Study of Donor Derived, Multi-virus-specific, Cytotoxic T-Lymphocytes for Relapsed/Refractory Neuroblastoma (STALLONe)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01460901|
Recruitment Status : Completed
First Posted : October 27, 2011
Results First Posted : July 23, 2019
Last Update Posted : July 23, 2019
|Condition or disease||Intervention/treatment||Phase|
|Neuroblastoma||Biological: GD2 CAR modified Tri-virus specific cytotoxic t-cells||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I Study of Donor Derived,Gene Modified, Multi-virus-specific, Cytotoxic T-Lymphocytes Redirected to GD2 for Relapsed/Refractory Neuroblastoma Post-allo Stem Cell Transplantation With Submyeloblative Conditioning|
|Study Start Date :||October 2012|
|Actual Primary Completion Date :||January 2015|
|Actual Study Completion Date :||January 2015|
Experimental: GD2 CAR modified Tri-virus CTL infusion
A single infusion of 2x10e6 cells per meter squared was performed 30 to 120 days following allogeneic stem cell transplant.
Biological: GD2 CAR modified Tri-virus specific cytotoxic t-cells
This is a feasibility study to assess safety of an infusion of chimeric-antigen receptor gene modified allogeneic virus specific T lymphocytes after reduced intensity allogeneic stem cell transplant. Three patients were treated and safety was evaluated. Patients received a single infusion of 2x10e6/m2 donor derived, GD2 CAR modified, tri-virus specific CTL performed 30-120 days after allogeneic stem cell transplantation
- Number of Participants With Immediate and Short Term Toxicity of Infusion Over 8 Weeks [ Time Frame: Post infusion week 8 ]
Immediate: Patients were monitored following infusion to assess for toxicity related to infusion. Potential toxicities related to cellular therapy infusions, such as allergic reaction to the cellular product or cryopreservation media, hemolytic reactions, volume overload, and hemodynamic instability, were monitored.
Short Term: Patients were monitored for 8 weeks for short term toxicity related to infusion. Such adverse reactions monitored were acute graft versus host disease and cytokine release syndrome.
- Peak Transgene Copy Number Per 1000ng PBMC DNA [ Time Frame: 1 year ]Peak Transgene Copy Number per 1000ng PBMC DNA from peripheral blood samples measured during study participation.
- Death Within 8 Weeks of Infusion [ Time Frame: 8 weeks ]
- Peak Viral Specific SFU/2x10e5 Mononuclear Cells Per Well [ Time Frame: up to 1 year ]
The following analyses were performed on peripheral blood samples from patients at protocol assigned time points (pre-infusion, post-infusion at 4 hrs, weeks 1,2,4,6 and 8, month 3, 6 and 12:
ELISPOT assay for CMV, Adenovirus and EBV specific CTL reported as SFU (spot forming unit) per 2x10e5 mononuclear cells
- Maximum Tumor Response (RECIST 1.1) [ Time Frame: 1 year ]Pre and post-therapy evaluation by modalities consistent with prior disease evaluation in each patient. When possible, tumors were assessed per Response Evaluation Criteria In Solid Tumors (RECIST v1.0): Complete Response (CR), disappearance of all target lesions; Partial Response (PR), >/=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease (PD) at least a 20% increase in the sum of diameters of target lesions; Stable Disease (SD) neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD. Bone marrow aspirations and biopsies were evaluated by histopathology and appropriate immunohistochemistry; Modified Curie score was used for MIBG evaluation.
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): NCT01460901
|United States, Missouri|
|Children's Mercy Hospital|
|Kansas City, Missouri, United States, 64108|
|Principal Investigator:||Doug Myers, MD||Children's Mercy Hospital Kansas City|