Isotretinoin in Treating Young Patients With High-Risk Neuroblastoma

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2009 by National Cancer Institute (NCI).
Recruitment status was  Recruiting
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: July 14, 2009
Last updated: August 9, 2013
Last verified: July 2009

RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors plan better treatment for patients receiving isotretinoin.

PURPOSE: This clinical trial is studying the side effects and best dose of isotretinoin in treating young patients with high-risk neuroblastoma.

Condition Intervention
Drug: isotretinoin
Genetic: DNA analysis
Genetic: polymerase chain reaction
Other: laboratory biomarker analysis
Other: pharmacological study

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study to Investigate the Feasibility of 13-cis-retinoic Acid Pharmacokinetic Monitoring in High-risk Neuroblastoma Patients

Resource links provided by NLM:

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

Primary Outcome Measures:
  • Pharmacokinetics of isotretinoin [ Designated as safety issue: No ]
  • Toxicity according to NCI CTCAE v.3 [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Clinical response [ Designated as safety issue: No ]

Estimated Enrollment: 75
Study Start Date: February 2009
Detailed Description:



  • To investigate the feasibility of implementing individualized dosing of isotretinoin in patients with high-risk neuroblastoma after course 1 of treatment, based on isotretinoin pharmacokinetics and toxicity.
  • To minimize the large inter-patient variation in plasma concentrations of isotretinoin.
  • To ensure that patients are not exposed to potentially sub-optimal plasma concentrations of isotretinoin during long-term treatment, particularly for patients who are not able to swallow isotretinoin capsules.


  • To obtain preliminary data on the potential impact of isotretinoin therapeutic monitoring on clinical response and toxicity in these patients.

OUTLINE: This is a multicenter study.

Patients receive oral isotretinoin twice daily on days 1-14. Courses repeat every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.

Blood samples are collected at baseline and during courses 1-3 for pharmacokinetic studies and determination of each patient's individual therapeutic-dose level requirement. Genotyping to identify genes that metabolize enzymes is conducted via PCR.

After completion of study therapy, patients are followed up periodically for up to 3 years.


Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Histologically confirmed high-risk neuroblastoma
  • Concurrent isotretinoin as part of clinical treatment


  • Not pregnant
  • Fertile patients must use effective contraception
  • Has a single- or double-lumen central venous catheter in place


  • See Disease Characteristics
  Contacts and Locations
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Please refer to this study by its identifier: NCT00939965

United Kingdom
Great Ormond Street Hospital for Children Recruiting
London, England, United Kingdom, WC1N 3JH
Contact: Penelope Brock, MD, PhD    44-20-7829-7924   
University of Newcastle-Upon-Tyne Northern Institute for Cancer Research Recruiting
Newcastle-Upon-Tyne, England, United Kingdom, NE2 4HH
Contact: Gareth Veal    44-191-246-4332      
Royal Marsden - Surrey Recruiting
Sutton, England, United Kingdom, SM2 5PT
Contact: Andrew David J. Pearson, MD, FRCP, DCh    44-208-661-3163      
Sponsors and Collaborators
Children's Cancer and Leukaemia Group
Principal Investigator: Gareth Veal University of Newcastle Upon-Tyne
  More Information Identifier: NCT00939965     History of Changes
Other Study ID Numbers: CCLG-PK-2008-03  CDR0000637053  EU-20914  EUDRACT-2008-003606-33 
Study First Received: July 14, 2009
Last Updated: August 9, 2013
Health Authority: Unspecified

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

Additional relevant MeSH terms:
Neoplasms by Histologic Type
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neuroectodermal Tumors, Primitive
Neuroectodermal Tumors, Primitive, Peripheral
Dermatologic Agents processed this record on May 26, 2016