A Phase 2 interventional study of anti-thymocyte globulin and busulfan in Neuroblastoma, sponsored by Nationwide Children's Hospital. Withdrawn at 4 sites in United States. Open to participants aged 1 Year to 18 Years. Per ClinicalTrials.gov, last updated 2015-10-16.
Sponsored by Nationwide Children's Hospital · Phase 2, Interventional, and Treatment
RATIONALE: - Relapsed or refractory Neuroblastoma (NBL) carries a very poor prognosis and children with relapsed NBL have an overall 3 year survival rate of \< 10%. Hematopoietic Stem Cell Transplant from a different donor (allogeneic), is a form of adoptive cellular therapy , such that infused donor cells find host tumors as foreign and fight them. After transplant, the donor immune cells (i.e. T cells, NK cells) mediate Graft versus Tumor (GVT) effect and may stop tumor from recurring. Also,reduced intensity transplants lead to minimal toxicity and less risk of mortality in heavily pre-treated NBL patients.
PURPOSE: This phase II trial is studying how well giving a reduced intensity(using Fludarabine, Busulfan and antithymocyte globulin)preparative regimen followed by donor stem cell transplant works in treating young patients with high-risk neuroblastoma that has relapsed or not responded to treatment.
OBJECTIVES:
Primary
Secondary
OUTLINE: This is a multicenter study.
Blood samples are collected at baseline and on days 30, 60, and 100 for correlative laboratory studies. Samples are analyzed for killer immunoglobulin-like receptor (KIR) mismatches by genotyping and immunophenotyping methods (PCR and flow cytometry); natural killer (NK) cell reconstitution by flow cytometry; and NK cell function, NK cell allo-reactivity by ELISPOT and ELISA.
After completion of study treatment, patients are followed periodically for 1 year.
625 studies on the registry are indexed under Neuroblastoma; 122 are open to participants now.
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DISEASE CHARACTERISTICS:
Diagnosis of high-risk neuroblastoma, meeting one of the following criteria:
Achieved a complete remission (CR), very good partial remission (VGPR), or partial remission (PR) after ≤ 2 different salvage regimens, as defined by the following:
In VGPR or PR after salvage therapy
Disease status meeting one of the following criteria:
Donors must meet one of the following criteria:
PATIENT CHARACTERISTICS:
PRIOR CONCURRENT THERAPY:
2.5 mg/kg/day for 4 doses on day -3, -2 , -1 and day +2.
Also known as: Thymoglobulin
0.8 mg/kg/dose for total of 8 doses.
Also known as: Busulfex
1.5 mg/kg/dose every 12 hours.
Also known as: Sandimmune, Gengraf, Neoral.
30 mg/m2/day for 5 days.
Also known as: Fludara
15 mg/kg/dose every 8 hours
Also known as: Cell-cept
0.03 mg/kg/day as continuous infusion or 12 hour divided doses
Donor stem cell transplantation from HLA matched sibling donor or an unrelated donor.
Feasibility as measured by the incidence of donor engraftment, transplant-related mortality, and grade III-IV acute graft-vs-host disease (GVHD) at day 100 and the incidence of extensive chronic GVHD within the first year post-transplantation
The safety and feasibility of reduced intensity allogeneic HSCT will be established in this population by monitoring the incidence of adverse events- 100 day mortality,incidence of severe acute GVHD and non-engraftment of donor cells.
Time frame: 100 days post-HSCT
Progression-free survival (PFS) at 1 year
Time frame: 1 year post- HSCT
Relationship between biologic endpoints (e.g., number of natural killer [NK] cells infused, NK cell recovery, and NK cell chimerism status) and clinical endpoints (e.g., donor engraftment, acute GVHD, transplant-related mortality, and PFS)
Time frame: 3 and 6 months post-SCT
Relationship between presence of killer immunoglobulin-like receptor (KIR) mismatches and clinical endpoints
Time frame: 3 and 6 months post-HSCT
This study is withdrawn, as verified in Oct 2015. You cannot join it, but the record below documents what was studied.
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