Phase 1
N=5
Study of Donor Derived, Multi-virus-specific, Cytotoxic T-Lymphocytes for Relapsed/Refractory Neuroblastoma
Neuroblastoma
Bottom Line
View on ClinicalTrials.gov: NCT01460901 ↗Enrolled (actual)
5
Serious AEs
100.0%
Results posted
Jul 2019
Primary outcome: Primary: Number of Participants With Immediate and Short Term Toxicity of Infusion Over 8 Weeks — 0; 0; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- GD2 CAR modified Tri-virus specific cytotoxic t-cells (Biological)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Children's Mercy Hospital Kansas City
- Primary completion
- Jan 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Immediate and Short Term Toxicity of Infusion Over 8 Weeks |
0; 0; 3 | — |
| PRIMARY Peak Transgene Copy Number Per 1000ng PBMC DNA |
10562; 44; 1464 | — |
| PRIMARY Death Within 8 Weeks of Infusion |
1 | — |
| SECONDARY Peak Viral Specific SFU/2x10e5 Mononuclear Cells Per Well |
180; 175; 150 | — |
| SECONDARY Maximum Tumor Response (RECIST 1.1) |
0; 3; 0 | — |
Summary
This is a single-center, investigator-initiated, single-arm, pilot study of post-allogeneic transplant, adoptive immunotherapy for the treatment of patients with relapsed/refractory neuroblastoma expressing the mesenchymal tumor marker GD2. Three patients will be treated. The study will focus on the safety and efficacy of allogeneic, donor derived viral specific cytotoxic T-lymphocytes, retrovirally transduced to express a chimeric antigen receptor specific for disialoganglioside, GD2, expressed on neuroblastoma.
Eligibility Criteria
Inclusion Criteria
- Allogeneic transduced tV-CTLs with >15% expression of 14g2a.zeta chimeric antigen receptor
- Patient or responsible person must be able to understand and sign a permission/assent or consent form for infusion
- Age 18 months through 17 years at time of relapse/progression
- Life expectancy >8weeks
- Karnofsky score 60% or greater if 10 yrs old or older. Lansky score 60% or greater if under 10 yrs old
- Patient must be HIV negative
- ANC >500
- Pulse ox>90% on room air
- AST/ALT/direct bili 50% donor engraftment
Exclusion Criteria
- Patient pregnant or lactating or refuses birth control methods
- HIV positive
- Uncontrolled intercurrent infection
- Renal failure (creatinine clearance 5xnormal
- Rapidly progressive disease
- Currently receiving any investigational drugs
- Tumor potentially causing airway obstruction
- Cardiomegaly or bilateral pulmonary infiltrates on CXR
- Receiving >0.25mg/kg/day methylprednisolone or equivalent systemic steroid. Topical steroid therapy is acceptable
- Receiving more than one lymphocyte inhibiting agent (ex. Tacrolimus/CSA and MMF or other similar agent
- Patients relapsing or progressing before the age of 18 months from Stage I/II disease, and/or those who, in the opinion of their oncologist, may benefit from further conventional therapy
- Donor lymphocyte infusion in last 28 days
- Evidence of GvHD greater than or equal to grade 2
Data sourced from ClinicalTrials.gov (NCT01460901). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.