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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00070200




Registration number
NCT00070200
Ethics application status
Date submitted
3/10/2003
Date registered
7/10/2003
Date last updated
13/02/2014

Titles & IDs
Public title
Induction Chemotherapy Using Cyclophosphamide and Topotecan in Treating Patients Who Are Undergoing Autologous Peripheral Stem Cell Transplantation for Newly Diagnosed or Progressive Neuroblastoma
Scientific title
A Pilot Induction Regimen Incorporating Topotecan for Treatment of Newly Diagnosed High Risk Neuroblastoma
Secondary ID [1] 0 0
CDR0000330140
Secondary ID [2] 0 0
ANBL02P1
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
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
Experimental: All patients - Induction Cycles 1 and 2 (CT) (21 days each), Cyclophosphamide (Days 1 thru 5) weight based dosage (\> 12 kg 400 mg/m2/day, \< 12 kg 13.3 mg/kg/day, \< 2 years old N/A. Topotecan (Days 1 thru 5) weight based dosage (\> 12 kg 1.2 mg/m2/day, \< 12 kg 0.04 mg/kg/day, \< 2 years old 0.04 mg/kg/day). Filgrastim (Days 6 ?) weight based dosage (\> 12 kg 5 micrograms/kg, \< 12 kg 5 micrograms /kg, \< 2 years old 5 micrograms /kg.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients who are classified as a "success"
Timepoint [1] 0 0
Length of study
Secondary outcome [1] 0 0
Number of toxic deaths
Timepoint [1] 0 0
Length of study
Secondary outcome [2] 0 0
Proportion of patients with dose limiting toxicities during induction cycle 1 and 2
Timepoint [2] 0 0
Length of study
Secondary outcome [3] 0 0
Tumor contamination of PBSCs
Timepoint [3] 0 0
Length of study
Secondary outcome [4] 0 0
Inability to adequately mobilize PBSCs
Timepoint [4] 0 0
Length of study
Secondary outcome [5] 0 0
Assessment of response
Timepoint [5] 0 0
Length of study

Eligibility
Key inclusion criteria
DISEASE CHARACTERISTICS:

* Histologically or cytologically confirmed neuroblastoma or ganglioneuroblastoma meeting 1 of the following staging criteria:

* Newly diagnosed disease, at least 1 year of age, and meets criteria for 1 of the following:

* International Neuroblastoma Staging System (INSS) stage 2a/2b with MYCN amplification (greater than 10) AND unfavorable pathology
* INSS stage 3 with MYCN amplification OR unfavorable pathology
* Newly diagnosed INSS stage 4 disease meeting criteria for 1 of the following:

* Over 18 months of age
* Age 12 to 18 months with any unfavorable biologic feature (MYCN amplification, unfavorable pathology, and/or DNA index=1) or any biologic feature that is indeterminant, unsatisfactory, or unknown

* No INSS stage 4 disease and age 12 to 18 months with all 3 favorable biologic features (i.e., nonamplified MYCN, favorable pathology, and DNA index greater than 1)
* Newly diagnosed INSS stage 3, 4, or 4S disease AND under 1 year of age with MYCN amplification
* At least 1 year of age and initially diagnosed with INSS stage 1, 2, or 4S disease that progressed to stage 4 without interval chemotherapy

* Must have been enrolled on COG-ANBL00B1 at initial diagnosis

PATIENT CHARACTERISTICS:

Age

* 30 and under at initial diagnosis

Performance status

* Not specified

Life expectancy

* Not specified

Hematopoietic

* Absolute neutrophil count at least 1,000/mm^3*
* Platelet count at least 100,000/mm^3* (transfusion independent)
* Hemoglobin at least 10.0 g/dL* (red blood cell transfusions allowed) NOTE: *Granulocytopenia, anemia, and/or thrombocytopenia allowed for patients with tumor metastatic to the bone marrow

Hepatic

* Bilirubin no greater than 1.5 mg/dL
* ALT less than 300 IU/L

Renal

* Creatinine no greater than 1.5 mg/dL
* Creatinine clearance or radioisotope glomerular filtration rate at least 60 mL/min

Cardiovascular

* ECG normal
* Shortening fraction at least 27% by echocardiogram OR
* Ejection fraction at least 50% by MUGA

Other

* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* HIV negative

PRIOR CONCURRENT THERAPY:

Biologic therapy

* Not specified

Chemotherapy

* See Disease Characteristics
* No more than 1 prior chemotherapy course on the low- or intermediate-risk neuroblastoma studies (COG-P9641, COG-A3961) prior to determination of MYCN amplification and Shimada histology

Endocrine therapy

* Not specified

Radiotherapy

* Prior localized emergency radiotherapy to sites of life-threatening or function-threatening disease allowed

Surgery

* Not specified

Other

* No other prior systemic therapy
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
Not applicable
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
Single group
Other design features
Phase
Phase 1
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
1/03/2004
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2013
Sample size
Target
Accrual to date
Final
31
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Tennessee
Country [4] 0 0
United States of America
State/province [4] 0 0
Washington

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
RATIONALE: Drugs used in chemotherapy, such as topotecan and cyclophosphamide, use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.

PURPOSE: This phase I trial is studying the side effects of induction chemotherapy using cyclophosphamide and topotecan in treating patients who are undergoing surgery and autologous stem cell transplantation followed by radiation therapy for newly diagnosed or progressive neuroblastoma.
Trial website
https://clinicaltrials.gov/study/NCT00070200
Trial related presentations / publications
Park JR, Scott JR, Stewart CF, London WB, Naranjo A, Santana VM, Shaw PJ, Cohn SL, Matthay KK. Pilot induction regimen incorporating pharmacokinetically guided topotecan for treatment of newly diagnosed high-risk neuroblastoma: a Children's Oncology Group study. J Clin Oncol. 2011 Nov 20;29(33):4351-7. doi: 10.1200/JCO.2010.34.3293. Epub 2011 Oct 17.
Public notes

Contacts
Principal investigator
Name 0 0
Julie R. Park, MD
Address 0 0
Seattle Children's Hospital
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00070200