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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00567567




Registration number
NCT00567567
Ethics application status
Date submitted
4/12/2007
Date registered
5/12/2007
Date last updated
28/04/2022

Titles & IDs
Public title
Comparing Two Different Myeloablation Therapies in Treating Young Patients Who Are Undergoing a Stem Cell Transplant for High-Risk Neuroblastoma
Scientific title
Phase III Randomized Trial of Single vs. Tandem Myeloablative Consolidation Therapy for High-Risk Neuroblastoma
Secondary ID [1] 0 0
NCI-2009-01065
Secondary ID [2] 0 0
ANBL0532
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Localized Resectable Neuroblastoma 0 0
Localized Unresectable Neuroblastoma 0 0
Recurrent Neuroblastoma 0 0
Regional Neuroblastoma 0 0
Stage 4 Neuroblastoma 0 0
Stage 4S Neuroblastoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Children's - Other

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - Autologous Hematopoietic Stem Cell Transplantation
Treatment: Drugs - Carboplatin
Treatment: Drugs - Cisplatin
Treatment: Drugs - Cyclophosphamide
Treatment: Drugs - Doxorubicin Hydrochloride
Treatment: Drugs - Etoposide
Treatment: Other - External Beam Radiation Therapy
Treatment: Other - Filgrastim
Treatment: Drugs - Isotretinoin
Other interventions - Laboratory Biomarker Analysis
Treatment: Drugs - Melphalan
Treatment: Surgery - Peripheral Blood Stem Cell Transplantation
Other interventions - Pharmacological Study
Treatment: Drugs - Thiotepa
Treatment: Drugs - Topotecan Hydrochloride
Treatment: Drugs - Vincristine Sulfate Liposome

Active comparator: Consolidation Arm A: single myeloablative consolidation - Patients receive melphalan IV over 15-30 minutes on days -7 to -5, etoposide IV over 24 hours and carboplatin IV over 24 hours on days -7 to -4, and G-CSF SC or IV beginning on day 0 and continuing until blood counts recover. Patients undergo autologous PBSCT on day 0.

Experimental: Consolidation Arm B: tandem myeloablative consolidation - Patients receive thiotepa IV over 2 hours on days -7 to -5, cyclophosphamide IV over 1 hour on days -5 to -2, and G-CSF SC or IV beginning on day 0 and continuing until blood counts recover. Following clinical recovery from initial myeloablative therapy, patients also receive melphalan, etoposide, and carboplatin as in Arm A. Patients undergo autologous PBSCT on day 0.


Treatment: Surgery: Autologous Hematopoietic Stem Cell Transplantation
Undergo autologous peripheral blood stem cell transplant

Treatment: Drugs: Carboplatin
Given IV

Treatment: Drugs: Cisplatin
Given IV

Treatment: Drugs: Cyclophosphamide
Given IV

Treatment: Drugs: Doxorubicin Hydrochloride
Given IV

Treatment: Drugs: Etoposide
Given IV

Treatment: Other: External Beam Radiation Therapy
Undergo EBRT

Treatment: Other: Filgrastim
Given IV or SC

Treatment: Drugs: Isotretinoin
Given orally

Other interventions: Laboratory Biomarker Analysis
Correlative studies

Treatment: Drugs: Melphalan
Given IV

Treatment: Surgery: Peripheral Blood Stem Cell Transplantation
Undergo autologous peripheral blood stem cell transplant

