Trial Outcomes & Findings for Buparlisib in Metastatic Transitional Cell Carcinoma of the Urothelium (NCT NCT01551030)
NCT ID: NCT01551030
Last Updated: 2021-06-21
Results Overview
at 2 months for the pan-class I selective PI3K inhibitor Buparlisib in patients with metastatic urothelial cancer that has progressed on prior cytotoxic chemotherapy. Response and progression will be evaluated in this study using the international criteria by the Response Evaluation Criteria in Solid Tumors (RECIST).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
2 months
Results posted on
2021-06-21
Participant Flow
Participant milestones
| Measure |
Buparlisib
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Overall Study
STARTED
|
19
|
|
Overall Study
COMPLETED
|
19
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Buparlisib in Metastatic Transitional Cell Carcinoma of the Urothelium
Baseline characteristics by cohort
| Measure |
Buparlisib
n=19 Participants
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Age, Continuous
|
65 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 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
|
2 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
19 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 monthsat 2 months for the pan-class I selective PI3K inhibitor Buparlisib in patients with metastatic urothelial cancer that has progressed on prior cytotoxic chemotherapy. Response and progression will be evaluated in this study using the international criteria by the Response Evaluation Criteria in Solid Tumors (RECIST).
Outcome measures
| Measure |
Buparlisib
n=19 Participants
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Percentage of Participants With Progression Free Survival (PFS) at 2 Months
|
54 % of participants with PFS
|
PRIMARY outcome
Timeframe: Up to 16 monthsOutcome measures
| Measure |
Buparlisib
n=19 Participants
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Progression Free Survival (PFS) in the Expansion Cohort
|
3.2 months
Interval 1.4 to 15.8
|
SECONDARY outcome
Timeframe: 2 monthsas determined by RECIST v1.1 for Buparlisib in patients with progressive metastatic urothelial cancer who have received prior cytotoxic chemotherapy
Outcome measures
| Measure |
Buparlisib
n=19 Participants
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Response Rate
Partial Response
|
1 Participants
|
|
Response Rate
Stable Disease
|
6 Participants
|
|
Response Rate
Progression of Disease
|
6 Participants
|
|
Response Rate
Not Entered
|
6 Participants
|
SECONDARY outcome
Timeframe: 2 yearsTo establish the safety and toxicity of Buparlisib , the frequency of toxicity will be tabulated according to the NCI Common Toxicity Criteria, version 4.0.
Outcome measures
| Measure |
Buparlisib
n=19 Participants
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Number of Participants Evaluated for Toxicity
|
19 Participants
|
Adverse Events
Buparlisib
Serious events: 9 serious events
Other events: 19 other events
Deaths: 17 deaths
Serious adverse events
| Measure |
Buparlisib
n=19 participants at risk
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
5.3%
1/19 • 2 years
|
|
Blood and lymphatic system disorders
Anemia
|
5.3%
1/19 • 2 years
|
|
Metabolism and nutrition disorders
Anorexia
|
5.3%
1/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.3%
1/19 • 2 years
|
|
General disorders
Death NOS
|
5.3%
1/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.3%
1/19 • 2 years
|
|
General disorders
Fatigue
|
10.5%
2/19 • 2 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
5.3%
1/19 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
5.3%
1/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
5.3%
1/19 • 2 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
5.3%
1/19 • 2 years
|
|
Infections and infestations
Infections and infestations - Other, specify
|
5.3%
1/19 • 2 years
|
|
General disorders
Malaise
|
5.3%
1/19 • 2 years
|
|
General disorders
Non-cardiac chest pain
|
5.3%
1/19 • 2 years
|
|
Investigations
Platelet count decreased
|
5.3%
1/19 • 2 years
|
|
Cardiac disorders
Sinus tachycardia
|
10.5%
2/19 • 2 years
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
5.3%
1/19 • 2 years
|
|
Nervous system disorders
Syncope
|
5.3%
1/19 • 2 years
|
|
Vascular disorders
Thromboembolic event
|
5.3%
1/19 • 2 years
|
|
Investigations
White blood cell decreased
|
5.3%
1/19 • 2 years
|
Other adverse events
| Measure |
Buparlisib
n=19 participants at risk
This is an open-label phase II study of the pan-class I selective phosphoinositide 3-kinase (PI3K) inhibitor Buparlisib in patients with metastatic urothelial carcinoma which has progressed despite treatment with prior cytotoxic chemotherapy.
