Trial Outcomes & Findings for Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas (NCT NCT01673009)

Last Updated: 2016-04-06

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

36 participants

Primary outcome timeframe

baseline to 6 months

Results posted on

2016-04-06

Participant Flow

Participant milestones

Participant milestones
Measure	Administration of Gleevec 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 Study STARTED	36
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Administration of Gleevec 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 Study Physician Decision	5
Overall Study Withdrawal by Subject	8

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Administration of Gleevec n=36 Participants 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.
Age, Categorical <=18 years	25 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	17 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	32 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	36 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline to 6 months

Outcome measures

Outcome measures
Measure	Administration of Gleevec n=23 Participants 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.
Percent Change From Baseline in Tumor Volume at 6 Months	17 percentage change of tumor volume Interval 6.0 to 33.0

SECONDARY outcome

Timeframe: 7 days and 1 month

Population: unable to measure this outcome because assay became unavailable

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)

Outcome measures

Outcome data not reported

Adverse Events

Administration of Gleevec

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Administration of Gleevec n=36 participants at risk 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.
Hepatobiliary disorders liver dysfunction/failure	2.8% 1/36 • Number of events 1 • 6 months

Other adverse events

Other adverse events
Measure	Administration of Gleevec n=36 participants at risk 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.
Skin and subcutaneous tissue disorders Rash	11.1% 4/36 • Number of events 5 • 6 months
General disorders Edema	8.3% 3/36 • Number of events 4 • 6 months
Nervous system disorders Pain	8.3% 3/36 • Number of events 4 • 6 months
Investigations Neutropenia	5.6% 2/36 • Number of events 2 • 6 months

Additional Information

Kent Robertson MD PhD

Indiana University

Phone: 317-944-8784

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place