Other interventions: Pharmacological Study
Correlative studies

Treatment: Drugs: Thiotepa
Given IV

Treatment: Drugs: Topotecan Hydrochloride
Given IV

Treatment: Drugs: Vincristine Sulfate Liposome
Given IV

Intervention code [1] 0 0
Treatment: Surgery
Intervention code [2] 0 0
Treatment: Drugs
Intervention code [3] 0 0
Treatment: Other
Intervention code [4] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Event-free Survival Rate
Timepoint [1] 0 0
Three years, from time of randomization
Primary outcome [2] 0 0
Response After Induction Therapy
Timepoint [2] 0 0
Study enrollment to the end of induction therapy
Primary outcome [3] 0 0
Incidence Rate of Local Recurrence
Timepoint [3] 0 0
Up to 3 years
Secondary outcome [1] 0 0
Duration of Greater Than or Equal to Grade 3 Neutropenia
Timepoint [1] 0 0
Through completion of a participant's first cycle during induction, including treatment delays, assessed up to 39 days
Secondary outcome [2] 0 0
Duration of Greater Than or Equal to Grade 3 Thrombocytopenia
Timepoint [2] 0 0
Through completion of a participant's first cycle during induction, including treatment delays, assessed up to 46 days
Secondary outcome [3] 0 0
Proportion of Patients With a Polymorphism
Timepoint [3] 0 0
Through completion of a participant's first two cycles during induction, including treatment delays, assessed up to 69 days
Secondary outcome [4] 0 0
Surgical Response
Timepoint [4] 0 0
Up to 3 years
Secondary outcome [5] 0 0
Type of Surgical or Radiotherapy Complication
Timepoint [5] 0 0
Up to 3 years
Secondary outcome [6] 0 0
Intraspinal Extension
Timepoint [6] 0 0
Up to 5 years
Secondary outcome [7] 0 0
Peak Serum Concentration of Isotretinoin in Patients Enrolled on Either A3973, ANBL0032, ANBL0931, ANBL0532 and Future High Risk Studies
Timepoint [7] 0 0
Day 1 of each course
Secondary outcome [8] 0 0
Pharmacogenetic Variants in Patients Enrolled on Either A3973, ANBL0032, ANBL0931, ANBL0532 and Future High Risk Studies
Timepoint [8] 0 0
At baseline
Secondary outcome [9] 0 0
Topotecan Systemic Clearance
Timepoint [9] 0 0
Day 1 of courses 1-2
Secondary outcome [10] 0 0
Presence and Function of T Cells Capable of Recognizing Neuroblastoma
Timepoint [10] 0 0
Up to 6 months (end of therapy)
Secondary outcome [11] 0 0
Enumeration of Peripheral Blood Cluster of Differentiation (CD)3, CD4, and CD8 Cells
Timepoint [11] 0 0
Up to 6 months after completion of assigned myeloablation therapy
Secondary outcome [12] 0 0
Proportion of Patients With Neuroblastoma Detected in Bone Marrow and Peripheral Blood Using RT-PCR Technique
Timepoint [12] 0 0
Baseline
Secondary outcome [13] 0 0
EFS Pts Non-randomly Assigned to Single CEM (12-18 Mths, Stg. 4, MYCN Nonamplified Tumor/Unfavorable or Indeterminant Histopathology/Diploid DNA Content & Pts>547 Days, Stg.3, MYCN Nonamplified Tumor AND Unfavorable or Indeterminant Histopathology).
Timepoint [13] 0 0
Up to 3 years
Secondary outcome [14] 0 0
OS in Patients 12-18 Months, Stage 4, MYCN Nonamplified Tumor/Unfavorable Histopathology/Diploid DNA Content/Indeterminant Histology/Ploidy and Patients > 547 Days, Stage 3, MYCN Nonamplified Tumor AND Unfavorable Histopathology/Indeterminant Histology
Timepoint [14] 0 0
Up to 3 years

Eligibility
Key inclusion criteria
* Diagnosis of neuroblastoma or ganglioneuroblastoma by histology or as evidenced by the presence of clumps of tumor cells in bone marrow and elevated catecholamine metabolites in urine meeting any of the following criteria:

* Patients with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) stage 4 disease are eligible with the following:

* MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
* Age > 18 months (i.e., > 547 days) regardless of biologic features
* Age 12-18 months (i.e., 365-547 days) with none of the following three favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and deoxyribonucleic acid [DNA] index > 1)
* Patients with newly diagnosed neuroblastoma with INSS stage 3 are eligible with the following:

* MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
* Age > 18 months (i.e., > 547 days) with unfavorable pathology, regardless of MYCN status
* Patients with newly diagnosed INSS stage 2a or 2b with MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
* Patients with newly diagnosed INSS stage 4s with MYCN amplification (i.e., greater than four-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features
* Patients >= 365 days initially diagnosed with INSS stage 1, 2, or 4S and who progressed to a stage 4 without interval chemotherapy

* Must have been enrolled on COG-ANBL00B1
* Creatinine clearance or radioisotope glomerular filtration rate ? 70mL/min OR serum creatinine based on age/gender as follows:

