Trial Outcomes & Findings for Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas (NCT NCT01140360)

Last Updated: 2017-07-02

Results Overview

To estimate the disease control rate (SD, PR, CR) with Gleevec/Imatinib Mesylate in patients with neurofibromas (NF1). The sum of the longest diameter (LD) for all target lesions will be calculated and reported as the disease measurement. Complete Response (CR) disappearance of target lesions. Partial Response (PR) at least a 30% decrease in the disease measurement, taking as reference the disease measurement done to confirm measurable disease at study entry. Progressive Disease (PD) at least a 20% increase in the disease measurement, taking as reference the smallest disease measurement recorded since the start of treatment. Stable Disease (SD) neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as reference the smallest disease measurement since the treatment started.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

21 participants

Primary outcome timeframe

1 year

Results posted on

2017-07-02

Participant Flow

Participant milestones

Participant milestones
Measure	Gleevec Gleevec will be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA \> 1.8 m2 or 55 mg/m2 twice daily for patients with BSA \< 1.8 m2. For patients with a BSA \> 1.8 m2 the dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum dose of 400 mg bid. For patients with a BSA \< 1.8 m2 the dose will increase by increments of 55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid.Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.
Overall Study STARTED	21
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Gleevec n=21 Participants Gleevec will be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA \> 1.8 m2 or 55 mg/m2 twice daily for patients with BSA \< 1.8 m2. For patients with a BSA \> 1.8 m2 the dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum dose of 400 mg bid. For patients with a BSA \< 1.8 m2 the dose will increase by increments of 55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid.Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.
Age, Categorical <=18 years	10 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	14 Participants n=93 Participants
Sex: Female, Male Male	7 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants
Race (NIH/OMB) White	19 Participants n=93 Participants
Race (NIH/OMB) More than one race	1 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	21 participants n=93 Participants

PRIMARY outcome

Timeframe: 1 year

Population: 13 participants reached the 1 year evaluation time-point. 6 participants withdrew prior to the 1 year evaluation time-point.

Outcome measures

Outcome measures
Measure	Gleevec n=13 Participants Gleevec will be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA \> 1.8 m2 or 55 mg/m2 twice daily for patients with BSA \< 1.8 m2. For patients with a BSA \> 1.8 m2 the dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum dose of 400 mg bid. For patients with a BSA \< 1.8 m2 the dose will increase by increments of 55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid.Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.
Disease Response Stable Disease (SD)	13 Participants
Disease Response Partial Response (PR)	0 Participants
Disease Response Complete Response (CR)	0 Participants
Disease Response Progressive Disease (PD)	0 Participants

Adverse Events

Gleevec

Serious events: 3 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Gleevec n=21 participants at risk Gleevec will be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA \> 1.8 m2 or 55 mg/m2 twice daily for patients with BSA \< 1.8 m2. For patients with a BSA \> 1.8 m2 the dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum dose of 400 mg bid. For patients with a BSA \< 1.8 m2 the dose will increase by increments of 55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid.Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.
Vascular disorders Pseudoaneurysm of carotid artery	4.8% 1/21 • Number of events 1 • 1 year
Metabolism and nutrition disorders Dehydration	4.8% 1/21 • Number of events 1 • 1 year
Hepatobiliary disorders Hepatic Failure	4.8% 1/21 • Number of events 1 • 1 year

Other adverse events

Other adverse events
Measure	Gleevec n=21 participants at risk Gleevec will be dosed orally with a starting dose of 100 mg twice daily for patients with a BSA \> 1.8 m2 or 55 mg/m2 twice daily for patients with BSA \< 1.8 m2. For patients with a BSA \> 1.8 m2 the dose will increase by increments of 100 mg bid every two weeks as tolerated up to a maximum dose of 400 mg bid. For patients with a BSA \< 1.8 m2 the dose will increase by increments of 55 mg/m2 bid every two weeks as tolerated up to a maximum dose of 220 mg/m2 bid.Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.
Skin and subcutaneous tissue disorders alopecia	9.5% 2/21 • Number of events 2 • 1 year
Investigations Alanine aminotransferase increased	14.3% 3/21 • Number of events 4 • 1 year
Investigations Neutrophil count decreased	33.3% 7/21 • Number of events 10 • 1 year
Blood and lymphatic system disorders anemia	42.9% 9/21 • Number of events 11 • 1 year
Metabolism and nutrition disorders anorexia	14.3% 3/21 • Number of events 3 • 1 year
Investigations Aspartate aminotransferase increased	14.3% 3/21 • Number of events 6 • 1 year
General disorders Back Pain	14.3% 3/21 • Number of events 4 • 1 year
Investigations blood bilirubin increased	14.3% 3/21 • Number of events 4 • 1 year
Metabolism and nutrition disorders hypocalcemia	9.5% 2/21 • Number of events 3 • 1 year
Gastrointestinal disorders Constipation	14.3% 3/21 • Number of events 3 • 1 year
Gastrointestinal disorders Diarrhea	14.3% 3/21 • Number of events 5 • 1 year
Nervous system disorders dizziness	9.5% 2/21 • Number of events 2 • 1 year
Gastrointestinal disorders dysphagia	9.5% 2/21 • Number of events 3 • 1 year
Respiratory, thoracic and mediastinal disorders dyspnea	9.5% 2/21 • Number of events 2 • 1 year
General disorders edema limbs	23.8% 5/21 • Number of events 6 • 1 year
General disorders fatigue	33.3% 7/21 • Number of events 9 • 1 year
Nervous system disorders Headache	28.6% 6/21 • Number of events 6 • 1 year
Skin and subcutaneous tissue disorders puritits	9.5% 2/21 • Number of events 2 • 1 year
Blood and lymphatic system disorders Lymphopenia	9.5% 2/21 • Number of events 4 • 1 year
Gastrointestinal disorders Nausea	47.6% 10/21 • Number of events 16 • 1 year
Musculoskeletal and connective tissue disorders Neck pain	14.3% 3/21 • Number of events 4 • 1 year
Metabolism and nutrition disorders hypokalemia	23.8% 5/21 • Number of events 9 • 1 year
Renal and urinary disorders Urinary frequency	14.3% 3/21 • Number of events 3 • 1 year
Gastrointestinal disorders vomitting	19.0% 4/21 • Number of events 4 • 1 year
Investigations white blood cell decreased	52.4% 11/21 • Number of events 16 • 1 year
Investigations weight loss	14.3% 3/21 • Number of events 3 • 1 year

Additional Information

Dr. Kent Robertson

Indiana University

Phone: 317-944-4969

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place