Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00033293




Registration number
NCT00033293
Ethics application status
Date submitted
9/04/2002
Date registered
27/01/2003
Date last updated
18/04/2023

Titles & IDs
Public title
Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma
Scientific title
A Phase III Randomized Trial of Intravenous Gammaglobulin Therapy for Patients With Neuroblastoma Associated Opsoclonus-Myoclonus-Ataxia Syndrome Treated With Chemotherapy and Prednisone
Secondary ID [1] 0 0
NCI-2009-00399
Secondary ID [2] 0 0
ANBL00P3
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
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
Other interventions - Clinical Observation
Treatment: Drugs - Cyclophosphamide
Other interventions - Laboratory Biomarker Analysis
Treatment: Surgery - Magnetic Resonance Imaging
Treatment: Drugs - Prednisone
Treatment: Other - Therapeutic Immune Globulin

Experimental: Arm I (chemotherapy, immunoglobulin therapy) - Patients with intermediate-risk or high-risk neuroblastoma receive chemotherapy (including cyclophosphamide) according to the standard of care for the stage of primary neuroblastoma, beginning on day 0. Patients with low-risk neuroblastoma (and not receiving other chemotherapy) receive cyclophosphamide IV over 1 hour on day 0. Treatment repeats every 4 weeks for 6 courses in the absence of disease progression or unacceptable toxicity. All patients receive oral prednisone twice daily for 3 months and then every other day for 7-15 months.

Patients receive immune globulin IV on days -2 and -1, at weeks 4, 8, 12, 16, 20, and 24, and then at months 8, 10, and 12 after therapy. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients with no response after 6 months go off treatment.

Active comparator: Arm II (chemotherapy, observation) - Patients with intermediate-risk or high-risk neuroblastoma receive chemotherapy (including cyclophosphamide) according to the standard of care for the stage of primary neuroblastoma, beginning on day 0. Patients with low-risk neuroblastoma (and not receiving other chemotherapy) receive cyclophosphamide IV over 1 hour on day 0. Treatment repeats every 4 weeks for 6 courses in the absence of disease progression or unacceptable toxicity. All patients receive oral prednisone twice daily for 3 months and then every other day for 7-15 months.

Patients do not receive immune globulin. Patients with unresponsive opsoclonus-myoclonus-ataxia syndrome after 2 months or progression after 6 months may cross over to arm I.


Other interventions: Clinical Observation
Undergo observation

Treatment: Drugs: Cyclophosphamide
Given IV

Other interventions: Laboratory Biomarker Analysis
Correlative studies

Treatment: Surgery: Magnetic Resonance Imaging
Correlative studies

Treatment: Drugs: Prednisone
Given orally

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

Outcomes
Primary outcome [1] 0 0
Number of Responders
Timepoint [1] 0 0
Changes from baseline to 2 months, 6 months, and 1 year
Secondary outcome [1] 0 0
Motor Coordination as Assessed by Neurological Examination and Vineland Adaptive Behavior Scale (VABS)
Timepoint [1] 0 0
Changes from baseline to the better of 6 months or 1 year
Secondary outcome [2] 0 0
Functional Outcome as Assessed by Age-appropriate Neuropsychological Testing
Timepoint [2] 0 0
Changes from baseline to the better of 6 months or 1 year
Secondary outcome [3] 0 0
Biology of Neuroblastoma Associated Opsoclonus-myoclonus-ataxia (OMA) Syndrome Specifically by MRI Findings, Anti-neuronal Antibodies, Cerebrospinal Fluid (CSF) Findings and Tumor Biology
Timepoint [3] 0 0
At diagnosis, 6 months, 1 year, 5 and 10 years after diagnosis
Secondary outcome [4] 0 0
Long-term Prognosis for Neurologic Recovery by Neurological Examination
Timepoint [4] 0 0
At diagnosis and yearly for 10 years after diagnosis
Secondary outcome [5] 0 0
Tumor Outcome in Terms of Event-free Survival (EFS) Rate Defined as a Relapse or Progression of Neuroblastoma, a Second Malignancy, or Death
Timepoint [5] 0 0
Up to 3 years
Secondary outcome [6] 0 0
Tumor Outcome in Terms of Overall Survival (OS) Rate
Timepoint [6] 0 0
Up to 3 years

