N2001-03: CEP-701 in Treating Young Patients With Recurrent or Refractory High-Risk Neuroblastoma
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00084422|
Recruitment Status : Completed
First Posted : June 11, 2004
Last Update Posted : August 29, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|Neuroblastoma||Drug: lestaurtinib||Phase 1|
- 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.
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||47 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study Of CEP-701 In Patients With Refractory Neuroblastoma (IND # 67,722)|
|Study Start Date :||August 2003|
|Actual Primary Completion Date :||September 2009|
|Actual Study Completion Date :||February 2011|
- 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.Other Name: CEP-701
- 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. [ Time Frame: Within 28 days of treatment at each dose level. ]
- To determine dose limiting toxicities (DLTs) of CEP-701 given on this schedule [ Time Frame: Within first 28 days of therapy. ]
- To characterize the pharmacokinetic (PK) behavior of CEP-701 in children with residual or refractory high-risk neuroblastoma. [ Time Frame: 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 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. [ Time Frame: 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. [ Time Frame: Evaluation at end of courses 1, 2, 4 and then every 4 courses until patient goes off therapy. ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00084422
|United States, California|
|Childrens Hospital Los Angeles|
|Los Angeles, California, United States, 90027-0700|
|Lucille Salter Packer Children's Hospital, Stanford University|
|Palo Alto, California, United States, 94305|
|UCSF Helen Diller Family Comprehensive Cancer Center|
|San Francisco, California, United States, 94143|
|United States, Georgia|
|AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|University of Chicago Comer Children's Hospital|
|Chicago, Illinois, United States, 60637|
|United States, Massachusetts|
|Children's Hospital Boston|
|Boston, Massachusetts, United States, 02115|
|United States, Michigan|
|University of Michigan Comprehensive Cancer Center|
|Ann Arbor, Michigan, United States, 48109|
|United States, New York|
|Morgan Stanley Children's Hospital of New York-Presbyterian|
|New York, New York, United States, 10032|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229-3039|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104-4318|
|United States, Washington|
|Children's Hospital and Regional Medical Center - Seattle|
|Seattle, Washington, United States, 98105|
|Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Principal Investigator:||John M. Maris, MD||Children's Hospital of Philadelphia|
|Study Chair:||Garrett M. Brodeur, MD||Children's Hospital of Philadelphia|