Clinical trial · Interventional
Allogeneic Hematopoietic Stem Cell Transplantation for Relapsed or Refractory High-Risk NBL.
A Multicenter Pilot Study of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation With In-vivo T-cell Depletion to Evaluate the Role of NK Cells and KIR Mis-matches in Relapsed or Refractory High-risk Neuroblastoma.
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): lack of accrual
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (7)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| allogeneic hematopoietic stem cell transplantation | Procedure | — | UNRESOLVED |
| anti-thymocyte globulin | Other | — | UNRESOLVED |
| busulfan | Drug | Busulfan | ALIAS |
| cyclosporine | Drug | — | UNRESOLVED |
| fludarabine phosphate | Drug | Fludarabine | ALIAS |
| mycophenolate mofetil | Drug | — | UNRESOLVED |
| tacrolimus | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- 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
- timeFrame
- 100 days post-HSCT
- description
- 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.
Secondary outcomes (3)
- measure
- Progression-free survival (PFS) at 1 year
- timeFrame
- 1 year post- HSCT
- measure
- 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)
- timeFrame
- 3 and 6 months post-SCT
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
- Maximum age
- 18 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of high-risk neuroblastoma, meeting one of the following criteria:
* Refractory disease, defined as no response, mixed response, or progressive disease after completion of induction therapy administered according to clinical trials COG-A3973 or COG-ANBL0532 (or other similar high-intensity induction regimen)
* Relapsed following high-dose chemoradiotherapy including autologous stem cell transplantation
* 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 CR after treatment with some form of salvage therapy (e.g., ¹³¹I-MIBG, antibody-based therapy, or any other COG or NANT salvage-therapy regimen)
* In VGPR or PR after salvage therapy
* No more than 3 sites of skeletal disease as determined by an ¹²³I-MIBG scan (for regional involvement of the skeleton \[e.g., pelvis, spine\], the tumor involvement should be \< 25% of the site)
* Bone marrow involvement (\< 25% neuroblasts) by morphologic exam within the past 2 weeks
* Patients with soft tissue disease are eligible provided they exhibit either a VGPR or PR in the primary soft tissue mass and in any sites of metastatic soft tissue disease
* Disease status meeting one of the following criteria:
* Minimal residual disease
* Disease considered responsive to a salvage regimen
* Stable disease
* No rapidly progressive disease
* Donors must meet one of the following criteria:
* Matched, related donor (6/6 or 5/6) (bone marrow donor allowed)
* HLA-matched unrelated donor (10/10 match on high-resolution \[HR\] typing of HLA-A, B, C, DRB1, and DQB1)
* One allele- or antigen-mismatched unrelated donor (9/10 match on HR typing), mismatched at HLA-C only
* One allele- or antigen-mismatched unrelated donor (9/10 match on HR typing), mismatched at HLA-A, B, DRB1, or DQB1 (only when HLA-C mismatch is not available)
PATIENT CHARACTERISTICS:
* Karnofsky/Lansky performance status 60-100%
* ANC \> 500/mm\^3
* Creatinine clearance or radioisotope GFR ≥ 60 mL/min
* Total bilirubin \< 3.0 mg/dL
* AST or ALT \< 5 times upper limit of normal
* Shortening fraction ≥ 25% by ECHO OR ejection fraction \> 30% by MUGA
* FEV\_1 and DLCO ≥ 30% OR normal chest x-ray, pulse oximetry, and venous blood gas
* Negative pregnancy test
* Fertile patients must use effective contraception
* HIV negative
* No active or recent (within the past 30 days) fungal infection
* No proven or suspected sepsis, pneumonia, or meningitis unless appropriate therapeutic measures have been initiated to control the infection and systemic signs are no longer life-threatening
* No requirement for oxygen or ventilator support
PRIOR CONCURRENT THERAPY:
* See Disease Characteristics
* Prior tandem autologous stem cell transplantations (according to clinical trial COG-ANBL0532) allowed
* No prior allogeneic hematopoietic stem cell transplantation
* More than 2 months since prior autologous stem cell transplantation, myeloablative therapy, total-body irradiation, whole abdominal radiotherapy, or therapeutic ¹³¹I-MIBG
* More than 3 weeks since prior chemotherapy, immunotherapy (including anti-GD2 regimen), or biologic response modifiers and recovered
* More than 2 weeks since prior local radiotherapy to the sites of metastatic diseaseReferences
Publications (0)
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