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Temozolomide in Treating Young Patients With High-Risk Relapsed or Refractory 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: NCT00276679
Recruitment Status : Completed
First Posted : January 13, 2006
Last Update Posted : September 17, 2013
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well temozolomide works in treating patients with high-risk relapsed or refractory neuroblastoma.

Condition or disease Intervention/treatment Phase
Neuroblastoma Drug: temozolomide Phase 2

Detailed Description:



  • Determine the anti-tumor activity of temozolomide in young patients with high-risk relapsed or refractory neuroblastoma.


  • Determine the duration of response in patients treated with this drug.
  • Determine tolerability of this drug in these patients
  • Determine the tumor expression of the cellular repair mechanisms which repair DNA damage (O6-methylguanine-DNA methyltransferase [MGMT] and mismatch repair [MMR] systems) in patients treated with this drug.
  • Correlate MGMT and MMR expression with outcomes in patients treated with this drug.
  • Determine if MGMT and MMR expression/activity changes in the tumor during initial presentation, treatment, and relapse/progression in patients treated with this drug.
  • Determine the activity of MGMT in bone marrow taken at relapse, in terms of hematological toxicity, in patients treated with this drug.

OUTLINE: This is a multicenter, open label, nonrandomized study.

Patients receive oral temozolomide once daily for 5 days. Treatment repeats every 28 days for 2 courses. Patients achieving stable or responding disease after completion of the 2 courses may receive up to 10 additional courses of treatment in the absence of disease progression or unacceptable toxicity.

PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Allocation: Non-Randomized
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of Temozolomide (Temodal) in Children Over 1 Year of Age With Relapsed or Refractory High Risk Neuroblastoma
Study Start Date : April 2003
Actual Study Completion Date : November 2006

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically proven neuroblastoma

    • High-risk relapsed or refractory disease, defined as 1 of the following:

      • Metastatic disease
      • Localized MYC-N amplified disease
      • Localized non MYC-N amplified disease at second relapse
  • Measurable primary or metastatic disease by cross-sectional imaging or MIBG scan


  • Lansky performance status 40-100%
  • Life expectancy > 2 months
  • Not pregnant or nursing
  • Fertile patients must use effective contraception during the course of this study and for 6 months after study completion
  • Organ toxicity < grade 2
  • Platelets ≥ 100,000/mm^3 (50,000/mm^3 after stem cell transplant or in case of marrow involvement)
  • Neutrophil count ≥ 500/mm^3
  • Bilirubin < 1.5 times normal
  • AST and ALT ≤ 2.5 times normal
  • No known HIV positivity


  • More than 21 days since prior chemotherapy treatment (isotretinoin is counted as chemotherapy for this purpose)
  • More than 30 days since prior radiotherapy except local palliative treatment for pain control
  • No more than 2 prior treatments for neuroblastoma
  • No other concurrent investigative treatment for neuroblastoma

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): NCT00276679

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Centre Hospitalier Regional de Purpan
Toulouse, France, 31026
Our Lady's Hospital for Sick Children
Dublin, Ireland, 12
United Kingdom
Birmingham Children's Hospital
Birmingham, England, United Kingdom, B4 6NH
Institute of Child Health at University of Bristol
Bristol, England, United Kingdom, BS2 8AE
Addenbrooke's Hospital at Cambridge University Hospitals NHS Foundation Trust
Cambridge, England, United Kingdom, CB2 2QQ
Leeds Cancer Centre at St. James's University Hospital
Leeds, England, United Kingdom, LS9 7TF
Leicester Royal Infirmary
Leicester, England, United Kingdom, LE1 5WW
Royal Liverpool Children's Hospital, Alder Hey
Liverpool, England, United Kingdom, L12 2AP
Royal London Hospital
London, England, United Kingdom, E1 1BB
Great Ormond Street Hospital for Children NHS Trust
London, England, United Kingdom, WC1N 3JH
Central Manchester and Manchester Children's University Hospitals NHS Trust
Manchester, England, United Kingdom, M27 4HA
Sir James Spence Institute of Child Health
Newcastle-Upon-Tyne, England, United Kingdom, NE1 4LP
Queen's Medical Centre
Nottingham, England, United Kingdom, NG7 2UH
Oxford Radcliffe Hospital
Oxford, England, United Kingdom, 0X3 9DU
Children's Hospital - Sheffield
Sheffield, England, United Kingdom, S10 2TH
Southampton General Hospital
Southampton, England, United Kingdom, SO16 6YD
Royal Marsden NHS Foundation Trust - Surrey
Sutton, England, United Kingdom, SM2 5PT
Royal Belfast Hospital for Sick Children
Belfast, Northern Ireland, United Kingdom, BT12 6BE
Royal Aberdeen Children's Hospital
Aberdeen, Scotland, United Kingdom, AB25 2ZG
Royal Hospital for Sick Children
Edinburgh, Scotland, United Kingdom, EH9 1LF
Royal Hospital for Sick Children
Glasgow, Scotland, United Kingdom, G3 8SJ
Childrens Hospital for Wales
Cardiff, Wales, United Kingdom, CF14 4XW
Sponsors and Collaborators
Children's Cancer and Leukaemia Group
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Study Chair: Herve Rubie, MD Centre Hospitalier Regional de Purpan
OverallOfficial: Andrew David J. Pearson, MD, FRCP, DCh University of Newcastle Upon-Tyne
OverallOfficial: Julia Chisholm, MD Great Ormond Street Hospital for Children NHS Foundation Trust

Publications of Results:
Layout table for additonal information Identifier: NCT00276679     History of Changes
Other Study ID Numbers: CDR0000454577
First Posted: January 13, 2006    Key Record Dates
Last Update Posted: September 17, 2013
Last Verified: January 2006

Keywords provided by National Cancer Institute (NCI):
recurrent neuroblastoma
disseminated neuroblastoma
localized unresectable neuroblastoma

Additional relevant MeSH terms:
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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
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents