• Comparison of clinical parameters of study participants versus associated outcomes for patients on other DC/Imiquimod studies. [ Time Frame: Up to 24 Months ]
  A comparison of whether the clinical parameters associated with outcomes described for patients on other DC/Imiquimod protocols hold for subjects treated on this study.
• Estimation of the Proportion of Subjects with Recurrent Pediatric Brian Tumors who are able to complete DC Vaccine therapy and DC Vaccine + Lysate Therapy. [ Time Frame: Up to 24 months ]
  Estimation of the proportion of subjects with recurrent pediatric brain tumors who are able to receive all administrations of DC, and the proportion who are able to receive all administrations of DC and Lysate.
• Identification of Parameters Associated with Poorer Activity of the Vaccine in Study Participants [ Time Frame: Up to 24 Months ]
  Identification of parameters associated with poorer activity of the DC Vaccine in Study Participants in order to develop therapies to augment vaccine therapy.

Original Secondary Outcome:

• Number of Subjects Experiencing Complete Response or Partial Response according to RECIST Criteria 1.1 [ Time Frame: 36 months ]
  To demonstrate that treatment with dendritic cell vaccines loaded with tumor lysate created and matured through the in vivo process cause benefit for subjects in the form of prolonged survival or prolonged progression free survival.
• Measurement levels of Myeloid Derived Suppressor Cells before and after treatment [ Time Frame: 36 months ]
  To demonstrate that treatment with dendritic cell vaccines loaded with tumor lysate injected through imiquimod treated skin cause immune responses that can be measured in subjects.
• Number of Patients with Recurrent Brian Tumor who complete Therapy [ Time Frame: 36 months ]
  To estimate the proportion of patients with recurrent pediatric brain tumors who are able to receive all adminstrations of DC and the proportion who are able to receive all administrations of DC and lysate.

Dates:
Date Received: July 16, 2013
Date Started: September 3, 2013
Date Completion:
Last Updated: March 8, 2017
Last Verified: March 2017