Trial Outcomes & Findings for Phase III Study of BKM120/Placebo With Fulvestrant in Postmenopausal Patients With Hormone Receptor Positive HER2-negative Locally Advanced or Metastatic Breast Cancer Refractory to Aromatase Inhibitor (NCT NCT01610284)

Last Updated: 2020-08-25

Results Overview

Progression Free Survival (PFS) is defined as the time from date of randomization to the date of first radiologically documented progression or death due to any cause. If a patient did not progress or die at the time of the analysis data cut-off or start of new antineoplastic therapy, PFS was censored at the date of the last adequate tumor assessment before the earliest of the cut-off date or the start date of additional anti-neoplastic therapy. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria RECIST v1.1, as 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline and/or unequivocal progression of the non-target lesions and/or appearance of a new lesion. In addition to the relative increase of 20%, the sum must demonstrate an absolute increase of at least 5 mm.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

1147 participants

Primary outcome timeframe

Date of randomization to the date of first documented tumor progression or death from any cause, whichever occurs first, reported between day of first patient randomized up to approximately 4 years

Results posted on

2020-08-25

Participant Flow

This study was conducted at 274 centers in 29 countries worldwide (Argentina, Australia, Austria, Belgium, Brazil, Canada, China, Czech Republic, France, Germany, Greece, Hungary, Israel, Italy, Japan, Republic of Korea, The Netherlands, Peru, Poland, Russia, Singapore, Slovakia, South Africa, Spain, Switzerland, Taiwan, Thailand, UK and USA.).

Approximately 1200 patients were planned to be enrolled in the study. A total of 1147 patients were randomized and analyzed (576 in the buparlisib + fulvestrant and 571 in the placebo + fulvestrant arm). Not completed: in Randomization Phase=Randomized and not Treated; in Treatment Phase=Discontinued study treatment per Protocol.

Participant milestones

Participant milestones
Measure	BKM120 100mg + Fulvestrant BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Randomization Phase STARTED	576	571
Randomization Phase Safety Set (SS)	573	570
Randomization Phase COMPLETED	574	569
Randomization Phase NOT COMPLETED	2	2
Treatment Phase STARTED	574	569
Treatment Phase BKM120 Pharmacokinetic Analysis Set	93	0
Treatment Phase Full Pharmacokinetic Analysis Set (FPAS)	35	0
Treatment Phase COMPLETED	0	0
Treatment Phase NOT COMPLETED	574	569
Post-Treatment Efficacy Follow-Up Phase STARTED	89	29
Post-Treatment Efficacy Follow-Up Phase COMPLETED	0	0
Post-Treatment Efficacy Follow-Up Phase NOT COMPLETED	89	29

Reasons for withdrawal

Reasons for withdrawal
Measure	BKM120 100mg + Fulvestrant BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Randomization Phase Adverse Event	1	0
Randomization Phase Physician Decision	1	1
Randomization Phase Death	0	1
Treatment Phase Adverse Event	80	12
Treatment Phase Lost to Follow-up	1	0
Treatment Phase Non-compliance with Study Treatment	8	1
Treatment Phase Physician Decision	27	24
Treatment Phase Progressive Disease	377	486
Treatment Phase Protocol Deviation	2	3
Treatment Phase Study terminated by Sponsor	18	15
Treatment Phase Subject/Guardian Decision	55	23
Treatment Phase Death	6	5
Post-Treatment Efficacy Follow-Up Phase Adverse Event	2	0
Post-Treatment Efficacy Follow-Up Phase Physician Decision	10	3
Post-Treatment Efficacy Follow-Up Phase Progressive Disease	24	8
Post-Treatment Efficacy Follow-Up Phase Subject/Guardian Decision	11	1
Post-Treatment Efficacy Follow-Up Phase Death	4	4
Post-Treatment Efficacy Follow-Up Phase New therapy for study indication	38	13

