NCT00021229

Brief Summary

Phase I/II trial to estimate the maximum tolerated dose of imatinib mesylate in newly diagnosed brain stem gliomas and recurrent high grade gliomas and to assess the effectiveness of imatinib mesylate in treating young patients who have newly diagnosed intrinsic brain stem glioma. Imatinib mesylate may interfere with the growth of tumor cells by blocking the enzymes necessary for their growth. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining imatinib mesylate with radiation therapy may kill more tumor cells.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
85

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started May 2001

Longer than P75 for phase_1

Geographic Reach
1 country

10 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2001

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

July 11, 2001

Completed
1.5 years until next milestone

First Posted

Study publicly available on registry

January 27, 2003

Completed
5.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2008

Completed
1.7 years until next milestone

Results Posted

Study results publicly available

April 9, 2010

Completed
Last Updated

July 31, 2014

Status Verified

February 1, 2013

Enrollment Period

7.3 years

First QC Date

July 11, 2001

Results QC Date

February 9, 2010

Last Update Submit

July 25, 2014

Conditions

Keywords

childhood central nervous system germ cell tumorchildhood high-grade cerebral astrocytomauntreated childhood brain stem gliomarecurrent childhood brain stem gliomarecurrent childhood cerebral astrocytoma

Outcome Measures

Primary Outcomes (3)

  • Number of Participants in Phase I Stratum I With Dose Limiting Toxicities (DLT) Observed During First 8 Weeks (Courses 1 and 2) of Imatinib Therapy

    The dose limiting toxicity (DLT) analysis population consists of phase I stratum I participants who developed DLT during the maximum tolerated dose (MTD) estimation period (course 1 and 2) or who completed the MTD estimation period (courses 1 and 2) without DLTs. DLTs observed during courses 1 and 2 were used to estimate the MTD. The estimated MTD based on the 23 participants who either had a DLT during course 1 or 2 or completed courses 1 and 2 without DLT is 265 mg/m2/day.

    Day 1 of Imatinib Mesylate Therapy to Week 8

  • Number of Participants in Phase I Stratum II With Dose Limiting Toxicities (DLT) Observed During First 8 Weeks (Courses 1 and 2) of Imatinib Therapy

    The dose limiting toxicity (DLT) analysis population consisted of phase I stratum II participants who developed DLT during the maximum tolerated dose (MTD) estimation period (courses 1 and 2) or who completed the MTD estimation period (courses 1 and 2) without DLTs. DLTs observed during courses 1 and 2 were used to estimate the MTD. The estimated MTD based on the DLT analysis population of 20 in stratum IIA was 465 mg/m2/day. An MTD was not established in stratum IIB as no DLTs were observed at the higher dose levels of 620 and 800 mg/m2/day.

    Day 1 of Imatinib Mesylate Therapy to Week 8

  • Median Progression-free Survival (PFS)

    Progression-free survival is defined as the interval from initiation of treatment to the earliest of disease progression (tumor increase of 25% over baseline tumor measurement; appearance of new lesion(s); or progressive/worsening neurological status) or death for patients who failed, or to the last date of follow up for patients without failure.

    Assessed pre-radiation, before the first dose of imatinib, and then every 8 weeks

Secondary Outcomes (7)

  • Change From Baseline in Volume FLAIR at Two Weeks After Completion of Radiation

    Baseline and two weeks post completion of radiation

  • Peak Concentration (Cmax)

    Day 1 of Course 1

  • Median Overall Survival

    Assessed before radiation therapy, before the first dose of imatinib, then every 8 weeks.

  • Pre-treatment Basic Fibroblast Growth Factor Values From Urine

    Pre-treatment

  • Pre-treatment Basic Fibroblast Growth Factor Values From Plasma

    Pre-treatment

  • +2 more secondary outcomes

Study Arms (1)

Imatinib mesylate

EXPERIMENTAL
Drug: imatinib mesylateRadiation: local irradiation therapy

Interventions

* Phase 1 Stratum I: Starting dose level of 350 mg/m2/day every 28 days X 13 courses (dose escalation) * Phase I Stratum IIA: Starting dose level of 465 mg/m2/day every 28 days X 13 courses (dose escalation) * Phase I Stratum IIB: Starting dose level of 465 mg/m2/day every 28 days X 13 courses (dose escalation) * Phase II: Phase I Stratum I determined dose (Maximum tolerated dose) every 28 days X 13 courses.

