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Bortezomib in Treating Children With Advanced Solid Tumors

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. Identifier: NCT00021216
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : April 15, 2015
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Brief Summary:
Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Phase I trial to study the effectiveness of bortezomib in treating children who have advanced solid tumors that have not responded to previous treatment.

Condition or disease Intervention/treatment Phase
Unspecified Childhood Solid Tumor, Protocol Specific Drug: bortezomib Other: laboratory biomarker analysis Other: pharmacological study Phase 1

Detailed Description:


I. Determine the maximum tolerated dose of bortezomib in pediatric patients with refractory solid tumors.

II. Determine the dose-limiting toxicity and other toxic effects of this regimen in these patients.

III. Preliminarily determine the antitumor activity of this regimen in these patients.

OUTLINE: This is a dose-escalation study.

Patients receive bortezomib IV on days 1, 4, 8, and 11. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of bortezomib 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. If dose-limiting toxicity in the form of myelosuppression occurs in stratum I, dose escalation continues with patients meeting the qualifications for stratum II.

PROJECTED ACCRUAL: Approximately 24-36 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study Of PS-341 In Pediatric Patients With Refractory Solid Tumors
Study Start Date : November 2001
Actual Primary Completion Date : December 2005
Actual Study Completion Date : December 2005

Resource links provided by the National Library of Medicine

Drug Information available for: Bortezomib

Arm Intervention/treatment
Experimental: Treatment (bortezomib)
Patients receive bortezomib IV on days 1, 4, 8, and 11. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Drug: bortezomib
Given IV
Other Names:
  • LDP 341
  • MLN341

Other: laboratory biomarker analysis
Correlative studies

Other: pharmacological study
Correlative studies
Other Name: pharmacological studies

Primary Outcome Measures :
  1. MTD defined as the dose at which fewer than 20% of patients experience DLT assessed using CTC version 2.0 [ Time Frame: 3 weeks ]
  2. 20S proteasome inhibition [ Time Frame: Up to 2 weeks ]
    The 95% confidence interval for the percent of patients who exhibit inhibition at a recommended dose level determined according to the table above is 61%-100%, if six patients are evaluated or 55%-100% if five patients are evaluated.

  3. Progression free survival [ Time Frame: Up to 24 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Histologically confirmed solid tumor that is refractory to standard therapy or for which no standard therapy exists

    • Histologic confirmation not required for brainstem glioma or optic pathway tumor
  • Ineligible for therapies of higher priority
  • Stratum II only:

    • No bone marrow involvement
  • Performance status - Karnofsky 50-100% (over 10 years of age)
  • Performance status - Lansky 50-100% (10 years of age and under)
  • At least 8 weeks
  • Absolute neutrophil count at least 1,500/mm^3
  • Platelet count at least 75,000/mm^3 (transfusion independent)
  • Hemoglobin at least 8 g/dL (RBC transfusions allowed)
  • Bilirubin less than 1.5 mg/dL
  • ALT less than 5 times normal for age
  • Albumin at least 2 g/dL
  • Creatinine no greater than upper limit of normal for age
  • Creatinine clearance or radioisotope glomerular filtration rate greater than 70 mL/min
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Neurologic deficits related to CNS tumors allowed if relatively stable for at least 2 weeks
  • No uncontrolled infection
  • At least 7 days since prior biologic therapy and recovered
  • At least 3 months since prior allogeneic stem cell transplantation
  • At least 1 week since prior growth factors
  • Stratum II only:

    • No prior stem cell transplantation with or without total body irradiation
  • At least 2 weeks since prior chemotherapy (4 weeks for nitrosoureas) and recovered
  • Stratum II only:

    • No more than 2 prior multi-agent chemotherapy regimens
    • More than 2 single-agent regimens allowed
  • Concurrent dexamethasone allowed for CNS tumors if stable dose for at least 2 weeks
  • See Biologic therapy
  • At least 2 weeks since prior palliative local radiotherapy
  • At least 6 months since prior craniospinal radiotherapy or radiotherapy to at least 50% of pelvis
  • At least 6 weeks since prior substantial bone marrow radiotherapy
  • Recovered from prior radiotherapy
  • Stratum II only:

    • No prior radiotherapy to more than 20% of bone marrow
  • No prior bortezomib
  • No concurrent anticonvulsants
  • No other concurrent investigational agents

Information from the National Library of Medicine

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 identifier (NCT number): NCT00021216

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United States, California
Children's Oncology Group
Arcadia, California, United States, 91006-3776
Sponsors and Collaborators
National Cancer Institute (NCI)
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Principal Investigator: Susan Blaney Children's Oncology Group
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Responsible Party: National Cancer Institute (NCI) Identifier: NCT00021216    
Other Study ID Numbers: NCI-2012-01860
NCI-2012-01860 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
ADVL0015 ( Other Identifier: Children's Oncology Group )
ADVL0015 ( Other Identifier: CTEP )
U01CA097452 ( U.S. NIH Grant/Contract )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: April 15, 2015
Last Verified: December 2013
Additional relevant MeSH terms:
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Antineoplastic Agents