Eligibility
Key inclusion criteria
* Newly diagnosed neuroblastoma (NBL) or ganglioneuroblastoma with tumor-associated opsoclonus-myoclonus-ataxia syndrome (OMA)

* Patients with NBL diagnosed within 6 months of OMA diagnosis AND patients with OMA diagnosed within 6 months of NBL diagnosis are eligible
* Must enroll on study within 4 weeks of diagnosis
* Presence of opsoclonus, myoclonus, and/or ataxia associated with neuroblastoma considered eligible
* Currently enrolled on COG neuroblastoma protocols: COG-ANBL00B1 or its successor
* Creatinine clearance or radioisotope GFR = 70 mL/min OR serum creatinine based on age/gender as follows:

* = 0.4 mg/dL (for patients 1 to 5 months of age)
* = 0.5 mg/dL (for patients 6 to 11 months of age)
* = 0.6 mg/dL (for patients 1 year of age)
* = 0.8 mg/dL (for patients 2 to 5 years of age)
* = 1.0 mg/dL (for patients 6 to 9 years of age)
* = 1.2 mg/dL (for patients 10 to 12 years of age)
* = 1.4 mg/dL (for female patients = 13 years of age)
* = 1.5 mg/dL (for male patients 13 to 15 years of age)
* = 1.6 mg/dL (for male patients = 16 years of age)
* No prior IV gamma globulin therapy
* No prior chemotherapy
* Concurrent chemotherapy allowed
* No prior prednisone or corticotropin

* Patients who have received = 14 days of steroids are eligible
* Concurrent surgery allowed
Minimum age
No limit
Maximum age
8 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
15/03/2004
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/12/2022
Sample size
Target
Accrual to date
Final
53
Recruitment in Australia
Recruitment state(s)
NSW,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
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 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
Idaho
Country [12] 0 0
United States of America
State/province [12] 0 0
Illinois
Country [13] 0 0
United States of America
State/province [13] 0 0
Indiana
Country [14] 0 0
United States of America
State/province [14] 0 0
Kentucky
Country [15] 0 0
United States of America
State/province [15] 0 0
Louisiana
Country [16] 0 0
United States of America
State/province [16] 0 0
Maryland
Country [17] 0 0
United States of America
State/province [17] 0 0
Massachusetts
Country [18] 0 0
United States of America
State/province [18] 0 0
Michigan
Country [19] 0 0
United States of America
State/province [19] 0 0
Mississippi
Country [20] 0 0
United States of America
State/province [20] 0 0
Missouri
Country [21] 0 0
United States of America
State/province [21] 0 0
Nevada
Country [22] 0 0
United States of America
State/province [22] 0 0
New Jersey
Country [23] 0 0
United States of America
State/province [23] 0 0
New York
Country [24] 0 0
United States of America
State/province [24] 0 0
North Carolina
Country [25] 0 0
United States of America
State/province [25] 0 0
Ohio
Country [26] 0 0
United States of America
State/province [26] 0 0
Oklahoma
Country [27] 0 0
United States of America
State/province [27] 0 0
Oregon
Country [28] 0 0
United States of America
State/province [28] 0 0
Pennsylvania
Country [29] 0 0
United States of America
State/province [29] 0 0
South Carolina
Country [30] 0 0
United States of America
State/province [30] 0 0
South Dakota
Country [31] 0 0
United States of America
State/province [31] 0 0
Tennessee
Country [32] 0 0
United States of America
State/province [32] 0 0
Texas
Country [33] 0 0
United States of America
State/province [33] 0 0
Vermont
Country [34] 0 0
United States of America
State/province [34] 0 0
Washington
Country [35] 0 0
United States of America
State/province [35] 0 0
West Virginia
Country [36] 0 0
United States of America
State/province [36] 0 0
Wisconsin
Country [37] 0 0
Canada
State/province [37] 0 0
British Columbia
Country [38] 0 0
Canada
State/province [38] 0 0
Nova Scotia
Country [39] 0 0
Canada
State/province [39] 0 0
Quebec
Country [40] 0 0
Canada
State/province [40] 0 0
Saskatchewan

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
Pedro A De Alarcon
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?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT00033293