Combination Chemotherapy Followed by Surgery and Peripheral Stem Cell or Bone Marrow Transplantation in Treating Infants With Newly Diagnosed Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00025649
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : September 17, 2013
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell or bone marrow 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 given before surgery followed by peripheral stem cell or bone marrow transplantation in treating infants who have newly diagnosed neuroblastoma.

Condition or disease Intervention/treatment Phase
Neuroblastoma Biological: filgrastim Drug: busulfan Drug: carboplatin Drug: cyclophosphamide Drug: doxorubicin hydrochloride Drug: etoposide Drug: melphalan Drug: vincristine sulfate Procedure: autologous bone marrow transplantation Procedure: conventional surgery Procedure: peripheral blood stem cell transplantation Phase 2

Detailed Description:


  • Determine the survival of infants with newly diagnosed stage II, III, IV, or IVS neuroblastoma with MYCN amplification treated with etoposide, carboplatin, cyclophosphamide, doxorubicin, and vincristine followed by surgery and busulfan and melphalan with autologous peripheral blood stem cell or bone marrow transplantation.
  • Determine whether there are prognostic criteria that could be used in future therapeutic stratification of these patients.

OUTLINE: This is a multicenter study.

Patients receive VP-CARBO chemotherapy comprising etoposide IV over 2 hours and carboplatin IV over 1 hour on days 1-3. Treatment repeats every 21 days for 2 courses. Patients then receive CADO chemotherapy comprising cyclophosphamide IV over 1 hour on days 1-5, doxorubicin IV over 6 hours on days 4 and 5, and vincristine IV on days 1 and 5. Treatment repeats every 21 days for 2 courses.

Patients receive filgrastim (G-CSF) subcutaneously daily for 5 days. Patients undergo leukapheresis to collect peripheral blood stem cells (PBSC). Patients who do not mobilize sufficient cells undergo bone marrow harvest.

Patients eligible for surgery undergo surgical resection. Patients with stage IV disease with less than complete response of metastatic disease after initial chemotherapy are removed from the study.

Beginning within 2 weeks after surgery, patients receive 1 additional course of VP-CARBO chemotherapy followed by 1 additional course of CADO chemotherapy.

After at least 3 weeks, patients receive high-dose chemotherapy comprising busulfan IV over 24 hours on days -7 to -3 and melphalan IV on day -2. PBSC or bone marrow are reinfused on day 0.

At least 2 months after the completion of high-dose chemotherapy and bone marrow or PBSC transplantation, patients undergo radiotherapy to the primary site, according to preoperative imaging studies. Patients are treated with oral tretinoin after megatherapy.

Patients are followed within 6 months and then annually for 5 years.

PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study within 4 years.

Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Official Title: European Infant Neuroblastoma Study - Stage 2, 3, 4, and 4S; MYCN Amplified Tumors
Study Start Date : July 1999
Study Completion Date : March 2009

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 1 Year   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed newly diagnosed stage II, III, IV, or IVS neuroblastoma or ganglioneuroblastoma
  • MYCN amplification (i.e., at least 10 copies)



  • Under 12 months at diagnosis

Performance status:

  • Not specified

Life expectancy:

  • Not specified


  • Not specified


  • Not specified


  • Not specified


Biologic therapy:

  • Not specified


  • Not specified

Endocrine therapy:

  • Not specified


  • Not specified


  • Not specified

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00025649

St. Anna Children's Hospital
Vienna, Austria, A-1090
Universitair Ziekenhuis Gent
Ghent, Belgium, B-9000
Copenhagen, Denmark, 2100
Centre Hospitalier Regional de Purpan
Toulouse, France, 31026
Istituto Giannina Gaslini
Genoa, Italy, 16148
Rikshospitalet University Hospital
Oslo, Norway, 0027
Instituto Portugues de Oncologia de Francisco Gentil - Centro Regional de Oncologia de Lisboa, S.A.
Lisboa, Portugal, 1099-023 Codex
Hospital Universitario LA FE
Valencia, Spain, 46009
Ostra Sjukhuset
Gothenburg, Sweden, 41685
Centre Hospitalier Universitaire Vaudois
Lausanne, Switzerland, CH-1011
United Kingdom
Bristol Royal Hospital for Children
Bristol, England, United Kingdom, BS2 8BJ
Sponsors and Collaborators
European Infant Neuroblastoma Study Group - 1999
Study Chair: Adela Canete, MD, PhD Hospital Universitario La Fe

Publications of Results: Identifier: NCT00025649     History of Changes
Other Study ID Numbers: CDR0000068982
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: September 17, 2013
Last Verified: December 2001

Keywords provided by National Cancer Institute (NCI):
localized resectable neuroblastoma
regional neuroblastoma
disseminated neuroblastoma
stage 4S neuroblastoma
localized unresectable 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
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antibiotics, Antineoplastic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors