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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)

Tracking Information
First Submitted Date  ICMJE July 11, 2001
First Posted Date  ICMJE January 27, 2003
Last Update Posted Date April 15, 2015
Study Start Date  ICMJE November 2001
Actual Primary Completion Date December 2005   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 15, 2013)
  • MTD defined as the dose at which fewer than 20% of patients experience DLT assessed using CTC version 2.0 [ Time Frame: 3 weeks ]
  • 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.
  • Progression free survival [ Time Frame: Up to 24 months ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Bortezomib in Treating Children With Advanced Solid Tumors
Official Title  ICMJE A Phase I Study Of PS-341 In Pediatric Patients With Refractory Solid Tumors
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Unspecified Childhood Solid Tumor, Protocol Specific
Intervention  ICMJE
  • Drug: bortezomib
    Given IV
    Other Names:
    • LDP 341
    • MLN341
  • Other: laboratory biomarker analysis
    Correlative studies
  • Other: pharmacological study
    Correlative studies
    Other Name: pharmacological studies
Study Arms  ICMJE 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
  • Other: laboratory biomarker analysis
  • Other: pharmacological study
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 15, 2013)
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE December 2005
Actual Primary Completion Date December 2005   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries Australia,   Canada
Administrative Information
NCT Number  ICMJE NCT00021216
Other Study ID Numbers  ICMJE 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 )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party National Cancer Institute (NCI)
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE National Cancer Institute (NCI)
Original Study Sponsor  ICMJE Children's Oncology Group
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Susan Blaney Children's Oncology Group
PRS Account National Cancer Institute (NCI)
Verification Date December 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP