Trial · NCT01339052
Phase II Study of BKM120 for Subjects With Recurrent Glioblastoma
NCT01339052, Phase 2, Completed, L. Nicolas Gonzalez Castro, MD, PhD; results first posted 2017-06-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase II Study of BKM120 for Subjects With Recurrent Glioblastoma. Trials, record NCT01339052. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01339052
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase II Study of BKM120 for Subjects With Recurrent Glioblastoma
- NCT number
- NCT01339052
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 65 participants, actual
- Lead sponsor
- L. Nicolas Gonzalez Castro, MD, PhD
- Conditions
- Glioblastoma
- Interventions
- BKM120, Surgery
- First posted
- 2011-04-20
- Last updated
- 2026-07-27
- Results first posted
- 2017-06-14
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1] | Samples were collected at baseline and at surgery (resected surgical tumor specimen) which occurred after up to 12 days of BKM120 treatment. | pAKT response was determined by pathologist-performed semi-quantitative IHC scoring for pAKT using previously established methods for glioblastoma (GBM) patients. Sample staining scored for intensity on a 0-2+ scale (0 none, 1+ weak positive, 2+ strong positive). Change in pAKT IHC score was the difference in score from baseline to surgery. Participants were classified into 3 groups: a reduction of staining score of one degree or more (considered a response), an increase in score and no change in score. |
| Primary | 6-Month Progression-Free Survival (PFS6) [Cohort 2] | Participants were assessed radiologically every other cycle on treatment; Relevant for this outcome is up to month 6 evaluation. | PFS6 is the proportion of participants remaining alive and progression-free at 6-months from cycle 1 day 1 of BKM120 treatment. Progressive disease is defined using RANO (Response Assessment in Neuro-Oncology) criteria (Wen et al JCO 2010), which takes modified Macdonald Criteria and adds assessment of non-enhancing lesions. Per RANO, progressive disease (PD) is defined either as \> 25% increase in sum of the products of perpendicular diameters of enhancing lesions on stable or increasing doses of corticosteroids, or one or more of the of the following: 1) Significant increase in T2/FLAIR non-enhancing lesion on stable or increasing doses of corticosteroids steroids not due to co-morbid events; 2) Any new lesion; 3) Clear clinical deterioration not attributable to other causes apart from the tumor; or 4) Failure to return for evaluation due to death or deteriorating condition. |
| Primary | BKM120 Brain-to-Plasma Ratio at Time of Surgery [Cohort 1] | Samples were collected at surgery (surgical tumor specimen and plasma sample) which occurred after up to 12 days of BKM120 treatment. | Levels of BKM120 were determined by liquid chromatography coupled with tandem mass spectrometry. BKM120 tumor-to-plasma ratio at time of surgery was calculated based on these levels. |
| Primary | BKM120 Tumor Tissue Concentration at Time of Surgery [Cohort 1] | Samples were collected at surgery (resected surgical tumor specimen) which occurred after up to 12 days of BKM120 treatment. | Levels of BKM120 were determined by liquid chromatography coupled with tandem mass spectrometry. |
| Primary | BKM120 Plasma Concentration at Time of Surgery [Cohort 1] | Samples were collected at surgery which occurred after up to 12 days of BKM120 treatment. | Levels of BKM120 were determined by liquid chromatography coupled with tandem mass spectrometry. |
| Secondary | % Ki-67 Reduction Using Immunohistochemistry (IHC) [Cohort 1] | Samples were collected at baseline (archival tumor specimen) and at surgery (surgical tumor specimen) which occurred after up to 12 days of BTK120 treatment. | Tumor cell proliferation and tumor cell death was using immunohistochemistry for Ki-67 based on established methods. Percent reduction was based on Ki-67 levels at baseline and at surgery. |
| Secondary | Radiographic Response [Cohort 2] | Participants were assessed radiologically every other cycle on treatment. Cohort 2 participants were on treatment up to 9.2 months. | Radiographic response was based on RANO (Response Assessment in Neuro-Oncology) criteria. Per RANO, complete response (CR): 1) Complete disappearance of all enhancing measurable and non-measurable disease sustained for at least 4 weeks, 2) No new lesions, 3) All lesions assessed using the same techniques as baseline, 4) No steroid use (or on physiologic replacement doses only), 5) Stable or improved non-enhancing (T2/FLAIR) lesions and 6) Stable or improved clinically; partial response (PR): 1) \>/= 50% decrease compared to baseline in the sum of products of perpendicular diameters of all measurable enhancing lesions (sum LD) sustained for at least 4 weeks and 2) No progression; progressive disease (PD): 1) \> 25% increase sum LD and/or 2) significant increase in T2/FLAIR, 3) any new lesion, 4) clear clinical deterioration; stable disease (SD): none of the above. |
