Trial Outcomes & Findings for Nilotinib in Advanced Gastrointestinal Stromal Tumors (GIST) (NCT NCT00782834)
NCT ID: NCT00782834
Last Updated: 2013-04-22
Results Overview
Number of participants that demonstrate progression free survival at 6 months
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
6 months
Results posted on
2013-04-22
Participant Flow
Patients were accrued between 7/03/2008 and 9/24/2009 at Fox Chase Cancer Center outpatient medical oncology clinics.
Advanced GIST with measurable disease; previously treated with at least imatinib and sunitinib; a 5-day washout from prior tyrosine kinase inhibitor therapy. Normal cardiac function with LVEF 45% or \>, no history of significant heart disease,arrhythmias,congenital long QT sydrome, heart block, MI within 12 mos of visit 1 or unstable angina, CHF
Participant milestones
| Measure |
Nilotinib Arm
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Screened Patients
STARTED
|
15
|
|
Screened Patients
COMPLETED
|
14
|
|
Screened Patients
NOT COMPLETED
|
1
|
|
Treatment Phase
STARTED
|
14
|
|
Treatment Phase
COMPLETED
|
13
|
|
Treatment Phase
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Nilotinib Arm
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Treatment Phase
fibrous tumor not GIST
|
1
|
Baseline Characteristics
Nilotinib in Advanced Gastrointestinal Stromal Tumors (GIST)
Baseline characteristics by cohort
| Measure |
Nilotinib Arm
n=15 Participants
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Age Categorical
<=18 years
|
0 participants
n=5 Participants
|
|
Age Categorical
Between 18 and 65 years
|
9 participants
n=5 Participants
|
|
Age Categorical
>=65 years
|
4 participants
n=5 Participants
|
|
Gender
Female
|
4 participants
n=5 Participants
|
|
Gender
Male
|
9 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=5 Participants
|
|
Primary Site of Cancer
Stomach
|
6 Participants
n=5 Participants
|
|
Primary Site of Cancer
Small Bowel
|
5 Participants
n=5 Participants
|
|
Primary Site of Cancer
Colon
|
1 Participants
n=5 Participants
|
|
Primary Site of Cancer
Rectum
|
1 Participants
n=5 Participants
|
|
Prior Tyrosine Kinase Inhibitor (TKI)
IM and SU
|
11 Participants
n=5 Participants
|
|
Prior Tyrosine Kinase Inhibitor (TKI)
IM, SU and NA
|
1 Participants
n=5 Participants
|
|
Prior Tyrosine Kinase Inhibitor (TKI)
IM, SU, RT and CT
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsNumber of participants that demonstrate progression free survival at 6 months
Outcome measures
| Measure |
Nilotinib Arm
n=13 Participants
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Progression Free Survival Rate at 6 Months
|
13 Participants
|
PRIMARY outcome
Timeframe: 1yearResponse rate of nilotinib by RECIST criteria evaluated every 2 months for the first 6 months then every 3 months for the duration of treatment period.
Outcome measures
| Measure |
Nilotinib Arm
n=13 Participants
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Response Rate
|
5 Participants
|
Adverse Events
Nilotinib Arm
Serious events: 12 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Nilotinib Arm
n=13 participants at risk
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Gastrointestinal disorders
Pain associated with constipation
|
15.4%
2/13 • Number of events 2
|
|
Gastrointestinal disorders
Gastric ulcer
|
7.7%
1/13 • Number of events 1
|
|
Gastrointestinal disorders
Nausea vomiting weakness followed by death
|
7.7%
1/13 • Number of events 1
|
|
General disorders
Profound weakness
|
7.7%
1/13 • Number of events 1
|
|
General disorders
Death
|
15.4%
2/13 • Number of events 2
|
|
Gastrointestinal disorders
Severe abdominal pain
|
7.7%
1/13 • Number of events 1
|
|
Blood and lymphatic system disorders
Severe anemia
|
7.7%
1/13 • Number of events 1
|
|
Psychiatric disorders
Altered mental status dec pulse ox
|
7.7%
1/13 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
7.7%
1/13 • Number of events 1
|
|
Renal and urinary disorders
Acute azotemia
|
7.7%
1/13 • Number of events 1
|
Other adverse events
| Measure |
Nilotinib Arm
n=13 participants at risk
All patients treated with nilotinib 400 mg BID orally daily; 4 weeks = one cycle
|
|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
46.2%
6/13 • Number of events 7
|
|
Blood and lymphatic system disorders
Neutropenia
|
15.4%
2/13 • Number of events 3
|
|
Blood and lymphatic system disorders
Anemia
|
92.3%
12/13 • Number of events 23
|
|
Infections and infestations
Urinary tract infection
|
7.7%
1/13 • Number of events 2
|
|
General disorders
Fatigue
|
100.0%
13/13 • Number of events 23
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
7.7%
1/13 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
38.5%
5/13 • Number of events 9
|
|
Cardiac disorders
Edema
|
23.1%
3/13 • Number of events 5
|
|
Gastrointestinal disorders
Nausea
|
38.5%
5/13 • Number of events 9
|
|
Gastrointestinal disorders
Vomiting
|
15.4%
2/13 • Number of events 2
|
|
Gastrointestinal disorders
Anorexia
|
53.8%
7/13 • Number of events 12
|
|
Gastrointestinal disorders
Diarrhea
|
23.1%
3/13 • Number of events 7
|
|
Gastrointestinal disorders
Constipation
|
53.8%
7/13 • Number of events 10
|
|
Gastrointestinal disorders
GI Pain
|
38.5%
5/13 • Number of events 9
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
7.7%
1/13 • Number of events 3
|
|
Investigations
Creatinine
|
30.8%
4/13 • Number of events 5
|
|
Investigations
Alk Phos
|
46.2%
6/13 • Number of events 10
|
|
Investigations
SGOT
|
30.8%
4/13 • Number of events 7
|
|
Investigations
Bilirubin
|
15.4%
2/13 • Number of events 3
|
|
Investigations
Lipase
|
15.4%
2/13 • Number of events 2
|
|
Investigations
Hypoalbuminemia
|
46.2%
6/13 • Number of events 9
|
|
Investigations
Hyponatremia
|
30.8%
4/13 • Number of events 6
|
|
Investigations
Hyperkalemia
|
38.5%
5/13 • Number of events 8
|
|
Investigations
Hypocalcemia
|
7.7%
1/13 • Number of events 1
|
|
Investigations
Hypomagnesemia
|
30.8%
4/13 • Number of events 4
|
|
General disorders
Hoarseness voice changes
|
30.8%
4/13 • Number of events 5
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
38.5%
5/13 • Number of events 11
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.4%
2/13 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
30.8%
4/13 • Number of events 6
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
30.8%
4/13 • Number of events 7
|
Additional Information
Margaret von Mehren, M.D., Director of Sarcoma Oncology
Fox Chase Cancer Center
Phone: 215-728-2814
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place