A Combined Cell Therapy Approach to the Treatment of Neuroblastoma

This study adds an experimental treatment with another type of cells, called dendritic cells. It is hoped that these cells may stimulate the immune system to react against neuroblastoma in much the same way that vaccines cause the immune system to react to certain viruses and bacteria. The physicians conducting this study have observed from previous research that neuroblastoma cells can be recognized by the immune system, and that they can be destroyed by immune cells.The main goal of this study is to see if giving participants this additional anti-Neuroblastoma vaccine reduces the risk of relapse following the Hematopoietic Stem Cell Transplant.

Study Overview

• Status: Withdrawn
• Conditions: Neoplasms, Nerve Tissue; Neuroblastoma
• Intervention / Treatment: Procedure: Autologous Hematopoietic Progenitor Cell Transplant; Biological: KLH and Tumor Lysate Pulsed DC Vaccine

Detailed Description

All patients who are enrolled in this study will receive all treatment at All Children's Hospital which is located at 501 6th Ave South, St. Petersburg, FL 33701. This includes autologous cell donation by apheresis, high dose cytotoxic therapy conditioning for autologous HPC transplant, post-transplant follow-up care, and all administration of dendritic cell vaccines and blood draws for post therapy immunological monitoring.

All preparation of cellular products, including hematopoietic progenitor cell products for autologous transplantation, and dendritic cell vaccine products, will be carried out in the Cell Therapy Facility located within the Moffitt Cancer Center, which is located at 12902 Magnolia Drive, Tampa, FL 33612.

• Study Type: Interventional
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Must have a histological diagnosis of neuroblastoma or ganglioneuroblastoma and be either newly diagnosed with high risk disease or have failed previous treatment: Patients who have failed previous treatment may have had no more than one earlier autologous HPC transplant.
• Participant is expected to undergo autologous HPC transplantation that is consistent with standard of care.
• Must have the presence of residual resectable disease for which surgery is clinically indicated, and will be performed at Johns Hopkins All Children's Hospital.

Exclusion Criteria:

• Not an eligible candidate for collection by apheresis or HPC transplant.
• History of autoimmune disorder or immune deficiency disorder.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Combined Cell Therapy; Hematopoietic progenitor cell (HPC) transplant (HPCT) with autologous tumor cell lysate and keyhole limpet hemocyanin (KLH) pulsed dendritic cell (DC) vaccine.

Procedure: Autologous Hematopoietic Progenitor Cell Transplant; Autologous Hematopoietic Progenitor Cell (HPC) Transplant (HPCT). Blood stem cells will be collected by apheresis during the induction phase as part of standard treatment. During apheresis, the participant's blood is collected into a machine that filters out the stem cells and the filtered blood is returned to their body. The stem cells will be separated by the type of protein within the cells. Only the stem cells with a protein called CD34 will be used for the stem cell transplant.; Other Names: stem cell transplant; Biological: KLH and Tumor Lysate Pulsed DC Vaccine; Keyhole limpet hemocyanin (KLH) pulsed dendritic cell (DC) vaccine. Post-transplant vaccine days: +14, +28, +56 (± 10 days), +84 (± 10 days). Vaccines will be administered by intradermal injection of 0.5 mL at two nodal basins.; Other Names: Dendritic Cell (DC) Vaccine

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Sufficient Tumor Cell Lysate and Dendritic Cells	The feasibility of manufacturing both a hematopoietic progenitor cell graft and multiple tumor lysate pulsed dendritic cell vaccine treatments from the same starting apheresis product, culminating in delivery of the vaccines in the immediate period following myeloablative therapy and autologous hematopoietic progenitor cell transplant period (autoHPCT). For what fraction of eligible patients can sufficient tumor cell lysate and dendritic cells, necessary for the production of the dendritic cell vaccines, be obtained? From what fraction would it be possible to make additional vaccines?	Up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Dendritic Cell Related Adverse Events	Toxicities resulting from the administration of dendritic cell vaccines in the immediate post hematopoietic cell graft period.	Up to 1 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Anti-tumor Effect	Whether a discernable anti-tumor effect resulting from autoHPCT therapy combined with dendritic cell vaccine therapy can be detected, either through monitoring of the patient's immune system for evidence of tumor specific immunity, or by monitoring for measureable clinical responses.	Up to 1 year

Collaborators and Investigators

• Sponsor: H. Lee Moffitt Cancer Center and Research Institute
• Collaborators: Johns Hopkins All Children's Hospital
• Investigators: Principal Investigator: Shari Pilon-Thomas, Ph.D., H. Lee Moffitt Cancer Center and Research Institute; Principal Investigator: Gregory Hale, M.D., Johns Hopkins All Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 14, 2016
• Primary Completion (Actual): August 21, 2017
• Study Completion (Actual): August 21, 2017

Study Registration Dates

• First Submitted: April 18, 2016
• First Submitted That Met QC Criteria: April 18, 2016
• First Posted (Estimate): April 20, 2016

Study Record Updates

• Last Update Posted (Actual): August 30, 2017
• Last Update Submitted That Met QC Criteria: August 29, 2017
• Last Verified: August 1, 2017

More Information

Terms related to this study

• Keywords: Cancer; Children; High-risk neuroblastoma
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma; Neoplasms, Nerve Tissue; Physiological Effects of Drugs; Immunologic Factors; Vaccines
• Other Study ID Numbers: MCC-17181