NCT00084422

N2001-03: CEP-701 in Treating Young Patients With Recurrent or Refractory High-Risk Neuroblastoma

A Phase I Study Of CEP-701 In Patients With Refractory Neuroblastoma (IND # 67,722)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 47 (actual)
• Sponsor: New Approaches to Neuroblastoma Therapy Consortium · Academic / Other
• Sex: All
• Age: 1 Day – 30 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: CEP-701 may stop the growth of tumor cells by blocking the enzymes necessary for their growth. PURPOSE: This phase I trial is studying the side effects and best dose of CEP-701 in treating young patients with recurrent or refractory high-risk neuroblastoma.

Detailed description

OBJECTIVES: Primary * Determine the maximum tolerated dose of CEP-701 in pediatric patients with recurrent or refractory high-risk neuroblastoma. * Determine the dose-limiting toxicity of this drug in these patients. * Determine the pharmacokinetic behavior of this drug in these patients. Secondary * Determine the degree of TrkB tyrosine kinase inhibition activity present in the serum of patients treated with this drug. * Correlate the degree of TrkB tyrosine kinase inhibition activity in these patients with dose level, pharmacokinetics, and antitumor activity data of this drug. * Determine the antitumor activity of this drug in these patients. OUTLINE: This is an open-label, dose-escalation, multicenter study. Patients receive oral CEP-701 twice daily\* on days 1-5, 8-12, 15-19, and 22-26. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. NOTE: \*On day 1 of course 1 only, patients receive oral CEP-701 once instead of twice. Cohorts of 3-6 patients receive escalating doses of CEP-701 until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. Once the MTD is determined, the dose level is expanded up to 9 patients. PROJECTED ACCRUAL: A total of 60 patients will be accrued for this study.

Conditions

• Neuroblastoma

Interventions

Type	Name	Description
DRUG	lestaurtinib	Given orally twice daily x 5 consecutive days followed by a two day rest. 28 days = 1 treatment course. Courses repeated indefinitely without gap provided patient has recovered course from toxicities and no DLTs. Dose level assigned according to the planned dose escalation schedule.

Timeline

• Start date: 2003-08-01
• Primary completion: 2009-09-01
• Completion: 2011-02-01
• First posted: 2004-06-11
• Last updated: 2023-04-10

Locations

12 sites across 2 countries: United States, Canada