Trial Outcomes & Findings for Phase II Trial of Palbociclib in Patients With Metastatic Urothelial Cancer After Failure of First-Line Chemotherapy (NCT NCT02334527)
NCT ID: NCT02334527
Last Updated: 2018-11-16
Results Overview
Estimate progression free survival (PFS) at 4 months in patients with metastatic urothelial cancer (UC) who have progressed after first-line chemotherapy. PFS is defined as the percent of patients who are alive and free from progression at 4 months.
TERMINATED
PHASE2
12 participants
4 Months
2018-11-16
Participant Flow
12 patients were enrolled between April 2015 and January 2017.
34 patients were screened, 25 were eligible based on tumor immunohistochemistry (IHC) demonstrating p16 loss and intact retinoblastoma. Of those, 12 patients were enrolled.
Participant milestones
| Measure |
Palbociclib (Single Arm Trial)
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II Trial of Palbociclib in Patients With Metastatic Urothelial Cancer After Failure of First-Line Chemotherapy
Baseline characteristics by cohort
| Measure |
Single Arm (Single Arm Trial)
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
|---|---|
|
Age, Continuous
|
68 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 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
|
0 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
|
10 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
|
12 Participants
n=5 Participants
|
|
Primary Tumor Site
Bladder
|
7 Participants
n=5 Participants
|
|
Primary Tumor Site
Upper Tract
|
4 Participants
n=5 Participants
|
|
Primary Tumor Site
Urethra
|
1 Participants
n=5 Participants
|
|
Number of prior systemic therapy regiments (including immunotherapy)
1
|
7 Participants
n=5 Participants
|
|
Number of prior systemic therapy regiments (including immunotherapy)
2
|
3 Participants
n=5 Participants
|
|
Number of prior systemic therapy regiments (including immunotherapy)
3
|
2 Participants
n=5 Participants
|
|
Setting of prior platinum-based chemotherapy
Perioperative only
|
8 Participants
n=5 Participants
|
|
Setting of prior platinum-based chemotherapy
Metastatic only
|
2 Participants
n=5 Participants
|
|
Setting of prior platinum-based chemotherapy
Both
|
2 Participants
n=5 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
0
|
5 Participants
n=5 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
1
|
6 Participants
n=5 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
2
|
1 Participants
n=5 Participants
|
|
Hemoglobin
<10 g/dL
|
2 Participants
n=5 Participants
|
|
Hemoglobin
>= 10 g/dL
|
10 Participants
n=5 Participants
|
|
Liver Metastases
Yes
|
0 Participants
n=5 Participants
|
|
Liver Metastases
No
|
12 Participants
n=5 Participants
|
|
Time from prior therapy
< 3 months
|
4 Participants
n=5 Participants
|
|
Time from prior therapy
3-6 months
|
5 Participants
n=5 Participants
|
|
Time from prior therapy
6-9 months
|
1 Participants
n=5 Participants
|
|
Time from prior therapy
>=12 months
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 MonthsEstimate progression free survival (PFS) at 4 months in patients with metastatic urothelial cancer (UC) who have progressed after first-line chemotherapy. PFS is defined as the percent of patients who are alive and free from progression at 4 months.
Outcome measures
| Measure |
Single Arm (Single Arm Trial)
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
Grade >=3 Adverse Event
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off
|
|---|---|---|
|
Progression Free Survival
|
2 Participants
|
—
|
SECONDARY outcome
Timeframe: 4 MonthsPFS is defined as the time from D1 of treatment until progression or death as a result of any cause. Progressive Disease (PD), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
Outcome measures
| Measure |
Single Arm (Single Arm Trial)
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
Grade >=3 Adverse Event
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off
|
|---|---|---|
|
Progression Free Survival (PFS)
|
1.9 Months
Interval 1.8 to 3.7
|
—
|
SECONDARY outcome
Timeframe: Patients will be followed for up to 5 years after removal from study therapy or death, whichever occurs first.Overall survival is defined as the time from day 1 of treatment until death as a result of any cause.
Outcome measures
| Measure |
Single Arm (Single Arm Trial)
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
Grade >=3 Adverse Event
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off
|
|---|---|---|
|
Overall Survival (OS)
|
6.3 Months
Interval 2.2 to 12.6
|
—
|
SECONDARY outcome
Timeframe: 4 MonthsEstimate response rate (RR) in patients with metastatic UC who have progressed after first-line chemotherapy. Response rate will be the number of patients with complete response and/or partial response. Response will be based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1. Radiographic response will be measured by RECIST, Response Evaluation Criteria In Solid Tumors Criteria, indicating if subject experienced a Complete Response (CR), disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), no response or less response than Partial or Progressive; or Progressive Disease (PD), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Outcome measures
| Measure |
Single Arm (Single Arm Trial)
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
Grade >=3 Adverse Event
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off
|
|---|---|---|
|
Response Rate (RR) - Total Number of Patients With Complete Response (CR) and/or Partial Response (PR)
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: 30 DaysCharacterize the safety profile of palbociclib in patients with metastatic UC after first-line chemotherapy. The NCI Common Terminology Criteria for Adverse Events is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental Activities of Daily Living (ADL). Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4 Life-threatening consequences; urgent intervention indicated. Grade 5 Death related to AE.