Buparlisib: Buparlisib will be administered at a dose of 100 mg orally once daily (two 50 mg capsules) continuously. Intra-patient dose reduction may be required depending on the type and severity of the individual toxicity encountered. Re-staging imaging studies will be performed after every two cycles of treatment (one cycle = 4 weeks). Patients may continue on study as long as they are tolerating therapy and are free of disease progression.
|
|---|---|
|
General disorders
Fatigue
|
78.9%
15/19 • 2 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
78.9%
15/19 • 2 years
|
|
Investigations
Weight loss
|
57.9%
11/19 • 2 years
|
|
Investigations
Alanine aminotransferase increased
|
47.4%
9/19 • 2 years
|
|
Investigations
Aspartate aminotransferase increased
|
47.4%
9/19 • 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
36.8%
7/19 • 2 years
|
|
Gastrointestinal disorders
Mucositis oral
|
36.8%
7/19 • 2 years
|
|
Gastrointestinal disorders
Nausea
|
36.8%
7/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
31.6%
6/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
31.6%
6/19 • 2 years
|
|
Blood and lymphatic system disorders
Anemia
|
21.1%
4/19 • 2 years
|
|
Investigations
Cholesterol high
|
21.1%
4/19 • 2 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
21.1%
4/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
21.1%
4/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
21.1%
4/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Photosensitivity
|
21.1%
4/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Skin & subcutaneous tissue disorders Other, spec
|
21.1%
4/19 • 2 years
|
|
Investigations
Activated partial thromboplastin time prolonged
|
15.8%
3/19 • 2 years
|
|
Investigations
Alkaline phosphatase increased
|
15.8%
3/19 • 2 years
|
|
Psychiatric disorders
Anxiety
|
15.8%
3/19 • 2 years
|
|
Gastrointestinal disorders
Constipation
|
15.8%
3/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.8%
3/19 • 2 years
|
|
Investigations
Creatinine increased
|
15.8%
3/19 • 2 years
|
|
Psychiatric disorders
Depression
|
15.8%
3/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
15.8%
3/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypernatremia
|
15.8%
3/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
15.8%
3/19 • 2 years
|
|
Gastrointestinal disorders
Vomiting
|
15.8%
3/19 • 2 years
|
|
Metabolism and nutrition disorders
Anorexia
|
15.8%
3/19 • 2 years
|
|
Investigations
Blood bilirubin increased
|
10.5%
2/19 • 2 years
|
|
Gastrointestinal disorders
Dyspepsia
|
10.5%
2/19 • 2 years
|
|
General disorders
Edema limbs
|
10.5%
2/19 • 2 years
|
|
Gastrointestinal disorders
Flatulence
|
10.5%
2/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
10.5%
2/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
10.5%
2/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
10.5%
2/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
10.5%
2/19 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.5%
2/19 • 2 years
|
|
Investigations
Platelet count decreased
|
10.5%
2/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
10.5%
2/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Resp, thoracic & mediastinal disorder Other, spec
|
10.5%
2/19 • 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
5.3%
1/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
5.3%
1/19 • 2 years
|
|
Gastrointestinal disorders
Bloating
|
5.3%
1/19 • 2 years
|
|
Nervous system disorders
Concentration impairment
|
5.3%
1/19 • 2 years
|
|
Nervous system disorders
Dysgeusia
|
5.3%
1/19 • 2 years
|
|
Endocrine disorders
Endocrine disorders - Other, specify
|
5.3%
1/19 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.3%
1/19 • 2 years
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
5.3%
1/19 • 2 years
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
5.3%
1/19 • 2 years
|
|
Ear and labyrinth disorders
Hearing impaired
|
5.3%
1/19 • 2 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
5.3%
1/19 • 2 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
5.3%
1/19 • 2 years
|
|
Investigations
INR increased
|
5.3%
1/19 • 2 years
|
|
Infections and infestations
Infections and infestations - Other, other
|
5.3%
1/19 • 2 years
|
|
Investigations
Lymphocyte count decreased
|
5.3%
1/19 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
5.3%
1/19 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness upper limb
|
5.3%
1/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Nail ridging
|
5.3%
1/19 • 2 years
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
5.3%
1/19 • 2 years
|
|
Nervous system disorders
Peripheral motor neuropathy
|
5.3%
1/19 • 2 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
5.3%
1/19 • 2 years
|
|
Infections and infestations
Rhinitis infective
|
5.3%
1/19 • 2 years
|
|
Gastrointestinal disorders
Stomach pain
|
5.3%
1/19 • 2 years
|
|
Nervous system disorders
Tremor
|
5.3%
1/19 • 2 years
|
|
Renal and urinary disorders
Urinary frequency
|
5.3%
1/19 • 2 years
|
|
Investigations
White blood cell decreased
|
5.3%
1/19 • 2 years
|
Additional Information
Dean Bajorin, MD
Memorial Sloan Kettering Cancer Center
Phone: 646-888-4700
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place