Clinical trial · Interventional
Autologous Transplant Followed by Allogeneic Transplant for High Risk Neuroblastoma
A Pilot Study of Sequential Autologous Stem Cell Transplantation and Immunotherapy With Reduced Intensity Allogeneic Stem Cell Transplant for 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): Poor accrual
Summary
Brief summary (as posted)
Neuroblastoma is a malignant tumor of the sympathetic nervous system. It is the second most common malignant tumor of childhood. Although modest advances have been made over the past 20 years children with high-risk neuroblastoma continue to have an unsatisfactory long-term survival. This study will administer induction chemotherapy followed by high-dose (myeloablative) chemotherapy with autologous stem cell transplantation, followed by radiation therapy, then immunotherapy with a non myeloablative allogeneic stem cell transplant for treatment of neuroblastoma. The purpose of this clinical research trial is to study the feasibility of giving immunotherapy with a non-myeloablative allogeneic transplant (NAT/AlloSCT), following myeloablative therapy and autologous stem cell transplant (MAT/AutoSCT). This study will also determine the side effects as well as the response rate for each group of patients (treatment arm).
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 (5)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Busulfan | Drug | Busulfan | ALIAS |
| Cord blood transplant | Procedure | — | UNRESOLVED |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Related donor transplant | Procedure | — | UNRESOLVED |
| Thymoglobulin | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Arm A - Related Donor
- description
- Related donor transplant: Patients with a related donor (5/6 or 6/6 HLA matched) will receive immunotherapy with a non-myeloablative preparative regimen of Busulfan and Fludarabine followed by allogeneic stem cell transplant (AlloSCT).
- interventionNames
- Procedure: Related donor transplant
- Drug: Busulfan
- Drug: Fludarabine
- type
- EXPERIMENTAL
- label
- Arm B - Cord Blood Donor
- description
- Unrelated cord blood transplant: Patients without a related donor will receive immunotherapy with a non-myeloablative preparative regimen of busulfan and fludarabine followed by allogeneic stem cell transplant (AlloSCT) with either an unrelated umbilical cord blood donor (4/6, 5/6, or 6/6 HLA matched), or a related umbilical cord blood donor (3/6, 4/6, 5/6, or 6/6 HLA matched). Patients will receive Thymoglobulin ((rabbit) Anti-Thymocyte Globulin (ATG)) during the preparative regimen. GVHD prophylaxis will be Tacrolimus and mycophenolate mofetil (MMF).
- interventionNames
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 30 Years
Show eligibility criteria text
Inclusion Criteria: * Age be \< 30 years of age at the time of initial diagnosis. * Patients must have a diagnosis of neuroblastoma (ICD-O morphology 9500/3) verified by histology and/or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Patients with the following disease stages at diagnosis are eligible, if they meet the other specified criteria. The revised International Neuroblastoma Staging System (INSS) will be used to stage all patients 58. (See 14.3 for risk assignment). * Patients with newly diagnosed neuroblastoma and age \> 547 days with the following: * INSS Stage 4 neuroblastoma regardless of biologic factors * INSS Stage 2A/2B with MYCN amplification (\> 10) * INSS Stage 3 with MYCN amplification (\> 10) OR Unfavorable histology * Patients with newly diagnosed neuroblastoma and age \< 365 days with the following: \* INSS Stage 3, 4, OR 4S neuroblastoma AND MYCN amplification (\> 10). * Patients with newly diagnosed Neuroblastoma and age 365 - \<547 days with the following: * INSS Stage 3 with MYCN amplification (\> 10) * INSS Stage 4 with MYCN amplification (\> 10) OR with deoxyribonucleic acid (DNA) Index (ploidy) = 1 OR with Unfavorable histology * Patients \> 365 days with INSS Stage 1, 2, and 4S who have progressed to Stage 4. * Newly Diagnosed patients should be entered on this study within 4 weeks of diagnosis, or after receiving only one cycle of intermediate dose chemotherapy for patients initially treated on/according to the low or intermediate risk Children's Oncology Group (COG) neuroblastoma studies, or within 4 weeks of progression to Stage 4 for INSS Stage 1, 2, 4S. * Patients treated with alternative induction regimens and/or consolidation regimens (AutoSCT) who were not enrolled at diagnosis but who achieve a complete response (CR), very good partial response (VGPR), partial response (PR), or minimal response (MR) and meet all other criteria will be eligible for either the consolidation MAT/AutoSCT and NAT/AlloSCT immunotherapy or NAT/AlloSCT, which ever is clinically appropriate after discussion with the Principal Investigators. * Liver Function: alanine aminotransferase (ALT) and bilirubin \< 3x normal * Cardiac Function: Shortening fraction \> 27%, or ejection fraction \> 47%, no clinical congestive heart failure. * Renal Function: Creatinine clearance and/or glomerular filtration rate (GFR) \> 60 ml/min/1.73m2. * Hematologic Function: Patients must have adequate hematopoietic function (absolute neutrophil count (ANC) \> 1000/mm3 and platelets \> 100,000/mm3) unless inadequate hematopoiesis documented to be due to bone marrow involvement with tumor (\> 10% tumor infiltration). Exclusion Criteria: * Patients who are pregnant. Patients of childbearing potential must practice an effective method of birth control while participating on this study.
References
Publications (0)
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