| Secondary | Maximum Observed Plasma Concentrations (Cmax) of BKM120 Day 1 and Day 8 [Cohort 1] | 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. | Plasma concentrations of BKM120 were analyzed by a validated liquid chromatography-tandem mass spectrometry assay developed by Novartis Pharma AG. Standard Pk parameters were determined using non-compartmental methods. NOTE: This outcome measure was previously titled: "Investigate Pharmacokinetics of BKM120 in This Population by Comparing Maximum Plasma Concentrations (Cmax) From Day 1 to Day 8" |
| Secondary | Cmax Accumulation Ratio of BKM120 Day 1 and Day 8 Ratio [Cohort 1] | 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. | Plasma concentrations of BKM120 were analyzed by a validated liquid chromatography-tandem mass spectrometry assay developed by Novartis Pharma AG. Standard Pk parameters were determined using non-compartmental methods. The accumulation ratio is day 8/day 1. |
| Secondary | Area Under the Concentration Curve From Time 0 to Last Concentration (AUC0-5h) of BKM120 Day 1 and Day 8 [Cohort 1] | 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. | Plasma concentrations of BKM120 were analyzed by a validated liquid chromatography-tandem mass spectrometry assay developed by Novartis Pharma AG. Standard Pk parameters were determined using non-compartmental methods. NOTE: This outcome measure was previously titled: "Investigate Pharmacokinetics of BKM120 in This Population by Comparing the Drug Exposure Area Under the Curve (AUC0-5h) From Day 1 to Day 8" |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1] | Samples were collected at baseline and at surgery (resected surgical tumor specimen) which occurred after up to 12 days of BKM120 treatment. | The analysis dataset is comprised of all treated participants. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | 6-Month Progression-Free Survival (PFS6) [Cohort 2] | Participants were assessed radiologically every other cycle on treatment; Relevant for this outcome is up to month 6 evaluation. | The analysis dataset is comprised of all treated participants. | proportion of participants | NUMBER |
| PRIMARY | BKM120 Brain-to-Plasma Ratio at Time of Surgery [Cohort 1] | Samples were collected at surgery (surgical tumor specimen and plasma sample) which occurred after up to 12 days of BKM120 treatment. | The analysis dataset is comprised of participants with a paired plasma and brain tumor sample (evaluable). Participants missing a brain tumor sample (n=1) or plasma sample (n=2) are excluded from the calculation. | ratio tumor-to-plasma | GEOMETRIC_MEAN |
| PRIMARY | BKM120 Tumor Tissue Concentration at Time of Surgery [Cohort 1] | Samples were collected at surgery (resected surgical tumor specimen) which occurred after up to 12 days of BKM120 treatment. | The analysis dataset is comprised of all participants with a resected surgical tumor specimen (evaluable). | ng/gm | GEOMETRIC_MEAN |
| PRIMARY | BKM120 Plasma Concentration at Time of Surgery [Cohort 1] | Samples were collected at surgery which occurred after up to 12 days of BKM120 treatment. | The analysis dataset is comprised of all participants with a plasma sample at surgery (evaluable). | ng/mL | MEAN |
| SECONDARY | % Ki-67 Reduction Using Immunohistochemistry (IHC) [Cohort 1] | Samples were collected at baseline (archival tumor specimen) and at surgery (surgical tumor specimen) which occurred after up to 12 days of BTK120 treatment. | The analysis dataset is comprised of all participants with a resected surgical tumor specimen (evaluable). | % reduction | MEDIAN |
| SECONDARY | Radiographic Response [Cohort 2] | Participants were assessed radiologically every other cycle on treatment. Cohort 2 participants were on treatment up to 9.2 months. | Only those participants who had measurable disease present at baseline and received at least one dose of therapy are considered evaluable for radiographic response | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Observed Plasma Concentrations (Cmax) of BKM120 Day 1 and Day 8 [Cohort 1] | 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. | The analysis dataset is comprised of all treated patients. | ng/mL | MEAN |
| SECONDARY | Cmax Accumulation Ratio of BKM120 Day 1 and Day 8 Ratio [Cohort 1] | 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. | The analysis dataset is comprised of all treated patients. | ratio day 8/day 1 | MEAN |
| SECONDARY | Area Under the Concentration Curve From Time 0 to Last Concentration (AUC0-5h) of BKM120 Day 1 and Day 8 [Cohort 1] | 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. | The analysis dataset is comprised of all treated patients. | ug*h/mL | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1] | Cohort 1: Surgical Subjects | reduced pAKT (S473) IHC Score | 6 | Participants | |
| Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1] | Cohort 1: Surgical Subjects | increased pAKT (S473) IHC Score | 1 | Participants | |
| Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1] | Cohort 1: Surgical Subjects | no change in pAKT (S473) IHC Score | 7 | Participants | |
| Change in pAKT (S473) Immunohistochemistry (IHC) Score From Baseline to Surgery [Cohort 1] | Cohort 1: Surgical Subjects | Unevaluable | 1 | Participants | |
| 6-Month Progression-Free Survival (PFS6) [Cohort 2] | Cohort 2: Non-surgical Subjects | 0.08 | 90% Confidence Interval 0.028 to 0.174 | proportion of participants | |
| BKM120 Brain-to-Plasma Ratio at Time of Surgery [Cohort 1] | Cohort 1: Surgical Subjects | 1.00 | Full Range 0.18 to 8.44 | ratio tumor-to-plasma | |
| BKM120 Tumor Tissue Concentration at Time of Surgery [Cohort 1] | Cohort 1: Surgical Subjects | 590 | Full Range 86 to 6947 | ng/gm | |
| BKM120 Plasma Concentration at Time of Surgery [Cohort 1] | Cohort 1: Surgical Subjects | 585 | Standard Deviation 192 | ng/mL | |
| % Ki-67 Reduction Using Immunohistochemistry (IHC) [Cohort 1] | Cohort I: Surgical Subjects | 11 | Full Range -81 to 100 | % reduction | |
| Radiographic Response [Cohort 2] | Cohort 2: Non-surgical Subjects | Stable Disease | 16 | Participants |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: Surgical Subjects | 15 | |
| Overall Study | STARTED | Cohort 2: Non-surgical Subjects | 50 | |
| Overall Study | Measurable Disease | Cohort 1: Surgical Subjects | 11 | |
| Overall Study | Measurable Disease | Cohort 2: Non-surgical Subjects | 40 | |
| Overall Study | Evaluable Tumor Sample at Surgery | Cohort 1: Surgical Subjects | 14 | |
| Overall Study | Evaluable Tumor Sample at Surgery | Cohort 2: Non-surgical Subjects | 0 | |
| Overall Study | Evaluable Plasma Sample at Surgery | Cohort 1: Surgical Subjects | 13 | |
| Overall Study | Evaluable Plasma Sample at Surgery | Cohort 2: Non-surgical Subjects | 0 | |
| Overall Study | COMPLETED | Cohort 1: Surgical Subjects | 0 | |
| Overall Study | COMPLETED | Cohort 2: Non-surgical Subjects | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events266
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hearing Impaired | Ear and labyrinth disorders | Cohorts 1: Surgical Subjects | 2 | 15 | 2 |
| other | Hearing Impaired | Ear and labyrinth disorders | Cohorts 2: Non-surgical Subjects | 2 | 50 | 2 |
| other | Constipation | Gastrointestinal disorders | Cohorts 1: Surgical Subjects | 2 | 15 | 4 |
| other | Constipation | Gastrointestinal disorders | Cohorts 2: Non-surgical Subjects | 8 | 50 | 8 |
| other | Diarrhea | Gastrointestinal disorders | Cohorts 1: Surgical Subjects | 5 | 15 | 8 |
| other | Diarrhea | Gastrointestinal disorders | Cohorts 2: Non-surgical Subjects | 12 | 50 | 15 |
| other | Mucositis oral | Gastrointestinal disorders | Cohorts 1: Surgical Subjects | 3 | 15 | 3 |
| other | Mucositis oral | Gastrointestinal disorders | Cohorts 2: Non-surgical Subjects | 6 | 50 | 6 |
| other | Nausea | Gastrointestinal disorders | Cohorts 1: Surgical Subjects | 6 | 15 | 6 |
| other | Nausea | Gastrointestinal disorders | Cohorts 2: Non-surgical Subjects | 8 | 50 | 8 |
1 to 10 of 266Source ClinicalTrials.gov
See all 266 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohorts 1: Surgical Subjects | 0 | 15 |
| Total, all-cause mortality | Cohorts 2: Non-surgical Subjects | 2 | 50 |
| Total, other adverse events | Cohorts 1: Surgical Subjects | 15 | 15 |
| Total, other adverse events | Cohorts 2: Non-surgical Subjects | 50 | 50 |
| Total, serious adverse events | Cohorts 1: Surgical Subjects | 5 | 15 |
| Total, serious adverse events | Cohorts 2: Non-surgical Subjects | 12 | 50 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 30715997 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohorts 1: Surgical Subjects vs Cohorts 2: Non-surgical Subjects: deaths from any cause | Trial comparisons |
| Cohorts 1: Surgical Subjects vs Cohorts 2: Non-surgical Subjects: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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