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

Phase 1
Completed
Conditions
Neuroblastoma
Interventions
Registration Number
NCT00084422
Lead Sponsor
New Approaches to Neuroblastoma Therapy Consortium
Brief 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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
47
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Single Grouplestaurtinib-
Primary Outcome Measures
NameTimeMethod
To determine dose limiting toxicities (DLTs) of CEP-701 given on this scheduleWithin first 28 days of therapy.
To characterize the pharmacokinetic (PK) behavior of CEP-701 in children with residual or refractory high-risk neuroblastoma.Days 1,5 and 26 of first course only.

Participation in PK studies is voluntary and not a requirement for study entry.

To determine the maximum tolerated dose (MTD) of CEP-701 given on a twice daily chronic administration schedule (two days on , two days off) to children with high risk relapsed or residual neuroblastoma.Within 28 days of treatment at each dose level.
Secondary Outcome Measures
NameTimeMethod
To determine the degree of TrkB tyrosine kinase inhibition activity present in the serum of patients treated with CEP-701, and correlate these findings with dose level, pharmacokinetic and anti-tumor activity data.Days 1,5 and 26 of first course only.
To define the antitumor activity of CEP-701, within the confines of a Phase I study.Evaluation at end of courses 1, 2, 4 and then every 4 courses until patient goes off therapy.

Trial Locations

Locations (12)

Childrens Hospital Los Angeles

🇺🇸

Los Angeles, California, United States

Lucille Salter Packer Children's Hospital, Stanford University

🇺🇸

Palo Alto, California, United States

UCSF Helen Diller Family Comprehensive Cancer Center

🇺🇸

San Francisco, California, United States

AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus

🇺🇸

Atlanta, Georgia, United States

University of Chicago Comer Children's Hospital

🇺🇸

Chicago, Illinois, United States

Children's Hospital Boston

🇺🇸

Boston, Massachusetts, United States

University of Michigan Comprehensive Cancer Center

🇺🇸

Ann Arbor, Michigan, United States

Morgan Stanley Children's Hospital of New York-Presbyterian

🇺🇸

New York, New York, United States

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

🇺🇸

Philadelphia, Pennsylvania, United States

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Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States

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