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Trial record 1 of 1 for:    NCT01339052
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Phase II Study of BKM120 for Subjects With Recurrent Glioblastoma

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
Brigham and Women's Hospital
Massachusetts General Hospital
Novartis Pharmaceuticals
M.D. Anderson Cancer Center
Memorial Sloan Kettering Cancer Center
University of California, San Francisco
University of California, Los Angeles
University of Utah
Information provided by (Responsible Party):
Patrick Y. Wen, MD, Dana-Farber/Brigham and Women's Cancer Center
ClinicalTrials.gov Identifier:
NCT01339052
First received: April 18, 2011
Last updated: May 15, 2017
Last verified: May 2017
Results First Received: March 14, 2017  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: No masking;   Primary Purpose: Treatment
Condition: Glioblastoma
Interventions: Drug: BKM120
Procedure: Surgery

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients enrolled from September 2011 through February 2014.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Cohort 1: Surgical Subjects

Subjects scheduled for surgery

BKM120: 100 mg once daily, orally, for 8-12 days prior to surgery

Surgery: Surgery

BKM120: 100 mg once daily, orally, for 28-day cycles

Participants continued treatment until disease progression or unacceptable toxicity.

Cohort 2: Non-surgical Subjects

Subjects not candidates for surgery

BKM120: 100 mg once daily, orally, for 28-day cycles

Participants continued treatment until disease progression or unacceptable toxicity.


Participant Flow:   Overall Study
    Cohort 1: Surgical Subjects   Cohort 2: Non-surgical Subjects
STARTED   15   50 
Measurable Disease [1]   11   40 [2] 
Evaluable Tumor Sample at Surgery   14   0 [3] 
Evaluable Plasma Sample at Surgery   13   0 [3] 
COMPLETED [4]   0   0 
NOT COMPLETED   15   50 
Disease progression                13                46 
Adverse Event                1                1 
Withdrawal by Subject                0                3 
Physician Decision                1                0 
[1] Measurable disease post-surgery for Cohort 1 participants.
[2] Radiographic response evaluated on participants with measurable disease only.
[3] Milestone is not relevant for non-surgical cohort.
[4] Since treatment was not of fixed duration, all participants were considered as 'Not Completed'.



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Cohort 1: Surgical Subjects

Subjects scheduled for surgery

BKM120: 100 mg once daily, orally, for 8-12 days prior to surgery

Surgery: Surgery

BKM120: 100 mg once daily, orally, for 28-day cycles

Participants continued treatment until disease progression or unacceptable toxicity.

Cohort 2: Non-surgical Subjects

Subjects not candidates for surgery

BKM120: 100 mg once daily, orally, for 28-day cycles

Participants continued treatment until disease progression or unacceptable toxicity.

Total Total of all reporting groups

Baseline Measures
   Cohort 1: Surgical Subjects   Cohort 2: Non-surgical Subjects   Total 
Overall Participants Analyzed 
[Units: Participants]
 15   50   65 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      0   0.0%      0   0.0%      0   0.0% 
Between 18 and 65 years      13  86.7%      43  86.0%      56  86.2% 
>=65 years      2  13.3%      7  14.0%      9  13.8% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      4  26.7%      13  26.0%      17  26.2% 
Male      11  73.3%      37  74.0%      48  73.8% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      0   0.0%      2   4.0%      2   3.1% 
Not Hispanic or Latino      11  73.3%      35  70.0%      46  70.8% 
Unknown or Not Reported      4  26.7%      13  26.0%      17  26.2% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      3   6.0%      3   4.6% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      12  80.0%      32  64.0%      44  67.7% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      3  20.0%      15  30.0%      18  27.7% 
Region of Enrollment 
[Units: Participants]
Count of Participants
     
United States   15   50   65 
Karnofsky performance status (KPS) [1] 
[Units: Participants]
Count of Participants
     
70      1   6.7%      3   6.0%      4   6.2% 
80      2  13.3%      15  30.0%      17  26.2% 
90      10  66.7%      23  46.0%      33  50.8% 
100      2  13.3%      9  18.0%      11  16.9% 
[1] 'Karnofsky Performance Status' (KPS) scale: 100 = Normal; no complaints; no evidence of disease; 90 = Able to carry on normal activity; minor signs or symptoms of disease; 80 = Normal activity with effort; some sign or symptoms of disease; 70 = Cares for self; unable to carry on normal activity or do active work.
Current Tumor Diagnosis 
[Units: Participants]
Count of Participants
     
Glioblstoma Multiforme (GBM)   15   47   62 
Gliosarcoma   0   3   3 
Initial Tumor Diagnosis 
[Units: Participants]
Count of Participants
     