Baseline Characteristics

Phase III Study of BKM120/Placebo With Fulvestrant in Postmenopausal Patients With Hormone Receptor Positive HER2-negative Locally Advanced or Metastatic Breast Cancer Refractory to Aromatase Inhibitor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.	Total n=1147 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
Age, Categorical Between 18 and 65 years	329 Participants n=5 Participants	378 Participants n=7 Participants	707 Participants n=5 Participants
Age, Categorical >=65 years	247 Participants n=5 Participants	193 Participants n=7 Participants	440 Participants n=5 Participants
Age, Continuous	62.2 Years STANDARD_DEVIATION 10.20 • n=5 Participants	60.6 Years STANDARD_DEVIATION 10.08 • n=7 Participants	61.4 Years STANDARD_DEVIATION 10.17 • n=5 Participants
Sex: Female, Male Female	576 Participants n=5 Participants	571 Participants n=7 Participants	1147 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Asian	132 Participants n=5 Participants	153 Participants n=7 Participants	285 Participants n=5 Participants
Race/Ethnicity, Customized Black	5 Participants n=5 Participants	16 Participants n=7 Participants	21 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	402 Participants n=5 Participants	376 Participants n=7 Participants	778 Participants n=5 Participants
Race/Ethnicity, Customized Other	18 Participants n=5 Participants	7 Participants n=7 Participants	25 Participants n=5 Participants
Race/Ethnicity, Customized Unknown	19 Participants n=5 Participants	18 Participants n=7 Participants	37 Participants n=5 Participants
Race/Ethnicity, Customized Missing	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
ECOG Performance Status Grade 0 = No Restrictions	333 Participants n=5 Participants	344 Participants n=7 Participants	677 Participants n=5 Participants
ECOG Performance Status Grade 1 = Only Light Work	231 Participants n=5 Participants	211 Participants n=7 Participants	442 Participants n=5 Participants
ECOG Performance Status Grade 2 = Only Self Care	11 Participants n=5 Participants	16 Participants n=7 Participants	27 Participants n=5 Participants
ECOG Performance Status Grade 3 = Only Limited Self-Care	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Date of randomization to the date of first documented tumor progression or death from any cause, whichever occurs first, reported between day of first patient randomized up to approximately 4 years

Population: Full Analysis Set (FAS) in the Full Population, the Main Study Cohort (known PI3K pathway activation status \[activated, non activated\]) and the PI3K unknown cohort (PI3K pathway unknown status) were considered.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Progression Free Survival (PFS) Based on Local Investigator Assessment - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Full population	6.9 Months Interval 6.8 to 7.8	5.0 Months Interval 4.0 to 5.2
Progression Free Survival (PFS) Based on Local Investigator Assessment - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Main cohort	6.8 Months Interval 5.0 to 7.0	4.5 Months Interval 3.3 to 5.0
Progression Free Survival (PFS) Based on Local Investigator Assessment - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway non-activated	6.9 Months Interval 4.6 to 7.2	4.6 Months Interval 3.3 to 5.1
Progression Free Survival (PFS) Based on Local Investigator Assessment - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway activated	6.8 Months Interval 4.9 to 7.1	4.0 Months Interval 3.1 to 5.2
Progression Free Survival (PFS) Based on Local Investigator Assessment - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway unknown	8.7 Months Interval 7.0 to 12.4	6.8 Months Interval 5.0 to 8.4

SECONDARY outcome

Timeframe: Every 3 months following end of treatment visit, assessed for approximately 5 years

Overall Survival (OS) is defined as the time from date of randomization to date of death due to any cause. If a patient was not known to have died by the date of analysis cut-off, OS was censored at the date of last known date patient alive. Patients were followed up for the duration of the study and for an expected average of every 3 months after end of treatment.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Overall Survival (OS) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway unknown	42.3 Months Interval 36.5 to NA: Not estimable due to insufficient number of participants with events	36.0 Months Interval 31.0 to 43.5
Overall Survival (OS) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Full population	33.2 Months Interval 30.0 to 37.3	30.4 Months Interval 27.9 to 32.2
Overall Survival (OS) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Main cohort	30.9 Months Interval 25.1 to 35.4	28.9 Months Interval 25.9 to 31.1
Overall Survival (OS) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway activated	33.6 Months Interval 23.8 to 40.0	27.5 Months Interval 24.4 to 31.3
Overall Survival (OS) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway non-activated	28.8 Months Interval 23.0 to 33.2	30.0 Months Interval 26.0 to 33.4

SECONDARY outcome

Timeframe: From the date of randomization until the date of the first documented disease progression or date of death from any cause whichever came first, assessed for approximately 5 years

Overall Response Rate (ORR) is defined as the percentage of participants with best overall response of complete response (CR) or partial response (PR) based on local investigator's assessment according to RECIST 1.1. ORR was analyzed in the full population. Response Evaluation Criteria in Solid Tumors (RECIST v1.1) for target/non target lesions: Complete Response (CR), disappearance of all target/non target lesions (all lymph nodes assigned as non-target lesions must be non-pathological in size (\< 10 mm short axis)); Partial response (PR), \>=30% decrease in the sum of the longest diameter of target lesions ; Overall Response (OR)= CR+PR. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Overall Response Rate (ORR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Full population	11.8 Percentage of Participants Interval 9.3 to 14.7	7.7 Percentage of Participants Interval 5.7 to 10.2
Overall Response Rate (ORR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Main cohort	11.0 Percentage of Participants Interval 8.2 to 14.4	7.8 Percentage of Participants Interval 5.4 to 10.8
Overall Response Rate (ORR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway activated	10.6 Percentage of Participants Interval 6.6 to 16.0	8.2 Percentage of Participants Interval 4.6 to 13.1
Overall Response Rate (ORR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway non-activated	11.3 Percentage of Participants Interval 7.6 to 16.0	7.5 Percentage of Participants Interval 4.5 to 11.6
Overall Response Rate (ORR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway unknown	14.1 Percentage of Participants Interval 8.9 to 20.7	7.5 Percentage of Participants Interval 3.8 to 13.0

SECONDARY outcome

Timeframe: From the date of randomization until the date of the first documented disease progression or date of death from any cause whichever came first, assessed for approximately 5 years

Clinical Benefit Rate (CBR) is defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) or stable disease (SD) or Non-CR/non-PD lasting more than 24 weeks based on local investigator's assessment according to RECIST 1.1. CBR was analyzed in the full population. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Clinical Benefit Rate (CBR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Full population	43.8 Percentage of Participants Interval 39.7 to 47.9	42.0 Percentage of Participants Interval 37.9 to 46.2
Clinical Benefit Rate (CBR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-Main cohort	41.7 Percentage of Participants Interval 37.0 to 46.5	39.6 Percentage of Participants Interval 34.9 to 44.5
Clinical Benefit Rate (CBR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway unknown	49.7 Percentage of Participants Interval 41.4 to 58.0	49.0 Percentage of Participants Interval 40.7 to 57.3
Clinical Benefit Rate (CBR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway activated	40.4 Percentage of Participants Interval 33.3 to 47.8	40.8 Percentage of Participants Interval 33.6 to 48.2
Clinical Benefit Rate (CBR) - Full Analysis Set (FAS) in Full Population, Main Study Cohort and PI3K Unknown Cohort FAS-PI3K pathway non-activated	42.7 Percentage of Participants Interval 36.3 to 49.2	38.8 Percentage of Participants Interval 32.6 to 45.2

SECONDARY outcome

Timeframe: From first dose of study treatment to 30 days after last dose of study treatment, assessed for approximately 5 years

Population: Safety Set (SS) in Full Population

Analysis of frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=573 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=570 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths On-treatment Adverse Event (AEs)	569 Participants	532 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Disease Progression	2 Participants	2 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Septic Shock	1 Participants	0 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Cerebral Haemorrhage	0 Participants	1 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths On-treatment Serious Adverse Event (SAEs)	144 Participants	101 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths On-treatment Deaths	12 Participants	13 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Study Indication	6 Participants	7 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Unknown reason	2 Participants	0 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Pneumonia	1 Participants	0 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Cerebral Accident	0 Participants	1 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Sudden Death	0 Participants	1 Participants
Number of Participants With On-Treatments Adverse Events, Serious Adverse Events and Deaths Primary cause of Death = Urosepsis	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Cycle2 Day1 (0, 0.5, 1, 1.5, 2, 3, 4, 6, 8 and 24 hours post-dose). Each cycle is 28 days.

