Trial Information

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

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.