N2003-01: Irinotecan, Temozolomide, and Cefixime in Treating Young Patients With Recurrent or Resistant Neuroblastoma

NCT ID: NCT00093353

Last Updated: 2010-10-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-05-31

Brief Summary

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RATIONALE: Drugs used in chemotherapy, such as irinotecan and temozolomide, work in different ways to stop tumor cells from dividing so they stop growing or die. Temozolomide may help irinotecan kill more tumor cells by making them more sensitive to the drug. Cefixime may be effective in preventing diarrhea that is caused by treatment with irinotecan.

PURPOSE: This phase I trial is studying the side effects and best dose of irinotecan when given together with temozolomide and cefixime in treating young patients with recurrent or resistant neuroblastoma.

Detailed Description

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OBJECTIVES:

Primary

* Determine the maximum tolerated dose of oral irinotecan when administered with fixed-dose temozolomide and cefixime in pediatric patients with recurrent or resistant high-risk neuroblastoma.
* Determine the toxic effects of this regimen in these patients.

Secondary

* Determine the response rate in patients treated with this regimen.
* Determine the pharmacokinetics of this regimen in these patients.
* Correlate UGT1A1 genotype with the occurrence of dose-limiting diarrhea in patients treated with this regimen.
* Correlate BCRP genotype with pharmacokinetic phenotype in patients treated with this regimen.
* Correlate p53 status in tumor cells with response in patients treated with this regimen.

OUTLINE: This is a multicenter, dose-escalation study of irinotecan.

Patients receive oral cefixime once daily beginning 5 days before the start of fixed-dose temozolomide and irinotecan and continuing for the duration of the study. Patients also receive oral temozolomide once daily on days 1-5 and oral irinotecan once daily on days 1-5 and 8-12. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of irinotecan 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. A maximum of 12 patients are treated at the MTD.

Patients are followed for toxicity, response, and survival.

PROJECTED ACCRUAL: A total of 15-30 patients will be accrued for this study within 1.25 years.

Conditions

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Diarrhea Drug/Agent Toxicity by Tissue/Organ Neuroblastoma

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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cefixime

Intervention Type DRUG

irinotecan hydrochloride

Intervention Type DRUG

temozolomide

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

DISEASE CHARACTERISTICS:

* Histologically confirmed neuroblastoma AND/OR demonstration of tumor cells in the bone marrow with increased urinary catecholamines

* High-risk disease meeting 1 of the following criteria:

* Recurrent or progressive disease
* Resistant or refractory disease (i.e., never achieved a complete response to therapy AND never had new sites of disease or progression of initial sites)
* Measurable disease meeting at least 1 of the following criteria:

* Unidimensionally measurable tumor ≥ 20 mm by MRI, CT scan, or x-ray OR ≥ 10 mm by spiral CT scan\*
* At least 1 site with positive uptake by metaiodobenzylguanidine (MIBG) scan\*
* Bone marrow with tumor cells seen on routine morphology (not by NSE staining only) of bilateral aspirate AND/OR biopsy on 1 bone marrow sample NOTE: \*Patients who never experienced disease recurrence or progression must demonstrate viable neuroblastoma in a biopsy of either bone marrow or bone and/or soft tissue site (biopsy must be performed ≥ 4 weeks after completion of prior radiotherapy if lesion was irradiated)

PATIENT CHARACTERISTICS:

Age

* 1 to 30 at diagnosis

Performance status

* ECOG 0-2

Life expectancy

* At least 2 months

Hematopoietic

* Absolute neutrophil count ≥ 750/mm\^3
* Platelet count ≥ 75,000/mm\^3 (without transfusion)
* Hemoglobin ≥ 8.0 g/dL (transfusion allowed)

Hepatic

* SGPT and SGOT \< 5 times normal
* Bilirubin ≤ 1.5 times normal

Renal

* Creatinine ≤ 1.5 times normal for age

* No greater than 0.8 mg/dL (≤ 5 years of age)
* No greater than 1.0 mg/dL (6 to 10 years of age)
* No greater than 1.2 mg/dL (11 to 15 years of age)
* No greater than 1.5 mg/dL (\> 15 years of age)

Other

* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* No allergy to cephalosporins
* No active diarrhea
* No uncontrolled infection

PRIOR CONCURRENT THERAPY:

Biologic therapy

* See Chemotherapy
* Recovered from prior immunotherapy
* More than 3 weeks since prior biologic therapy and recovered
* More than 2 days since prior hematopoietic growth factors
* No concurrent epoetin alfa
* No concurrent prophylactic hematopoietic growth factors during the first treatment course
* No concurrent immunomodulating agents except steroids to control intracranial pressure

Chemotherapy

* Prior myeloablative therapy and autologous stem cell transplantation allowed

* No prior allogeneic stem cell transplantation
* More than 3 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered
* Prior temozolomide, irinotecan, or topotecan allowed

* No prior temozolomide and irinotecan as combination therapy
* No other concurrent chemotherapy

Endocrine therapy

* See Biologic therapy

Radiotherapy

* At least 6 weeks since prior large field radiotherapy (e.g., total body irradiation, craniospinal therapy, whole abdomen, total lung, or \> 50% bone marrow space) and recovered
* At least 4 weeks since prior radiotherapy to biopsied lesions (for study entry) and recovered
* At least 6 weeks since prior MIBG therapy
* Concurrent radiotherapy to painful lesions allowed provided the lesions are not used to assess treatment response

Surgery

* Not specified

Other

* No concurrent enzyme-inducing anticonvulsants (e.g., phenobarbital, phenytoin, or carbamazepine)
* No other concurrent anticancer agents
Minimum Eligible Age

1 Year

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Children's Hospital Los Angeles

OTHER

Sponsor Role lead

Responsible Party

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Cincinnati Children's Hospital Medical Center

Principal Investigators

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Lars M. Wagner, MD

Role: STUDY_CHAIR

Children's Hospital Medical Center, Cincinnati

Katherine K. Matthay, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Locations

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Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Lucile Packard Children's Hospital at Stanford University Medical Center

Palo Alto, California, United States

Site Status

UCSF Comprehensive Cancer Center

San Francisco, California, United States

Site Status

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

Atlanta, Georgia, United States

Site Status

Children's Memorial Hospital - Chicago

Chicago, Illinois, United States

Site Status

Indiana University Cancer Center

Indianapolis, Indiana, United States

Site Status

Children's Hospital Boston

Boston, Massachusetts, United States

Site Status

Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital

Houston, Texas, United States

Site Status

Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, United States

Site Status

Countries

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United States

References

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Wagner LM, Villablanca JG, Stewart CF, Crews KR, Groshen S, Reynolds CP, Park JR, Maris JM, Hawkins RA, Daldrup-Link HE, Jackson HA, Matthay KK. Phase I trial of oral irinotecan and temozolomide for children with relapsed high-risk neuroblastoma: a new approach to neuroblastoma therapy consortium study. J Clin Oncol. 2009 Mar 10;27(8):1290-6. doi: 10.1200/JCO.2008.18.5918. Epub 2009 Jan 26.

Reference Type RESULT
PMID: 19171709 (View on PubMed)

Other Identifiers

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P01CA081403

Identifier Type: NIH

Identifier Source: secondary_id

View Link

N2003-01

Identifier Type: OTHER

Identifier Source: secondary_id

CDR0000373759

Identifier Type: -

Identifier Source: org_study_id