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Comparing Two Different Myeloablation Therapies in Treating Young Patients Who Are Undergoing a Stem Cell Transplant for High-Risk Neuroblastoma
Phase 3 trial testing Autologous Hematopoietic Stem Cell Transplantation in Localized Resectable Neuroblastoma in 665 participants. Completed in 31 March 2022.
| Lead sponsor | Children's Oncology Group |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 665 |
| Start date | 5 November 2007 |
| Primary completion | 27 February 2015 |
| Estimated completion | 31 March 2022 |
| Sites | 190 locations across New Zealand, Canada, Puerto Rico, Australia, Switzerland, United States |
Children's Oncology Group — full company profile →
Under 30, any sex, with Localized Resectable Neuroblastoma or Localized Unresectable Neuroblastoma. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Comparison of EFS curves, starting from the time of randomization, by treatment group (single CEM vs. tandem CEM)
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 48.8 | 41.1 – 56.5 |
| Tandem HST (CEM), Randomly Assigned | 61.8 | 53.8 – 69.8 |
Per the International Response Criteria: measurable tumor defined as product of longest x widest perpendicular diameter. Elevated catecholamine levels, tumor cell invasion of bone marrow also considered measurable tumor. Complete Response (CR)-no evidence of primary tumor or metastases. Very Good Partial Response (VGPR)-\>90% reduction of primary tumor; no metastases; no new bone lesions, all pre-existing lesions improved. Partial Response (PR)-50-90% reduction of primary tumor; \>50% reduction in measurable sites of metastases; 0-1 bone marrow samples with tumor; number of positive bone sites
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 0.54 | 0.47 – 0.61 |
| Tandem HST (CEM), Randomly Assigned | 0.48 | 0.41 – 0.56 |
| Not Assigned | 0.35 | 0.29 – 0.41 |
Cumulative incidence rate of local recurrence comparison between ANBL0532 patients randomized or assigned to receive single CEM transplant and boost radiation versus the historical A3973 patients who were transplanted and received boost radiation.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 15.7 | 7.5 – 23.9 |
A logistic regression model will be used to test the ability of the number of days of neutropenia to predict the presence of a polymorphism.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 7 | 0 – 20 |
| Tandem HST (CEM), Randomly Assigned | 7 | 0 – 39 |
| Not Assigned | 7 | 0 – 35 |
A logistic regression model will be used to test the ability of the number of days of thrombocytopenia to predict the presence of a polymorphism.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 4 | 0 – 30 |
| Tandem HST (CEM), Randomly Assigned | 4 | 0 – 46 |
| Not Assigned | 4 | 0 – 44 |
A chi-square test will be used to test whether the response rate after two cycles of induction therapy and the presence of a polymorphism are independent in the study population.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 0.96 | |
| Tandem HST (CEM), Randomly Assigned | 0.96 | |
| Not Assigned | 0.97 |
Percentage of patients who achieved a surgical complete resection
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 83.98 | |
| Tandem HST (CEM), Randomly Assigned | 84.09 | |
| Not Assigned | 58.89 |
The Percentage of patients who experienced surgical or radiotherapy complications will be calculated. The complications are: bowel obstruction, chylous leaf, renal injury/atrophy/loss and diarrhea.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 13.11 | |
| Tandem HST (CEM), Randomly Assigned | 12.50 | |
| Not Assigned | 11.85 |
Percentage of patients with primary tumors with intraspinal extension.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 8.25 | |
| Tandem HST (CEM), Randomly Assigned | 9.66 | |
| Not Assigned | 7.78 |
Median peak serum concentration level of isotretinoin for patients enrolled on ANBL0532
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 1.00 | 0 – 9.22 |
| Tandem HST (CEM), Randomly Assigned | 1.36 | 0 – 12.93 |
| Not Assigned | 1.26 | 0 – 11.78 |
Median topotecan systemic clearance for courses 1 and 2.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 28.1 | 14.9 – 49.4 |
| Tandem HST (CEM), Randomly Assigned | 28.1 | 11.6 – 42.7 |
| Not Assigned | 28.5 | 16.3 – 54.7 |
A descriptive comparison of the median number of T-cells (CD3, CD4, CD8) between treatment arms (single vs. tandem myeloablative regimens) will be performed.
