Clinical trial NCT00939965: Isotretinoin in Treating Young Patients With High-Risk Neuroblastoma
Official title: Pilot Study to Investigate the Feasibility of 13-cis-retinoic Acid Pharmacokinetic Monitoring in High-risk Neuroblastoma Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Neuroblastoma
Keywords: localized unresectable neuroblastoma
recurrent neuroblastoma
stage 4S neuroblastoma
Lead sponsor: National Cancer Institute (NCI)
Sponsor class: NIH
First posted: 2009-07-15
Enrollment: 75
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Pharmacokinetics of isotretinoin
measure: Toxicity according to NCI CTCAE v.3
Secondary outcomes:
measure: Clinical response
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
state: England
city: Newcastle-Upon-Tyne
country: United Kingdom
facility: University of Newcastle-Upon-Tyne Northern Institute for Cancer Research
state: England
city: Sutton
country: United Kingdom
facility: Royal Marsden - Surrey
state: England
Study officials:
affiliation: University of Newcastle Upon-Tyne
name: Gareth Veal
role: PRINCIPAL_INVESTIGATOR
References: