Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

BKM120 Combined With Vemurafenib (PLX4032) in BRAFV600E/K Mutant Advanced Melanoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01512251
Recruitment Status : Completed
First Posted : January 19, 2012
Results First Posted : May 16, 2018
Last Update Posted : August 17, 2020
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
Alain Algazi, University of California, San Francisco

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition BRAF Mutant Metastatic Melanoma
Intervention Drug: BKM120 Combined with Vemurafenib (PLX4032)
Enrollment 8
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Period Title: Dose Level -1
Started 3 5
Completed 3 5
Not Completed 0 0
Period Title: Phase I, Dose 1
Started 3 5
Completed 0 0
Not Completed 3 5
Period Title: Phase I, Dose 2
Started 0 0
Completed 0 0
Not Completed 0 0
Period Title: Phase I, Dose 3
Started 0 0
Completed 0 0
Not Completed 0 0
Period Title: Phase I, Dose 4
Started 0 0
Completed 0 0
Not Completed 0 0
Period Title: Phase II
Started 0 0
Completed 0 0
Not Completed 0 0
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant Total
Hide Arm/Group Description [Not Specified] [Not Specified] Total of all reporting groups
Overall Number of Baseline Participants 3 5 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 3 participants 5 participants 8 participants
44
(26 to 75)
51
(40 to 62)
50
(26 to 75)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 5 participants 8 participants
Female
1
  33.3%
1
  20.0%
2
  25.0%
Male
2
  66.7%
4
  80.0%
6
  75.0%
1.Primary Outcome
Title Phase 1 - Safety & Recommended Phase 2 Dose (RP2D)
Hide Description RP2D determined by maximum tolerated dose (MTD), post-dose-limiting toxicity (DLT) period toxicity, and pharmacokinetic data
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Determination of MTD
Hide Arm/Group Description:

Vemurafenib-Naïve and Vemurafenib-Resistant populations received:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: mg
60
2.Primary Outcome
Title Phase 2 - Progression-free Survival Rate
Hide Description 6 month progression-free survival rate (PFS6) determined by tumor assessments, clinical tests and laboratory tests
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected, study never advanced to Phase II.
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Secondary Outcome 1 Phase 2 - Objective Response Rate
Hide Description Objective response rate determined by tumor assessments, clinical tests and laboratory tests.
Time Frame Day 28 (+/- 3) of even-numbered treatment cycles until progression
Hide Outcome Measure Data
Hide Analysis Population Description
Data not collected
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Secondary Outcome 2 Phase 2 - Safety and Tolerability
Hide Description Determined by clinical and laboratory tests, and adverse events (AE) assessments
Time Frame During study treatment, up to 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Data not collected
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Secondary Outcome 3 Phase 2 - Phosphatase and TENsin (PTEN) Expression
Hide Description PTEN expression associated with better PFS determined by laboratory tests.
Time Frame No time limit
Hide Outcome Measure Data
Hide Analysis Population Description
Data not collected
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Secondary Outcome 4 Phase 2 - Phosphatidylinositol 3-kinase (PI3K)-Pathway Signaling Reduction Levels
Hide Description Greater reduction in PI3K-pathway signaling associated with better PFS determined by laboratory tests and tumor assessments.
Time Frame No time limit
Hide Outcome Measure Data
Hide Analysis Population Description
Data not collected: study was terminated early due to dose limiting toxicities.
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Secondary Outcome 5 Phase 2 - PI3K Pathway Gene Expression Levels
Hide Description Responding tumors lack gene expression signatures of PI3K pathway activation, and progressing tumors demonstrate gene expression signatures of PI3K pathway activation - determined by laboratory tests and tumor assessments.
Time Frame No time limit
Hide Outcome Measure Data
Hide Analysis Population Description
Data not collected
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title Secondary Outcome 6 Phase 2 - MAPK Pathway Gene Expression Levels
Hide Description Responding tumors lack gene expression signatures of MAPK pathway activation, and progressing tumors demonstrate gene expression signatures of MAPK pathway activation - determined by laboratory tests and tumor assessments.
Time Frame No time limit
Hide Outcome Measure Data
Hide Analysis Population Description
Data not collected
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description:

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid

Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Vemurafenib-Naïve Vemurafenib-Resistant
Hide Arm/Group Description

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

Dose Level -1:

BKM120 60 mg daily Vemurafenib 480 mg bid

Phase I, Dose Level 1:

BKM120 60 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 2:

BKM120 80 mg daily Vemurafenib 720 mg bid

Phase I, Dose Level 3:

BKM120 100 mg daily Vemurafenib 720 mg bid Phase I, Dose Level 4 BKM120 100 mg daily Vemurafenib 960 mg bid Phase II 150 mg oral dabrafenib twice a day (bid) until disease progression, death, or unacceptable adverse events.

BKM120 Combined with Vemurafenib (PLX4032): Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)

All-Cause Mortality
Vemurafenib-Naïve Vemurafenib-Resistant
Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/5 (0.00%) 
Hide Serious Adverse Events
Vemurafenib-Naïve Vemurafenib-Resistant
Affected / at Risk (%) Affected / at Risk (%)
Total   2/3 (66.67%)   2/5 (40.00%) 
Blood and lymphatic system disorders     
Febrile neutropenia  1  0/3 (0.00%)  1/5 (20.00%) 
Renal insufficiency  1  1/3 (33.33%)  0/5 (0.00%) 
Hepatobiliary disorders     
Biliary Fistula  1  0/3 (0.00%)  1/5 (20.00%) 
Nervous system disorders     
Seizure  1  1/3 (33.33%)  0/5 (0.00%) 
Vascular disorders     
Hypotension  1  1/3 (33.33%)  0/5 (0.00%) 
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Vemurafenib-Naïve Vemurafenib-Resistant
Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   5/5 (100.00%) 
Blood and lymphatic system disorders     
elevated LDH  1  1/3 (33.33%)  0/5 (0.00%) 
Anemia  1  0/3 (0.00%)  1/5 (20.00%) 
high eosinophil count  1  1/3 (33.33%)  0/5 (0.00%) 
LDH increased  1  1/3 (33.33%)  1/5 (20.00%) 
Neutropenia  1  0/3 (0.00%)  1/5 (20.00%) 
low hematocrit  1  0/3 (0.00%)  1/5 (20.00%) 
Endocrine disorders     
adrenal insufficiency  1  1/3 (33.33%)  0/5 (0.00%) 
Eye disorders     
eye redness  1  1/3 (33.33%)  0/5 (0.00%) 
Gastrointestinal disorders     
abdominal pain  1  1/3 (33.33%)  1/5 (20.00%) 
vomiting  1  1/3 (33.33%)  2/5 (40.00%) 
diarrhea  1  2/3 (66.67%)  0/5 (0.00%) 
nausea  1  1/3 (33.33%)  1/5 (20.00%) 
Mucositis oral  1  1/3 (33.33%)  0/5 (0.00%) 
Constipation  1  0/3 (0.00%)  1/5 (20.00%) 
Oral Pain  1  1/3 (33.33%)  0/5 (0.00%) 
General disorders     
Fever  1  1/3 (33.33%)  0/5 (0.00%) 
Chills  1  1/3 (33.33%)  1/5 (20.00%) 
fatigue  1  2/3 (66.67%)  0/5 (0.00%) 
Hypothermia  1  1/3 (33.33%)  0/5 (0.00%) 
Decreased activity level  1  1/3 (33.33%)  0/5 (0.00%) 
Infections and infestations     
Rash on lower abdomen  1  0/3 (0.00%)  1/5 (20.00%) 
MRSA infection  1  0/3 (0.00%)  1/5 (20.00%) 
Injury, poisoning and procedural complications     
Bruising  1  1/3 (33.33%)  0/5 (0.00%) 
Investigations     
Blood bilirubin increased  1  0/3 (0.00%)  1/5 (20.00%) 
weight loss  1  2/3 (66.67%)  0/5 (0.00%) 
QtC Prolongation  1  1/3 (33.33%)  0/5 (0.00%) 
