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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00017368




Registration number
NCT00017368
Ethics application status
Date submitted
6/06/2001
Date registered
9/10/2003
Date last updated
13/02/2014

Titles & IDs
Public title
Combination Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Neuroblastoma
Scientific title
A Pilot Study Of Tandem High Dose Chemotherapy With Stem Cell Rescue Following Induction Therapy In Children With High Risk Neuroblastoma
Secondary ID [1] 0 0
COG-ANBL00P1
Secondary ID [2] 0 0
ANBL00P1
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
Other interventions - filgrastim
Other interventions - sargramostim
Treatment: Drugs - carboplatin
Treatment: Drugs - cisplatin
Treatment: Drugs - cyclophosphamide
Treatment: Drugs - doxorubicin hydrochloride
Treatment: Drugs - etoposide
Treatment: Drugs - etoposide phosphate
Treatment: Drugs - ifosfamide
Treatment: Drugs - isotretinoin
Treatment: Drugs - melphalan
Treatment: Drugs - thiotepa
Treatment: Drugs - vincristine sulfate
Treatment: Surgery - conventional surgery
Treatment: Surgery - peripheral blood stem cell transplantation
Treatment: Other - radiation therapy

Experimental: All Patients -


Other interventions: filgrastim


Other interventions: sargramostim


Treatment: Drugs: carboplatin


Treatment: Drugs: cisplatin


Treatment: Drugs: cyclophosphamide


Treatment: Drugs: doxorubicin hydrochloride


Treatment: Drugs: etoposide


Treatment: Drugs: etoposide phosphate


Treatment: Drugs: ifosfamide


Treatment: Drugs: isotretinoin


Treatment: Drugs: melphalan


Treatment: Drugs: thiotepa


Treatment: Drugs: vincristine sulfate


Treatment: Surgery: conventional surgery


Treatment: Surgery: peripheral blood stem cell transplantation


Treatment: Other: radiation therapy


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
Transplant-related mortality - The endpoint used for early stopping rule 9.51 will be transplant-related mortality (TRM). A TRM is defined as any death occurring within 30 days after either the first or second HDC/SCR. The acceptable TRM rate is 7.5%. This rate is based on TRM rates previously observed in the prior CCG study 594, CCG study 3891, and POG study 9640 of 7%, 6%, and 0%, respectively.
Timepoint [1] 0 0
Secondary outcome [1] 0 0
Incidence of symptomatic CMV, disseminated adenovirus infection, or EBV-LPD - The endpoint to be used in assessing early stopping rule 9.52 will be the incidence of symptomatic CMV, disseminated adenovirus infection, or EBV-LPD. Patients with positive CMV antigenemia or CMV urine culture only (without symptoms of CMV infection), or patients who have a positive culture for adenovirus without evidence of dissemination, or patients with a positive EBV PCR who do not exhibit significant clinical signs (such as adenopathy) or symptoms will not trigger the stopping rule.
Timepoint [1] 0 0
Secondary outcome [2] 0 0
Event-free Survival - Assessment of feasibility and toxicity at the end of the study is time to event. Time to event is defined as the time from registration on the study until the first of the following events occurs: relapse, progression, secondary malignancy, or death, including toxic death. If none of those events occurs, then the time of last contact with the patient is used. Time to event will be used to calculate the one-year Event-free Survival (EFS) rate.
Timepoint [2] 0 0
1 year

Eligibility
Key inclusion criteria
DISEASE CHARACTERISTICS:

- Newly diagnosed high-risk neuroblastoma

- Histologically proven AND/OR

- Bone marrow specimen showing clumps of tumor cells accompanied by elevated
urinary catecholamines

- Age 1-30:

- Must meet one of the following INSS staging criteria:

- Stage IV regardless of biologic factors

- Stage IIa/IIb with MYCN oncogene amplification (greater than 10) and
unfavorable pathology

- Stage III with MYCN oncogene amplification (greater than 10) or
unfavorable pathology

- Initially stage I, II, or IVS, that has progressed without interval
chemotherapy

- Under age 1:

- INSS stage III, IV, or IVS with MYCN amplification (greater than 10)

- Must enter neuroblastoma biology study COG-ANBL00B1 within 2 weeks of diagnosis and
before entry on this study

PATIENT CHARACTERISTICS:

Age:

- 30 and under at original diagnosis

Performance status:

- Not specified

Life expectancy:

- Not specified

Hematopoietic:

- Not specified

Hepatic:

- Not specified

Renal:

- Not specified

Other:

- Not pregnant or nursing

- Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

- Not specified

Chemotherapy:

- See Disease Characteristics

- No more than 1 prior course of chemotherapy on the intergroup low- or
intermediate-risk neuroblastoma studies prior to determination of MYCN status and
Shimada histology

Endocrine therapy:

- Not specified

Radiotherapy:

- Prior emergent radiotherapy to sites of function- or life-threatening neuroblastoma
allowed

Surgery:

- Not specified

Other:

- No other prior systemic therapy for neuroblastoma
Minimum age
No limit
Maximum age
30 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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 2
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)
WA
Recruitment hospital [1] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
6001 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Oregon
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
United States of America
State/province [6] 0 0
Wisconsin

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 use different ways to stop tumor cells from dividing so
they stop growing or die. Combining more than one drug may kill more tumor cells. Peripheral
stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and
kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy followed by
peripheral stem cell transplantation in treating children who have newly diagnosed
neuroblastoma.
Trial website
https://clinicaltrials.gov/show/NCT00017368
Trial related presentations / publications
Marcus KJ, Shamberger R, Litman H, von Allmen D, Grupp SA, Nancarrow CM, Goldwein J, Grier HE, Diller L. Primary tumor control in patients with stage 3/4 unfavorable neuroblastoma treated with tandem double autologous stem cell transplants. J Pediatr Hematol Oncol. 2003 Dec;25(12):934-40.
Kletzel M, Katzenstein HM, Haut PR, Yu AL, Morgan E, Reynolds M, Geissler G, Marymount MH, Liu D, Kalapurakal JA, Shore RM, Bardo DM, Schmoldt J, Rademaker AW, Cohn SL. Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue: results of the Chicago Pilot II Study. J Clin Oncol. 2002 May 1;20(9):2284-92.
Donovan J, Temel J, Zuckerman A, Gribben J, Fang J, Pierson G, Ross A, Diller L, Grupp SA. CD34 selection as a stem cell purging strategy for neuroblastoma: preclinical and clinical studies. Med Pediatr Oncol. 2000 Dec;35(6):677-82.
Grupp SA, Stern JW, Bunin N, Nancarrow C, Adams R, Gorlin JB, Griffin G, Diller L. Rapid-sequence tandem transplant for children with high-risk neuroblastoma. Med Pediatr Oncol. 2000 Dec;35(6):696-700.
Grupp SA, Stern JW, Bunin N, Nancarrow C, Ross AA, Mogul M, Adams R, Grier HE, Gorlin JB, Shamberger R, Marcus K, Neuberg D, Weinstein HJ, Diller L. Tandem high-dose therapy in rapid sequence for children with high-risk neuroblastoma. J Clin Oncol. 2000 Jul;18(13):2567-75.
Public notes

Contacts
Principal investigator
Name 0 0
Stephan A. Grupp, MD, PhD
Address 0 0
Children's Hospital of Philadelphia
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
Other publications