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Phase III Randomized Trial of Single vs. Tandem Myeloablative Consolidation Therapy for High-Risk Neuroblastoma

Unique Dataset IDNCT00567567
SponsorChildren's Oncology GroupData ProviderNational Cancer InstituteTotal Study Enrolled Patients665RandomizationYesPubMed (PMID)31454045.0
Study PhaseClinical Study Phase IIIBlinding MethodOpen Label StudyType(s) of dataN/AIntervention TypeN/ADataset TypeOther

Clinical Trial Title

Phase III Randomized Trial of Single vs. Tandem Myeloablative Consolidation Therapy for High-Risk Neuroblastoma

Trial Summary and Conditions

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.

Data Summary

See Data Dictionary.

Study Objectives

Primary: To improve the 3-year event-free survival (EFS) rate of high-risk neuroblastoma patients through treatment with a tandem consolidation of thiotepa/cyclophosphamide followed by carboplatin/etoposide/melphalan (CEM) as compared to single CEM consolidation; To improve the rate of end-induction complete response and very good partial response, compared to historical controls, by use of a topotecan-containing induction regimen; To improve the 3-year local control rate, compared to historical controls, by increasing the local dose of radiation to the residual primary tumor for patients with less than a gross total resection. Secondary: To evaluate the pharmacogenetic relationship of cyclophosphamide metabolizing enzymes (CYP2B6, CYP2C9, and GSTA1 genotypes) with toxicity and response following dose-intensive cyclophosphamide and topotecan induction chemotherapy; To determine if resection completeness is predictive of a) local control rate; or b) EFS rate in patients with high-risk neuroblastoma; To prospectively describe the complications related to efforts at local control (surgery and radiation therapy) in patients with high-risk neuroblastoma; To describe the neurologic outcome of patients with paraspinal primary neuroblastoma tumors; To determine the variability of 13-cis-retinoic-acid pharmacokinetics and relationship to pharmacogenomic parameters and determine if pharmacokinetics and/or genetic variations correlate with EFS or systemic toxicity as follows: a) To determine the variability of 13-cis-retinoic-acid pharmacokinetics and relationship to pharmacogenomic parameters. b) To determine if 13-cis-retinoic-acid pharmacokinetic levels are predictive of the EFS rate or associated with systemic toxicity following 13-cis-retinoic acid. c) To determine if pharmacogenomic variations are predictive of the EFS rate or associated with systemic toxicity following 13-cis-retinoic acid; To evaluate total topotecan pharmacokinetics and correlate with patient specific data for use in an ongoing topotecan population pharmacokinetic analysis; To evaluate the presence and function of T cells capable of recognizing neuroblastoma by assessing: a) if T cells recognizing the neuroblastoma antigen, survivin, circulate at diagnosis; b) if these T cells can be expanded using autologous antigen presenting cells (APCs); c) if these T cells will kill neuroblastoma cells as detected in functional assays; and d) if the presence and activity of anti-neuroblastoma immunity is decreased by stem cell transplantation; To characterize the recovery of T- cell numbers after myeloablative consolidation and hematopoietic stem cell transplant (HSCT) and assess the impact of tandem myeloablative consolidation on T- cell recovery; To characterize minimal residual disease burden using reverse transcriptase-polymerase chain reaction (RT-PCR) evaluation of a panel of neuroblastoma specific transcripts in patient bone marrow and peripheral blood following induction chemotherapy and after single versus tandem myeloablative chemotherapy and to evaluate impact on EFS; To evaluate the EFS and overall survival (OS) rate for patients 12-18 months with Stage 4, MYCN nonamplified tumor with unfavorable histopathology or diploid DNA content or with indeterminant histology or ploidy and patients who are greater than 547 days of age with Stage 3, MYCN nonamplified tumor AND unfavorable histopathology or indeterminant histology following treatment with single myeloablative transplant.

Outcome Measures

Primary: Event-free Survival Rate; Response After Induction Therapy; Incidence Rate of Local Recurrence. Secondary: Duration of Greater Than or Equal to Grade 3 Neutropenia; Duration of Greater Than or Equal to Grade 3 Thrombocytopenia; Proportion of Patients With a Polymorphism; Surgical Response; Type of Surgical or Radiotherapy Complication; Intraspinal Extension; Peak Serum Concentration of Isotretinoin in Patients Enrolled on Either A3973, ANBL0032, ANBL0931, ANBL0532 and Future High Risk Studies; Pharmacogenetic Variants in Patients Enrolled on Either A3973, ANBL0032, ANBL0931, ANBL0532 and Future High Risk Studies; Topotecan Systemic Clearance; Presence and Function of T Cells Capable of Recognizing Neuroblastoma; Enumeration of Peripheral Blood Cluster of Differentiation (CD)3, CD4, and CD8 Cells; Proportion of Patients With Neuroblastoma Detected in Bone Marrow and Peripheral Blood Using RT-PCR Technique; EFS Pts Non-randomly Assigned to Single CEM (12-18 Mths, Stg. 4, MYCN

Below are the clinical trial(s) associated with this dataset (all links are to ClinicalTrials.Gov):

NCI Description

There is one dataset associated with the publication: NCT00567567-D1. USI is the patient identifier and blanks or "." represent missing data or not applicable for analyses. Data can be used to approximate published study findings, but exact reproduction of previous manuscripts may not be possible in some cases (e.g., when data must be modified for de-identification purposes or have undergone further data cleaning). NCT00567567-D1: There is one row for each patient enrolled on the study ANBL0532. The dataset provides the information necessary to reproduce the manuscript.

Available Downloads: NCT00567567-D1

To gain access to the data and analytic tools click here.

Data Dictionary: NCT00567567-D1-Data-Dictionary_2.pdf

DATA: NCT00567567-D1-Dataset_3.csv