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Observation, Combination Chemotherapy, Radiation Therapy, and/or Autologous Stem Cell Transplant in Treating Young Patients With Neuroblastoma

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2008 by National Cancer Institute (NCI).
Recruitment status was:  Recruiting
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: December 11, 2006
Last updated: August 6, 2013
Last verified: January 2008

RATIONALE: 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 may kill more tumor cells. Radiation therapy uses high-energy x-rays to kill tumor cells. An autologous stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. This may allow more chemotherapy to be given so that more tumor cells are killed. Sometimes, after surgery, the tumor may not need more treatment until it progresses. In this case, observation may be sufficient. It is not yet known whether observation is more effective than combination chemotherapy, radiation therapy, and/or autologous stem cell transplant in treating neuroblastoma.

PURPOSE: This randomized phase III and phase IV trial is studying observation, combination chemotherapy, radiation therapy, and/or autologous stem cell transplant to compare how well they work in treating young patients with neuroblastoma.

Condition Intervention Phase
Biological: filgrastim
Drug: carboplatin
Drug: cisplatin
Drug: cyclophosphamide
Drug: dacarbazine
Drug: doxorubicin hydrochloride
Drug: etoposide phosphate
Drug: ifosfamide
Drug: isotretinoin
Drug: melphalan
Drug: topotecan hydrochloride
Drug: vincristine sulfate
Drug: vindesine
Procedure: autologous hematopoietic stem cell transplantation
Procedure: conventional surgery
Procedure: peripheral blood stem cell transplantation
Radiation: iobenguane I 131
Radiation: radiation therapy
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Open Label
Primary Purpose: Treatment
Official Title: NB2004 Trial Protocol for Risk Adapted Treatment of Children With Neuroblastoma

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Event-free survival (EFS)
  • Locoregional EFS

Secondary Outcome Measures:
  • Time from diagnosis to transition to stage 4 disease, to death from disease, or to the last follow-up (if no transition to stage 4 disease is observed)
  • Overall survival
  • Time to the beginning of primary tumor regression (in patients in the low-risk group [LRG])
  • Time to the normalization of tumor markers HVA and VMA in urine
  • Time to no evidence of disease (in patients in the LRG with stage 4S disease)
  • Status of the primary tumor 12 months after diagnosis (LRG)
  • Best status of the primary tumor within the first 12 months (LRG)
  • Status of chromosome 1p (unblinded) and status of chromosome 11q (blinded)
  • Comparison of the extent of initial surgery (incomplete resection vs macroscopic complete resection) (LRG)
  • Comparison of the extent of best surgery during protocol treatment (incomplete resection vs macroscopic complete resection)
  • Surgery-related complications (i.e., bleeding, infection, intestinal obstruction, or other)
  • Disease progression and symptoms controlled after the first, second, third, and fourth N4 course (LRG)
  • Disease progression and symptoms not controlled after four N4 courses (LRG)
  • Transition to stage 4 disease at any time (LRG)
  • Acute and late side effects of external-beam radiotherapy (medium-risk group [MRG] and high-risk group [HRG])
  • Early response after 2 courses of induction therapy (N5 and N6 or two courses of N8) (HRG)
  • Response to induction therapy prior to conditioning therapy or after 280 days (HRG)
  • Grade of toxicity observed during induction therapy course 1 (N5 or N8) (HRG)
  • Grade of toxicity observed during induction therapy course 2 (N6 or N8) (HRG)
  • Frequency of grade 3 or 4 toxicity observed during the last 6 courses of induction therapy (3 courses of N5 and N6) (HRG)
  • Activity and whole body dose of radiotherapy

Estimated Enrollment: 642
Study Start Date: October 2004
Estimated Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
  Show Detailed Description


Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of neuroblastoma by histology using tumor tissue or as evidenced by the presence of distinct neuroblastoma cells in the bone marrow AND elevated catecholamine metabolites (i.e., homovanillic acid [HVA] and vanillylmandelic acid [VMA]) in blood or urine

    • Newly diagnosed disease (for patients in the low-risk group)
    • Diagnosis from tumor tissue (for patients in the medium-risk group)
  • Meets criteria for 1 of the following risk groups:

    • Low-risk group

      • No MYCN amplification AND meets 1 of the following criteria:

        • Stage 1 disease
        • Stage 2 disease with no chromosome 1p deletion or imbalance
        • Stage 3 disease with no chromosome 1p deletion or imbalance (for patients < 2 years of age)
        • Stage 4S disease (for patients < 1 year of age)
    • Medium-risk group

      • No MYCN amplification AND meets 1 of the following criteria:

        • Stage 2 disease with chromosome 1p deletion or imbalance
        • Stage 3 disease with chromosome 1p deletion or imbalance

          • Any chromosome 1p status (for patients ≥ 2 years of age)
        • Stage 4 disease (for patients < 1 year of age)
    • High-risk group, meeting 1 of the following criteria:

      • Any stage disease with MYCN amplification

        • Any MYCN status (for patients ≥ 1 year of age)


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception


  • No prior nephrectomy or other mutilating surgery as initial surgery (for patients in the low-risk group)
  • No other concurrent anticancer therapy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00410631

  Show 80 Study Locations
Sponsors and Collaborators
Gesellschaft fur Padiatrische Onkologie und Hamatologie - Germany
Study Chair: Frank Berthold, MD Children's Hospital Medical Center, Cincinnati
  More Information

Publications automatically indexed to this study by Identifier (NCT Number): Identifier: NCT00410631     History of Changes
Other Study ID Numbers: GPOH-NB2004
CDR0000517312 ( Registry Identifier: PDQ (Physician Data Query) )
Study First Received: December 11, 2006
Last Updated: August 6, 2013

Keywords provided by National Cancer Institute (NCI):
localized resectable neuroblastoma
localized unresectable neuroblastoma
regional neuroblastoma
stage 4S neuroblastoma
disseminated neuroblastoma
recurrent neuroblastoma

Additional relevant MeSH terms:
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Liposomal doxorubicin
Etoposide phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action processed this record on May 24, 2017