Isotretinoin in Treating Young Patients With High-Risk Neuroblastoma

Study Overview
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Brief Summary
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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.

Detailed Description
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OBJECTIVES: Primary 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. Secondary 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.

Official Title
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Pilot Study to Investigate the Feasibility of 13-cis-retinoic Acid Pharmacokinetic Monitoring in High-risk Neuroblastoma Patients

Conditions
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Neuroblastoma

Intervention / Treatment
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* Drug: isotretinoin
* Genetic: DNA analysis
* Genetic: polymerase chain reaction
* Other: laboratory biomarker analysis
* Other: pharmacological study


Participation Criteria
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Eligibility Criteria
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DISEASE CHARACTERISTICS: Histologically confirmed high-risk neuroblastoma Concurrent isotretinoin as part of clinical treatment PATIENT CHARACTERISTICS: Not pregnant Fertile patients must use effective contraception Has a single- or double-lumen central venous catheter in place PRIOR CONCURRENT THERAPY: See Disease Characteristics

Ages Eligible for Study
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Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: isotretinoin|nan|
|Genetic: DNA analysis|nan|
|Genetic: polymerase chain reaction|nan|
|Other: laboratory biomarker analysis|nan|
|Other: pharmacological study|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics of isotretinoin |  |  | 
| Toxicity according to NCI CTCAE v.3 |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical response |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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localized unresectable neuroblastoma, recurrent neuroblastoma, stage 4S neuroblastoma