Trial Outcomes & Findings for BEZ235 in Patients With Advanced Renal Cell Carcinoma (RCC) (NCT NCT01453595)
NCT ID: NCT01453595
Last Updated: 2016-02-29
Results Overview
In patients with advanced clear cell RCC, progressing after prior first-line or second-line mTOR therapy. The determination of antitumor efficacy will be based on objective tumor assessments made according to the RECIST1.1.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
10 participants
Primary outcome timeframe
1 year
Results posted on
2016-02-29
Participant Flow
Participant milestones
| Measure |
Cohort 1: BEZ235 400mg
Cohort 1: BEZ235 400mg by mouth twice daily
|
Cohort -1: BEZ235 200mg
Cohort -1: BEZ235 200mg by mouth twice daily
|
Cohort -1a: BEZ235 300mg
Cohort -1a: BEZ235 300mg by mouth twice daily
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
6
|
2
|
|
Overall Study
COMPLETED
|
0
|
5
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
2
|
Reasons for withdrawal
| Measure |
Cohort 1: BEZ235 400mg
Cohort 1: BEZ235 400mg by mouth twice daily
|
Cohort -1: BEZ235 200mg
Cohort -1: BEZ235 200mg by mouth twice daily
|
Cohort -1a: BEZ235 300mg
Cohort -1a: BEZ235 300mg by mouth twice daily
|
|---|---|---|---|
|
Overall Study
Dose Limiting Toxicity (DLT)
|
2
|
1
|
2
|
Baseline Characteristics
BEZ235 in Patients With Advanced Renal Cell Carcinoma (RCC)
Baseline characteristics by cohort
| Measure |
Cohort 1: BEZ235 400mg
n=2 Participants
Cohort 1: BEZ235 400mg by mouth twice daily
|
Cohort -1: BEZ235 200mg
n=6 Participants
Cohort -1: BEZ235 200mg by mouth twice daily
|
Cohort -1a: BEZ235 300mg
n=2 Participants
Cohort -1a: BEZ235 300mg by mouth twice daily
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
6 participants
n=7 Participants
|
2 participants
n=5 Participants
|
10 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 1 yearPopulation: ORR was only assessed for participants who completed the study, which were only 5 patients on Cohort -1
In patients with advanced clear cell RCC, progressing after prior first-line or second-line mTOR therapy. The determination of antitumor efficacy will be based on objective tumor assessments made according to the RECIST1.1.
Outcome measures
| Measure |
Cohort -1: BEZ235 200mg
n=5 Participants
Cohort -1: BEZ235 200mg by mouth twice daily
|
|---|---|
|
Objective Response Rate (ORR)
Progression of Disease
|
3 participants
|
|
Objective Response Rate (ORR)
Stable Disease
|
2 participants
|
Adverse Events
Cohort 1: BEZ235 400mg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort -1: BEZ235 200mg
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort -1a: BEZ235 300mg
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: BEZ235 400mg
n=2 participants at risk
Cohort 1: BEZ235 400mg by mouth twice daily
|
Cohort -1: BEZ235 200mg
n=6 participants at risk
Cohort -1: BEZ235 200mg by mouth twice daily
|
Cohort -1a: BEZ235 300mg
n=2 participants at risk
Cohort -1a: BEZ235 300mg by mouth twice daily
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/2
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
General disorders
Fatigue
|
0.00%
0/2
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/2
|
0.00%
0/6
|
50.0%
1/2 • Number of events 1
|
Other adverse events
| Measure |
Cohort 1: BEZ235 400mg
n=2 participants at risk
Cohort 1: BEZ235 400mg by mouth twice daily
|
Cohort -1: BEZ235 200mg
n=6 participants at risk
Cohort -1: BEZ235 200mg by mouth twice daily
|
Cohort -1a: BEZ235 300mg
n=2 participants at risk
Cohort -1a: BEZ235 300mg by mouth twice daily
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/2
|
16.7%
1/6 • Number of events 3
|
0.00%
0/2
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/2
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
General disorders
Fatigue
|
100.0%
2/2 • Number of events 2
|
16.7%
1/6 • Number of events 1
|
50.0%
1/2 • Number of events 2
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/2
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
Investigations
INR increased
|
0.00%
0/2
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
0.00%
0/2
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
Gastrointestinal disorders
Mucositis oral
|
50.0%
1/2 • Number of events 1
|
16.7%
1/6 • Number of events 1
|
0.00%
0/2
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • Number of events 1
|
0.00%
0/6
|
50.0%
1/2 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Number of events 1
|
0.00%
0/6
|
0.00%
0/2
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
50.0%
1/2 • Number of events 1
|
0.00%
0/6
|
0.00%
0/2
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/2
|
0.00%
0/6
|
50.0%
1/2 • Number of events 1
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/2
|
0.00%
0/6
|
50.0%
1/2 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place