Clinical trial · Interventional
Haploidentical Stem Cell Transplantation in Neuroblastoma
High-dose MIBG With Subsequent Transplantation of Haploidentical Stem Cells in Children With Therapy Resistant Neuroblastoma
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Children with primary resistant or relapsed neuroblastoma who do not achieve remission with conventional chemotherapy have extremely dismal prognosis. A novel treatment strategy combining tumor targeted radioisotope treatment with metaiodobenzylguanidine (MIBG) and immunotherapeutic effect of haploidentical stem cell transplantation (haploSCT) followed by low-dose donor lymphocyte infusions will be piloted. The use of the isotope is aimed to decrease pre-transplant tumour burden. Reduced intensity conditioning containing Fludarabine, Thiotepa and Melfalan will enable sustained engraftment as well as will serve as additional anti-tumor treatment. A prompt natural killer (NK)-cell mediated tumour control may be achieved by haploidentical stem cell transplantation. The investigators hypothesize that tumour cells potentially evading NK-cell mediated immunity may be targeted by infused donor T-cells and eliminated by either MHC-dependent manner or through a bystander effect. The possible graft versus tumor effect will be evaluated in children with therapy resistant neuroblastoma.
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 (8)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Co-transplantation of mesenchymal stem cells | Procedure | — | UNRESOLVED |
| Donor Lymphocyte Infusion | Procedure | — | UNRESOLVED |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Haploidentical stem cell transplantation | Procedure | — | UNRESOLVED |
| iodine I 131 metaiodobenzylguanidine | Drug | — | UNRESOLVED |
| Rituximab | Drug | Rituximab | ALIAS |
| T-cell depletion | Procedure | — | UNRESOLVED |
| Thiotepa | Drug | Thiotepa | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- High-dose MIBG with haploidentical stem cell transplantation
- description
- High-dose MIBG followed by Fludarabine, Thiotepa and Melfalan as conditioning Before haploidentical transplantation of T-cell depleted graft
- interventionNames
- Drug: iodine I 131 metaiodobenzylguanidine
- Drug: Fludarabine
- Drug: Thiotepa
- Procedure: T-cell depletion
- Procedure: Haploidentical stem cell transplantation
- Procedure: Donor Lymphocyte Infusion
- Drug: Rituximab
- Procedure: Co-transplantation of mesenchymal stem cells
Primary outcomes (1)
- measure
- Engraftment rate
- timeFrame
- day 100
Secondary outcomes (3)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 Months
- Maximum age
- 21 Years
Show eligibility criteria text
Inclusion Criteria: * Refractory neuroblastoma (any chemo/radiosensitive stable disease) * Relapse incl. autologous HSCT 3 m earlier * Primary induction failure * Cardiac output SF ≥25% * Creatinine clearance ≥40 cc/min/1.73 m2 * Performance score of ≥50% (Lansky or Karnofsky) * Available haploidentical family donor, aged ≥18 yrs, HIV-neg Exclusion Criteria: * Rapidly progressive disease * Pregnancy
References
Publications (8)
- BACKGROUNDInoue M, Nakano T, Yoneda A, Nishikawa M, Nakayama M, Yumura-Yagi K, Sakata N, Yasui M, Okamura T, Kawa K. Graft-versus-tumor effect in a patient with advanced neuroblastoma who received HLA haplo-identical bone marrow transplantation. Bone Marrow Transplant. 2003 Jul;32(1):103-6. doi: 10.1038/sj.bmt.1704070. PMID 12815485
- BACKGROUNDMatthay KK, Tan JC, Villablanca JG, Yanik GA, Veatch J, Franc B, Twomey E, Horn B, Reynolds CP, Groshen S, Seeger RC, Maris JM. Phase I dose escalation of iodine-131-metaiodobenzylguanidine with myeloablative chemotherapy and autologous stem-cell transplantation in refractory neuroblastoma: a new approaches to Neuroblastoma Therapy Consortium Study. J Clin Oncol. 2006 Jan 20;24(3):500-6. doi: 10.1200/JCO.2005.03.6400. PMID 16421427
- BACKGROUNDPrigione I, Corrias MV, Airoldi I, Raffaghello L, Morandi F, Bocca P, Cocco C, Ferrone S, Pistoia V. Immunogenicity of human neuroblastoma. Ann N Y Acad Sci. 2004 Dec;1028:69-80. doi: 10.1196/annals.1322.008. PMID 15650233
- BACKGROUNDNeal ZC, Imboden M, Rakhmilevich AL, Kim KM, Hank JA, Surfus J, Dixon JR, Lode HN, Reisfeld RA, Gillies SD, Sondel PM. NXS2 murine neuroblastomas express increased levels of MHC class I antigens upon recurrence following NK-dependent immunotherapy. Cancer Immunol Immunother. 2004 Jan;53(1):41-52. doi: 10.1007/s00262-003-0435-2. Epub 2003 Sep 18. PMID 14504825
- BACKGROUNDRaffaghello L, Prigione I, Bocca P, Morandi F, Camoriano M, Gambini C, Wang X, Ferrone S, Pistoia V. Multiple defects of the antigen-processing machinery components in human neuroblastoma: immunotherapeutic implications. Oncogene. 2005 Jul 7;24(29):4634-44. doi: 10.1038/sj.onc.1208594. PMID 15897905
- BACKGROUNDLang P, Pfeiffer M, Muller I, Schumm M, Ebinger M, Koscielniak E, Feuchtinger T, Foll J, Martin D, Handgretinger R. Haploidentical stem cell transplantation in patients with pediatric solid tumors: preliminary results of a pilot study and analysis of graft versus tumor effects. Klin Padiatr. 2006 Nov-Dec;218(6):321-6. doi: 10.1055/s-2006-942256.