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 200 | 23 – 1549 |
| Tandem HST (CEM), Randomly Assigned | 255.5 | 13 – 2987 |
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 73 | 8 – 626 |
| Tandem HST (CEM), Randomly Assigned | 81 | 4 – 1186 |
| Group | Value | 95% CI |
|---|---|---|
| Single HST (CEM) | 104 | 8 – 1170 |
| Tandem HST (CEM), Randomly Assigned | 151 | 4 – 2529 |
Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Single HST (CEM) | Tandem HST (CEM), Randomly… | Not Assigned |
|---|---|---|---|---|
| 55700-Multi-organ failure | General disorders | — | — | — |
| 44800-Infections and infestations - Other specify | Infections and infestations | — | — | — |
| 17400-Blood bilirubin increased | Investigations | — | — | — |
| 43900-Hypoxia | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 17200-Blood and lymphatic system disorders - Other specify | Blood and lymphatic system disorders | — | — | — |
| 20000-Cardiac arrest | Cardiac disorders | — | — | — |
| 11600-Alanine aminotransferase increased | Investigations | — | — | — |
| 15000-Aspartate aminotransferase increased | Investigations | — | — | — |
| 24100-Creatinine increased | Investigations | — | — | — |
| 11100-Acute kidney injury | Renal and urinary disorders | — | — | — |
| 13200-Anemia | Blood and lymphatic system disorders | — | — | — |
| 25800-Disseminated intravascular coagulation | Blood and lymphatic system disorders | — | — | — |
| 63100-Pericardial effusion | Cardiac disorders | — | — | — |
| 14900-Ascites | Gastrointestinal disorders | — | — | — |
| 55600-Mucositis oral | Gastrointestinal disorders | — | — | — |
| 24600-Death NOS | General disorders | — | — | — |
| 41800-Hypernatremia | Metabolism and nutrition disorders | — | — | — |
| 43100-Hypokalemia | Metabolism and nutrition disorders | — | — | — |
| 65900-Pleural effusion | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 71500-Respiratory failure | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 71600-Respiratory thoracic and mediastinal disorders - Other specify | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 33300-Febrile neutropenia | Blood and lymphatic system disorders | — | — | — |
| 39600-Hemolysis | Blood and lymphatic system disorders | — | — | — |
| 39700-Hemolytic uremic syndrome | Blood and lymphatic system disorders | — | — | — |
| 51700-Left ventricular systolic dysfunction | Cardiac disorders | — | — | — |
| Reaction | System | Single HST (CEM) | Tandem HST (CEM), Randomly… | Not Assigned |
|---|---|---|---|---|
| 58300-Neutrophil count decreased | Investigations | — | — | — |
| 65800-Platelet count decreased | Investigations | — | — | — |
| 33300-Febrile neutropenia | Blood and lymphatic system disorders | — | — | — |
| 44800-Infections and infestations - Other specify | Infections and infestations | — | — | — |
| 43100-Hypokalemia | Metabolism and nutrition disorders | — | — | — |
| 88500-White blood cell decreased | Investigations | — | — | — |
| 13200-Anemia | Blood and lymphatic system disorders | — | — | — |
| 25700-Diarrhea | Gastrointestinal disorders | — | — | — |
| 55600-Mucositis oral | Gastrointestinal disorders | — | — | — |
| 15000-Aspartate aminotransferase increased | Investigations | — | — | — |
| 11600-Alanine aminotransferase increased | Investigations | — | — | — |
| 73700-Sepsis | Infections and infestations | — | — | — |
| 13500-Anorexia | Metabolism and nutrition disorders | — | — | — |
| 53700-Lymphocyte count decreased | Investigations | — | — | — |
| 41400-Hyperglycemia | Metabolism and nutrition disorders | — | — | — |
| 42700-Hypocalcemia | Metabolism and nutrition disorders | — | — | — |
| 43300-Hyponatremia | Metabolism and nutrition disorders | — | — | — |
| 43600-Hypotension | Vascular disorders | — | — | — |
| 44600-Ileus | Gastrointestinal disorders | — | — | — |
| 87900-Vomiting | Gastrointestinal disorders | — | — | — |
| 43900-Hypoxia | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 24700-Dehydration | Metabolism and nutrition disorders | — | — | — |
| 41600-Hyperkalemia | Metabolism and nutrition disorders | — | — | — |
| 38900-Hearing impaired | Ear and labyrinth disorders | — | — | — |
| 10300-Abdominal pain | Gastrointestinal disorders | — | — | — |
| 57600-Nausea | Gastrointestinal disorders | — | — | — |
| 20500-Catheter related infection | Infections and infestations | — | — | — |
| 65900-Pleural effusion | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 42100-Hypertension | Vascular disorders | — | — | — |
| 59700-Oral pain | Gastrointestinal disorders | — | — | — |
| 37500-GGT increased | Investigations | — | — | — |
| 33900-Fever | General disorders | — | — | — |
| 60600-Pain | General disorders | — | — | — |
| 29500-Enterocolitis infectious | Infections and infestations | — | — | — |
| 53100-Lung infection | Infections and infestations | — | — | — |
| 41300-Hypercalcemia | Metabolism and nutrition disorders | — | — | — |
| 43500-Hypophosphatemia | Metabolism and nutrition disorders | — | — | — |
| 71600-Respiratory thoracic and mediastinal disorders - Other specify | Respiratory, thoracic and mediastinal disorders | — | — | — |
| 14900-Ascites | Gastrointestinal disorders | — | — | — |
| 75700-Small intestinal obstruction | Gastrointestinal disorders | — | — | — |
Most-reported serious reactions: 55700-Multi-organ failure, 44800-Infections and infestations - Other specify, 17400-Blood bilirubin increased, 43900-Hypoxia, 17200-Blood and lymphatic system disorders - Other specify, 20000-Cardiac arrest, 11600-Alanine aminotransferase increased, 15000-Aspartate aminotransferase increased.
Data from ClinicalTrials.gov NCT00567567 adverse events section.
This randomized phase III trial compares two different high-dose chemotherapy regimens followed by a stem cell transplant in treating younger patients with high-risk neuroblastoma. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving these treatments before a peripheral blood stem cell transplant helps kill any tumor cells that are in the body and helps make room in the patient?s bone marrow for new blood-forming cells (stem cells) to grow. After treatment, stem cells are collected from the patient's blood and stored. High-dose chemotherapy and radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the high- chemotherapy. It is not yet known which regimen of high-dose chemotherapy is more effective for patients with high-risk neuroblastoma undergoing a peripheral blood stem cell transplant.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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