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Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas

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ClinicalTrials.gov Identifier: NCT01673009
Recruitment Status : Completed
First Posted : August 27, 2012
Results First Posted : April 6, 2016
Last Update Posted : April 6, 2016
Sponsor:
Information provided by (Responsible Party):
Indiana University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Neurofibromatosis
Intervention Drug: Gleevec
Enrollment 36

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Administration of Gleevec
Hide Arm/Group Description

Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Gleevec: Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Period Title: Overall Study
Started 36
Completed 23
Not Completed 13
Reason Not Completed
Physician Decision             5
Withdrawal by Subject             8
Arm/Group Title Administration of Gleevec
Hide Arm/Group Description

Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Gleevec: Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Overall Number of Baseline Participants 36
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
<=18 years
25
  69.4%
Between 18 and 65 years
11
  30.6%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
Female
17
  47.2%
Male
19
  52.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
Hispanic or Latino
2
   5.6%
Not Hispanic or Latino
34
  94.4%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   2.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
   5.6%
White
32
  88.9%
More than one race
0
   0.0%
Unknown or Not Reported
1
   2.8%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 36 participants
36
1.Primary Outcome
Title Percent Change From Baseline in Tumor Volume at 6 Months
Hide Description Volumetric measures were performed using MRI scan analysis. Response criteria include greater than 20 percent decrease in tumor volume as responsive. Greater than 20 percent increase in tumor volume as tumor progression. Less then 20 percent increase or decrease in tumor volume is stable disease
Time Frame baseline to 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Administration of Gleevec
Hide Arm/Group Description:

Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Gleevec: Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Overall Number of Participants Analyzed 23
Median (95% Confidence Interval)
Unit of Measure: percentage change of tumor volume
17
(6 to 33)
2.Secondary Outcome
Title Serum Bioactivity
Hide Description The investigators will quantitate the biologic activity of patient serum on fibroblast proliferation, migration, and collagen synthesis pre and post-Gleevec (7 days and 1 month)
Time Frame 7 days and 1 month
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
unable to measure this outcome because assay became unavailable
Arm/Group Title Administration of Gleevec
Hide Arm/Group Description:

Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Gleevec: Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Administration of Gleevec
Hide Arm/Group Description

Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

Gleevec: Gleevec® will be dosed orally 440 mg/m2/day (max 800 mg/day) for pediatric subjects and 800 mg/day for adult patients.

All-Cause Mortality
Administration of Gleevec
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Administration of Gleevec
Affected / at Risk (%) # Events
Total   1/36 (2.78%)    
Hepatobiliary disorders   
liver dysfunction/failure  1  1/36 (2.78%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Administration of Gleevec
Affected / at Risk (%) # Events
Total   12/36 (33.33%)    
General disorders   
Edema  1  3/36 (8.33%)  4
Investigations   
Neutropenia  1  2/36 (5.56%)  2
Nervous system disorders   
Pain  1  3/36 (8.33%)  4
Skin and subcutaneous tissue disorders   
Rash  1  4/36 (11.11%)  5
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Kent Robertson MD PhD
Organization: Indiana University
Phone: 317-944-8784
Responsible Party: Indiana University
ClinicalTrials.gov Identifier: NCT01673009     History of Changes
Other Study ID Numbers: 0512-25
First Submitted: August 22, 2012
First Posted: August 27, 2012
Results First Submitted: February 24, 2016
Results First Posted: April 6, 2016
Last Update Posted: April 6, 2016