Outcome measures
| Measure |
Single Arm (Single Arm Trial)
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
Grade >=3 Adverse Event
n=12 Participants
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off
|
|---|---|---|
|
Number of Participants With Adverse Events
Any Treatment Related Adverse Event
|
11 Participants
|
9 Participants
|
|
Number of Participants With Adverse Events
Any Adverse Event
|
12 Participants
|
11 Participants
|
Adverse Events
Single Arm (Single Arm Trial)
Serious adverse events
| Measure |
Single Arm (Single Arm Trial)
n=12 participants at risk
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
|---|---|
|
Psychiatric disorders
Confusion
|
8.3%
1/12 • 32 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
8.3%
1/12 • 32 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
8.3%
1/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
8.3%
1/12 • 32 weeks
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
8.3%
1/12 • 32 weeks
|
Other adverse events
| Measure |
Single Arm (Single Arm Trial)
n=12 participants at risk
Palbociclib
Palbociclib: 125 mg capsule will be taken once per day orally and continuously for 3 weeks followed by 1 week off.
|
|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
8.3%
1/12 • 32 weeks
|
|
Investigations
Alanine aminotransferase increased
|
16.7%
2/12 • 32 weeks
|
|
Investigations
Alkaline phosphatase increased
|
16.7%
2/12 • 32 weeks
|
|
Blood and lymphatic system disorders
Anemia
|
83.3%
10/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Anorexia
|
8.3%
1/12 • 32 weeks
|
|
Investigations
Aspartate aminotransferase increased
|
25.0%
3/12 • 32 weeks
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
2/12 • 32 weeks
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
8.3%
1/12 • 32 weeks
|
|
Psychiatric disorders
Confusion
|
8.3%
1/12 • 32 weeks
|
|
Gastrointestinal disorders
Constipation
|
25.0%
3/12 • 32 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.3%
1/12 • 32 weeks
|
|
Investigations
Creatinine increased
|
58.3%
7/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Dehydration
|
16.7%
2/12 • 32 weeks
|
|
Psychiatric disorders
Depression
|
16.7%
2/12 • 32 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
8.3%
1/12 • 32 weeks
|
|
Nervous system disorders
Dizziness
|
8.3%
1/12 • 32 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
16.7%
2/12 • 32 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
16.7%
2/12 • 32 weeks
|
|
General disorders
Fatigue
|
41.7%
5/12 • 32 weeks
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
8.3%
1/12 • 32 weeks
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
8.3%
1/12 • 32 weeks
|
|
Renal and urinary disorders
Hematuria
|
8.3%
1/12 • 32 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
8.3%
1/12 • 32 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
8.3%
1/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
8.3%
1/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
16.7%
2/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
8.3%
1/12 • 32 weeks
|
|
Vascular disorders
Hypertension
|
8.3%
1/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
8.3%
1/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
8.3%
1/12 • 32 weeks
|
|
Metabolism and nutrition disorders
Hyponatremia
|
41.7%
5/12 • 32 weeks
|
|
Investigations
Lymphocyte count decreased
|
75.0%
9/12 • 32 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
2/12 • 32 weeks
|
|
Gastrointestinal disorders
Nausea
|
8.3%
1/12 • 32 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
|
8.3%
1/12 • 32 weeks
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
8.3%
1/12 • 32 weeks
|
|
Investigations
Neutrophil count decreased
|
66.7%
8/12 • 32 weeks
|
|
General disorders
Pain
|
16.7%
2/12 • 32 weeks
|
|
Investigations
Platelet count decreased
|
41.7%
5/12 • 32 weeks
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
8.3%
1/12 • 32 weeks
|
|
Renal and urinary disorders
Renal hemorrhage
|
8.3%
1/12 • 32 weeks
|
|
General disorders
Right Mid-Back Pain
|
8.3%
1/12 • 32 weeks
|
|
Cardiac disorders
Sinus tachycardia
|
8.3%
1/12 • 32 weeks
|
|
Investigations
Weight loss
|
16.7%
2/12 • 32 weeks
|
|
Investigations
White blood cell decreased
|
66.7%
8/12 • 32 weeks
|
Additional Information
Robin V. Johnson
UNC Lineberger Comprehensive Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60