Glioblastoma Multiforme (GBM)   13   41   54 
Anaplastic Astrocytoma (AA) merging w Glioblastoma   1   0   1 
Gr4 Right Parietal Glioma   1   0   1 
Astrocytoma   0   1   1 
Anaplastic Astrocytoma (AA)   0   3   3 
Gemistocytic Astrocyoma   0   1   1 
Glioma consistent w/ mixed Oligoastrocytoma   0   1   1 
Gliosarcoma   0   1   1 
High grade astrocytoma/glioblastoma   0   1   1 
Oligodendroglioma   0   1   1 
PTEN Status at Registration via Central Path Review 
[Units: Participants]
Count of Participants
     
PTEN Negative   7   34   41 
Not PTEN Negative   8   12   20 
PTEN loss by FISH   0   1   1 
PTEN Mutant   0   1   1 
Unknown/Pending   0   2   2 
pAKT StatusStatus at Registration via Central Path Review 
[Units: Participants]
Count of Participants
     
pAKT Positive   15   39   54 
pAKT Negative   0   2   2 
Unknown/Pending   0   9   9 
Mutation Status at Registration 
[Units: Participants]
Count of Participants
     
PIK3CA mutation   1   2   3 
Negative   0   1   1 
Unknown/Pending   14   47   61 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1]   [ Time Frame: Samples were collected at baseline and at surgery (resected surgical tumor specimen) which occurred after up to 12 days of BKM120 treatment. ]

2.  Primary:   6-Month Progression-Free Survival (PFS6) [Cohort 2]   [ Time Frame: Participants were assessed radiologically every other cycle on treatment; Relevant for this outcome is up to month 6 evaluation. ]

3.  Primary:   BKM120 Brain-to-Plasma Ratio at Time of Surgery [Cohort 1]   [ Time Frame: Samples were collected at surgery (surgical tumor specimen and plasma sample) which occurred after up to 12 days of BKM120 treatment. ]

4.  Primary:   BKM120 Tumor Tissue Concentration at Time of Surgery [Cohort 1]   [ Time Frame: Samples were collected at surgery (resected surgical tumor specimen) which occurred after up to 12 days of BKM120 treatment. ]

5.  Primary:   BKM120 Plasma Concentration at Time of Surgery [Cohort 1]   [ Time Frame: Samples were collected at surgery which occurred after up to 12 days of BKM120 treatment. ]

6.  Secondary:   % Ki-67 Reduction Using Immunohistochemistry (IHC) [Cohort 1]   [ Time Frame: Samples were collected at baseline (archival tumor specimen) and at surgery (surgical tumor specimen) which occurred after up to 12 days of BTK120 treatment. ]

7.  Secondary:   Radiographic Response [Cohort 2]   [ Time Frame: Participants were assessed radiologically every other cycle on treatment. Cohort 2 participants were on treatment up to 9.2 months. ]

8.  Secondary:   Maximum Observed Plasma Concentrations (Cmax) of BKM120 Day 1 and Day 8 [Cohort 1]   [ Time Frame: On days 1 and 8 (+/- 1 day) prior to surgery, 5 blood samples were collected at the following timepoints: pre-dose, and at 0.5, 1.5, 3, and 5 hours post-dose. ]

9.  Secondary:   Time to Maximum Observed Plasma Concentration (Tmax) of BKM120 Day 1 and Day 8 [Cohort 1]   [ Time Frame: On days 1 and 8 (+/- 1 day) prior to surgery, 5 blood samples were collected at the following timepoints: pre-dose, and at 0.5, 1.5, 3, and 5 hours post-dose. ]

10.  Secondary:   Area Under the Concentration Curve From Time 0 to Last Concentration (AUC0-5h) of BKM120 Day 1 and Day 8 [Cohort 1]   [ Time Frame: On days 1 and 8 (+/- 1 day) prior to surgery, 5 blood samples were collected at the following timepoints: pre-dose, and at 0.5, 1.5, 3, and 5 hours post-dose. ]

11.  Secondary:   Overall Survival (OS) [Cohort 2]   [ Time Frame: Participants were followed long-term for survival via medical record review. Cohort 2 participants were followed for survival up to 52 months in this study cohort. ]

12.  Secondary:   Progression-Free Survival (PFS) [Cohort 2]   [ Time Frame: Participants were assessed radiologically every other cycle on treatment and off-treatment via medical record review until death. Cohort 2 participants were followed for progression-free survival up to 12 months in this study cohort. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Patrick Y. Wen, MD, Director, DFCI Center for Neuro-Oncology
Organization: Dana-Farber Cancer Institute
phone: 617-632-2166
e-mail: pwen@partners.org



Responsible Party: Patrick Y. Wen, MD, Dana-Farber/Brigham and Women's Cancer Center
ClinicalTrials.gov Identifier: NCT01339052     History of Changes
Other Study ID Numbers: 11-033
CBKM120XUS07T ( Other Identifier: Novartis )
Study First Received: April 18, 2011
Results First Received: March 14, 2017
Last Updated: May 15, 2017