* 1 month to < 6 months: 0.4 mg/dL
* 6 months to < 1 year: 0.5 mg/dL
* 1 to < 2 years: 0.6 mg/dL
* 2 to < 6 years: 0.8 mg/dL
* 6 to < 10 years: 1 mg/dL
* 10 to < 13 years: 1.2 mg/dL
* 10 to < 16 years: 1.5 mg/dL (male), 1.4 mg/dL (female)
* >= 16 years: 1.7 mg/dL (male), 1.4 mg/dL (female)
* Total bilirubin ? 1.5 times upper limit of normal (ULN) for age
* Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) < 10 times ULN for age
* Not pregnant or nursing
* Negative pregnancy test
* Shortening fraction >= 27% by echocardiogram (ECHO) OR left ventricular ejection fraction (LVEF) >= 50% by radionuclide angiogram
* No known contraindication (e.g., size, weight or physical condition) to peripheral blood stem cell collection
* No prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease
* No more than one course of chemotherapy per low- or intermediate-risk neuroblastoma therapy prior to determination of MYCN amplification and histology
Minimum age
No limit
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [4] 0 0
Royal Children's Hospital-Brisbane - Herston
Recruitment hospital [5] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4029 - Herston
Recruitment postcode(s) [3] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
Arkansas
Country [4] 0 0
United States of America
State/province [4] 0 0
California
Country [5] 0 0
United States of America
State/province [5] 0 0
Colorado
Country [6] 0 0
United States of America
State/province [6] 0 0
Connecticut
Country [7] 0 0
United States of America
State/province [7] 0 0
Delaware
Country [8] 0 0
United States of America
State/province [8] 0 0
District of Columbia
Country [9] 0 0
United States of America
State/province [9] 0 0
Florida
Country [10] 0 0
United States of America
State/province [10] 0 0
Georgia
Country [11] 0 0
United States of America
State/province [11] 0 0
Illinois
Country [12] 0 0
United States of America
State/province [12] 0 0
Indiana
Country [13] 0 0
United States of America
State/province [13] 0 0
Iowa
Country [14] 0 0
United States of America
State/province [14] 0 0
Kansas
Country [15] 0 0
United States of America
State/province [15] 0 0
Kentucky
Country [16] 0 0
United States of America
State/province [16] 0 0
Louisiana
Country [17] 0 0
United States of America
State/province [17] 0 0
Maine
Country [18] 0 0
United States of America
State/province [18] 0 0
Maryland
Country [19] 0 0
United States of America
State/province [19] 0 0
Massachusetts
Country [20] 0 0
United States of America
State/province [20] 0 0
Michigan
Country [21] 0 0
United States of America
State/province [21] 0 0
Minnesota
Country [22] 0 0
United States of America
State/province [22] 0 0
Mississippi
Country [23] 0 0
United States of America
State/province [23] 0 0
Missouri
Country [24] 0 0
United States of America
State/province [24] 0 0
Nebraska
Country [25] 0 0
United States of America
State/province [25] 0 0
Nevada
Country [26] 0 0
United States of America
State/province [26] 0 0
New Hampshire
Country [27] 0 0
United States of America
State/province [27] 0 0
New Jersey
Country [28] 0 0
United States of America
State/province [28] 0 0
New Mexico
Country [29] 0 0
United States of America
State/province [29] 0 0
New York
Country [30] 0 0
United States of America
State/province [30] 0 0
North Carolina
Country [31] 0 0
United States of America
State/province [31] 0 0
North Dakota
Country [32] 0 0
United States of America
State/province [32] 0 0
Ohio
Country [33] 0 0
United States of America
State/province [33] 0 0
Oklahoma
Country [34] 0 0
United States of America
State/province [34] 0 0
Oregon
Country [35] 0 0
United States of America
State/province [35] 0 0
Pennsylvania
Country [36] 0 0
United States of America
State/province [36] 0 0
South Carolina
Country [37] 0 0
United States of America
State/province [37] 0 0
South Dakota
Country [38] 0 0
United States of America
State/province [38] 0 0
Tennessee
Country [39] 0 0
United States of America
State/province [39] 0 0
Texas
Country [40] 0 0
United States of America
State/province [40] 0 0
Utah
Country [41] 0 0
United States of America
State/province [41] 0 0
Vermont
Country [42] 0 0
United States of America
State/province [42] 0 0
Virginia
Country [43] 0 0
United States of America
State/province [43] 0 0
Washington
Country [44] 0 0
United States of America
State/province [44] 0 0
West Virginia
Country [45] 0 0
United States of America
State/province [45] 0 0
Wisconsin
Country [46] 0 0
Canada
State/province [46] 0 0
Alberta
Country [47] 0 0
Canada
State/province [47] 0 0
British Columbia
Country [48] 0 0
Canada
State/province [48] 0 0
Manitoba
Country [49] 0 0
Canada
State/province [49] 0 0
Nova Scotia
Country [50] 0 0
Canada
State/province [50] 0 0
Ontario
Country [51] 0 0
Canada
State/province [51] 0 0
Quebec
Country [52] 0 0
New Zealand
State/province [52] 0 0
Auckland
Country [53] 0 0
Puerto Rico
State/province [53] 0 0
San Juan
Country [54] 0 0
Switzerland
State/province [54] 0 0
Bern

Funding & Sponsors
Primary sponsor type
Other
Name
Children's Oncology Group
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Cancer Institute (NCI)
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Julie R Park
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.