creatinine increased  1  1/3 (33.33%)  0/5 (0.00%) 
alkaline phosphatase increased  1  1/3 (33.33%)  0/5 (0.00%) 
ALT increased  1  1/3 (33.33%)  0/5 (0.00%) 
AST increased  1  1/3 (33.33%)  0/5 (0.00%) 
Decreased wbc  1  0/3 (0.00%)  1/5 (20.00%) 
Metabolism and nutrition disorders     
Hyperglycemia  1  0/3 (0.00%)  1/5 (20.00%) 
hypokalemia  1  1/3 (33.33%)  0/5 (0.00%) 
hypoglycemia  1  1/3 (33.33%)  0/5 (0.00%) 
hyponatremia  1  2/3 (66.67%)  0/5 (0.00%) 
hypoalbuminemia  1  1/3 (33.33%)  0/5 (0.00%) 
anorexia  1  2/3 (66.67%)  0/5 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  2/3 (66.67%)  2/5 (40.00%) 
myalgia  1  2/3 (66.67%)  0/5 (0.00%) 
Neck pain  1  1/3 (33.33%)  0/5 (0.00%) 
shoulder pain  1  1/3 (33.33%)  0/5 (0.00%) 
Back pain  1  1/3 (33.33%)  0/5 (0.00%) 
Generalized muscle weakness  1  1/3 (33.33%)  1/5 (20.00%) 
Joint pain (pain in extremity)  1  0/3 (0.00%)  1/5 (20.00%) 
Nervous system disorders     
fingertip numbness  1  1/3 (33.33%)  0/5 (0.00%) 
headache  1  1/3 (33.33%)  0/5 (0.00%) 
Restlessness  1  0/3 (0.00%)  1/5 (20.00%) 
Tingling Extremities (Parasthesia)  1  0/3 (0.00%)  1/5 (20.00%) 
somnolence  1  1/3 (33.33%)  0/5 (0.00%) 
tremor  1  1/3 (33.33%)  0/5 (0.00%) 
Psychiatric disorders     
anxiety  1  1/3 (33.33%)  0/5 (0.00%) 
depression  1  1/3 (33.33%)  0/5 (0.00%) 
Insomnia  1  0/3 (0.00%)  1/5 (20.00%) 
Agitation  1  1/3 (33.33%)  0/5 (0.00%) 
Confusion  1  1/3 (33.33%)  0/5 (0.00%) 
Decreased concentration  1  1/3 (33.33%)  0/5 (0.00%) 
Renal and urinary disorders     
proteinuria  1  1/3 (33.33%)  0/5 (0.00%) 
Reproductive system and breast disorders     
Groin/testicular pain  1  0/3 (0.00%)  1/5 (20.00%) 
Respiratory, thoracic and mediastinal disorders     
cough  1  2/3 (66.67%)  0/5 (0.00%) 
Rhinnorhea (Pharyngeal mucositis)  1  0/3 (0.00%)  1/5 (20.00%) 
Skin and subcutaneous tissue disorders     
Rash maculopapular  1  3/3 (100.00%)  1/5 (20.00%) 
photosensitivity  1  1/3 (33.33%)  0/5 (0.00%) 
alopecia  1  1/3 (33.33%)  0/5 (0.00%) 
desquamation of hands & feet  1  1/3 (33.33%)  0/5 (0.00%) 
Dry Skin  1  0/3 (0.00%)  1/5 (20.00%) 
Crust on elbow  1  1/3 (33.33%)  0/5 (0.00%) 
Pruritis  1  1/3 (33.33%)  0/5 (0.00%) 
Erythemia nodosum  1  0/3 (0.00%)  1/5 (20.00%) 
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Study was terminated early due to dose limiting toxicities.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alain Algazi
Organization: University of California, San Francisco
Phone: (415) 353-7552
EMail: alain.algazi@ucsf.edu
Layout table for additonal information
Responsible Party: Alain Algazi, University of California, San Francisco
ClinicalTrials.gov Identifier: NCT01512251    
Other Study ID Numbers: 11952
CBKM120ZUS21T ( Other Identifier: Novartis )
First Submitted: December 20, 2011
First Posted: January 19, 2012
Results First Submitted: February 23, 2018
Results First Posted: May 16, 2018
Last Update Posted: August 17, 2020