Imatinib mesylate

* Phase I Stratum I: Total dose of 5580 cGy using conventional or conformal volume-based delivery techniques once daily, 5 days/week for six weeks prior to receiving imatinib. * Phase II: Total dose of 5580 cGy using conventional or conformal volume-based delivery techniques once daily, 5 days/week for six weeks prior to receiving imatinib.

Imatinib mesylate

Eligibility Criteria

Age3 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age 3 to 21
  • Performance status of Karnofsky 50-100% OR Lansky 50-100%
  • Absolute neutrophil count greater than 1,000/mm3
  • Platelet count greater than 100,000/mm3 (transfusion independent)
  • Hemoglobin greater than 8 g/dL (transfusion allowed)
  • Bilirubin no greater than 1.5 times normal for age
  • SGPT less than 3 times normal for age
  • Albumin at least 2 g/dL
  • Creatinine less than 1.5 times normal for age OR Glomerular filtration rate greater than 70 mL/min
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective barrier contraception during and for 6 months after study participation
  • Stratum I
  • Newly diagnosed diffuse intrinsic brainstem malignant glioma
  • No disseminated disease
  • +14 more criteria

You may not qualify if:

  • Receiving other anticancer or experimental drug therapy.
  • Ongoing uncontrolled infection.
  • Significant cardiac, hepatic, gastrointestinal, renal, pulmonary, or psychiatric disease.
  • Deep venous or arterial thrombosis within 6 weeks of registration.
  • Taking warfarin.
  • Newly diagnosed diffuse intrinsic brainstem malignant glioma with disseminated disease (stratum I)
  • Intratumoral hemorrhage

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

UCSF Comprehensive Cancer Center

San Francisco, California, 94143, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010-2970, United States

Location

Children's Memorial Hospital - Chicago

Chicago, Illinois, 60614, United States

Location

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

Boston, Massachusetts, 02115, United States

Location

Duke Comprehensive Cancer Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104-4318, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105-2794, United States

Location

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

Houston, Texas, 77030-2399, United States

Location

Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, 98105, United States

Location

Related Publications (2)

  • Williams G, Fahey FH, Treves ST, Kocak M, Pollack IF, Boyett JM, Kun LE, Poussaint TY. Exploratory evaluation of two-dimensional and three-dimensional methods of FDG PET quantification in pediatric anaplastic astrocytoma: a report from the Pediatric Brain Tumor Consortium (PBTC). Eur J Nucl Med Mol Imaging. 2008 Sep;35(9):1651-8. doi: 10.1007/s00259-008-0780-7. Epub 2008 Apr 19.

  • Pollack IF, Jakacki RI, Blaney SM, Hancock ML, Kieran MW, Phillips P, Kun LE, Friedman H, Packer R, Banerjee A, Geyer JR, Goldman S, Poussaint TY, Krasin MJ, Wang Y, Hayes M, Murgo A, Weiner S, Boyett JM. Phase I trial of imatinib in children with newly diagnosed brainstem and recurrent malignant gliomas: a Pediatric Brain Tumor Consortium report. Neuro Oncol. 2007 Apr;9(2):145-60. doi: 10.1215/15228517-2006-031. Epub 2007 Feb 9.

MeSH Terms

Conditions

Central Nervous System NeoplasmsAstrocytoma

Interventions

Imatinib Mesylate

Condition Hierarchy (Ancestors)

Nervous System NeoplasmsNeoplasms by SiteNeoplasmsNervous System DiseasesGliomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsBenzoatesAcids, CarbocyclicCarboxylic AcidsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPyrimidines

Limitations and Caveats

The phase II component of the trial was terminated because of poor accrual. Only one patient enrolled to the phase II component and did not receive the investigational drug.

Results Point of Contact

Title
Pediatric Brain Tumor Consortium (James M. Boyett, Ph.D)
Organization
Pediatric Brain Tumor Consortium

Study Officials

  • Ian F. Pollack, MD

    University of Pittsburgh

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 11, 2001

First Posted

January 27, 2003

Study Start

May 1, 2001

Primary Completion

August 1, 2008

Study Completion

August 1, 2008

Last Updated

July 31, 2014

Results First Posted

April 9, 2010

Record last verified: 2013-02

Locations