Trial Outcomes & Findings for Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas (NCT NCT01275586)
NCT ID: NCT01275586
Last Updated: 2017-04-12
Results Overview
To estimate the disease control rate (PD,SD, PR, CR) with Tasigna® in patients with neurofibromas (NF1) using standard RECIST criteria. Complete Response (CR) is defined as; disappearance of all target lesions. Partial Response (PR) is defined as at least a 30% decrease in the sum of the longest diameter of target lesions, taking as a reference the baseline sum longest diameter. Stable Disease (SD) is defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum longest diameter since the treatment started. Disease Progression (PD) is defined as at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or the appearance of one or more new lesions.
Recruitment status
COMPLETED
Study phase
EARLY_PHASE1
Target enrollment
6 participants
Primary outcome timeframe
6 months
Results posted on
2017-04-12
Participant Flow
Participant milestones
| Measure |
Tasigna
Following enrollment each subject will initially receive Tasigna orally at 200 mg twice daily for two weeks. If tolerated, the dose will be increased to 300 mg twice daily after a minimum of two weeks and will be increase to a maximum dose of 400mg twice daily after an additional two weeks if tolerated.
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|---|---|
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Overall Study
STARTED
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6
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Overall Study
COMPLETED
|
3
|
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Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
Baseline characteristics by cohort
| Measure |
Tasigna
n=6 Participants
Following enrollment each subject will initially receive Tasigna orally at 200 mg twice daily for two weeks. If tolerated, the dose will be increased to 300 mg twice daily after a minimum of two weeks and will be increase to a maximum dose of 400mg twice daily after an additional two weeks if tolerated.
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
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0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 6 monthsTo estimate the disease control rate (PD,SD, PR, CR) with Tasigna® in patients with neurofibromas (NF1) using standard RECIST criteria. Complete Response (CR) is defined as; disappearance of all target lesions. Partial Response (PR) is defined as at least a 30% decrease in the sum of the longest diameter of target lesions, taking as a reference the baseline sum longest diameter. Stable Disease (SD) is defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum longest diameter since the treatment started. Disease Progression (PD) is defined as at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or the appearance of one or more new lesions.
Outcome measures
| Measure |
Tasigna
n=3 Participants
Following enrollment each subject will initially receive Tasigna orally at 200 mg twice daily for two weeks. If tolerated, the dose will be increased to 300 mg twice daily after a minimum of two weeks and will be increase to a maximum dose of 400mg twice daily after an additional two weeks if tolerated.
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|---|---|
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Disease Response
Progressive Disease (PD)
|
1 Participants
|
|
Disease Response
Complete Response (CR)
|
0 Participants
|
|
Disease Response
Partial Response (PR)
|
0 Participants
|
|
Disease Response
Stable Disease (SD)
|
2 Participants
|
Adverse Events
Tasigna
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Tasigna
n=6 participants at risk
Following enrollment each subject will initially receive Tasigna orally at 200 mg twice daily for two weeks. If tolerated, the dose will be increased to 300 mg twice daily after a minimum of two weeks and will be increase to a maximum dose of 400mg twice daily after an additional two weeks if tolerated.
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|---|---|
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Investigations
Elevated Lipase
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
Other adverse events
| Measure |
Tasigna
n=6 participants at risk
Following enrollment each subject will initially receive Tasigna orally at 200 mg twice daily for two weeks. If tolerated, the dose will be increased to 300 mg twice daily after a minimum of two weeks and will be increase to a maximum dose of 400mg twice daily after an additional two weeks if tolerated.
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|---|---|
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Eye disorders
Conjunctivitis
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
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Gastrointestinal disorders
Nausea
|
66.7%
4/6 • Number of events 4 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
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|
Gastrointestinal disorders
dyspepsia
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
General disorders
fatigue
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
General disorders
pain
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Infections and infestations
scrotal infection
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Investigations
Blood Bilirubin increased
|
50.0%
3/6 • Number of events 3 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Investigations
Alanine Aminotransferase Increased
|
33.3%
2/6 • Number of events 2 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Investigations
Elevated Amylase
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Investigations
Elevated Lipase
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Investigations
Weight Loss
|
33.3%
2/6 • Number of events 2 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
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Metabolism and nutrition disorders
Hypokalemia
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Musculoskeletal and connective tissue disorders
Myalagia
|
33.3%
2/6 • Number of events 2 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
33.3%
2/6 • Number of events 2 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Musculoskeletal and connective tissue disorders
Pain in chest wall
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
50.0%
3/6 • Number of events 3 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Musculoskeletal and connective tissue disorders
Join Pain
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
50.0%
3/6 • Number of events 3 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Renal and urinary disorders
Urinary Incontinence
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Renal and urinary disorders
Bladder Infection
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Renal and urinary disorders
Kidney Infection
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Renal and urinary disorders
Bladder Spasm
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.7%
1/6 • Number of events 1 • adverse event data was collected while participants were on study treatment and for 30 days after last day of study treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place