Population: Full Pharmacokinetic Analysis Set (FPAS) included the subset of the patients in the buparlisib PAS who: * Received all planned doses of BKM120 100mg + Fulvestrant preceding full PK profile assessment * Did not vomit within 4 hours of buparlisib dosing after administration * Had an evaluable full PK profile available

Plasma samples were collected from the first 200 BKM120-treated patients on Cycle 2 Day 1 (at pre-dose, 0.5h, 1h, 1.5h, 2h, 3h, 4h, 6h, 8h and 24h \[before Cycle 2 Day 2 dose\] post-dose). Each cycle is 28 days. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=35 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 0.5hr	750.767 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 55.4	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 8hr	808.886 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 50.5	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 24hr	712.336 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 52.7	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 0hr (predose)	768.306 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 69.1	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 1hr	988.341 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 49.2	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 1.5hr	1082.086 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 45.0	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 2hr	1099.517 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 36.4	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 3hr	1081.123 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 43.2	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 4hr	935.485 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 43.0	—
Plasma Concentration-time Profiles of BKM120 in Combination With Fulvestrant at Cycle 2 Day 1 C2D1 6hr	795.555 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 45.1	—

SECONDARY outcome

Timeframe: Cycle 2 Day 1, Cycle 2 Day 15, Cycle 3 Day 1. Each cycle is 28 days.

Population: BKM120 Pharmacokinetic Analysis Set or Buparlisib pharmacokinetic analysis set (Buparlisib PAS) included all patients who received at least one dose of study medication buparlisib and had at least one evaluable post-treatment buparlisib concentration measurement.

Pre-dose samples were collected for trough concentrations at Cycle 2 Day 1, Cycle 2 Day 15 and Cycle 3 Day 1. Each cycle is 28 days. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=93 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Predose Trough Concentration-time Profile of BKM120 in Combination With Fulvestrant Over Time - Pharmacokinetic Analysis Set (PAS) Cycle 2 Day 1	733.278 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 75.6	—
Predose Trough Concentration-time Profile of BKM120 in Combination With Fulvestrant Over Time - Pharmacokinetic Analysis Set (PAS) Cycle 2 Day 15	735.172 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 51.2	—
Predose Trough Concentration-time Profile of BKM120 in Combination With Fulvestrant Over Time - Pharmacokinetic Analysis Set (PAS) Cycle 3 Day 1	716.414 nanogram per milliliter (ng/mL) Geometric Coefficient of Variation 84.8	—

SECONDARY outcome

Timeframe: Up to approx 27 months

Population: Full Analysis (FAS)

Time to definitive deterioration of the ECOG PS was defined as the time between the date of randomization and the date of the assessment at which definitive deterioration was seen. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Median Time to Definitive Deterioration of the ECOG Performance Status - Full Analysis Set (FAS)	24.0 Months Interval 17.1 to NA: Not Estimable due to number of events censored	26.4 Months Interval 19.9 to NA: Not Estimable due to number of events censored

SECONDARY outcome

Timeframe: Cycle 1 day 1, cycle 1 day 15, 6 weeks after randomisation and then every 8 weeks until end of treatment

Population: Full Analysis (FAS)

The global health status/QoL scale score of the QLQ-C30 is identified as the primary PRO variable of interest. Physical Functioning (PF), Emotional Functioning (EF) and Social Functioning (SF) scale scores of the QLQ-C30. The time to definitive 10% deterioration is defined as the time from the randomization date to the date of an event, which is defined as a worsening (decrease) in score by at least 10% compared to baseline, with no later increase above this threshold observed during the course of the study or death due to any cause. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high /healthy level of functioning, a high score for the global health status / QoL represents a high QoL. Patients were assessed up to approx. 8.3 months. Only descriptive analysis performed.

Outcome measures

Outcome measures
Measure	BKM120 100mg + Fulvestrant n=576 Participants BKM120 100 mg per day and fulvestrant given until progression or as described in the protocol.	Placebo + Fulvestrant n=571 Participants BKM120 matching placebo daily and fulvestrant given until progression or as described in the protocol.
Health-related Quality of Life (HRQoL):Time to 10% Definitive Deterioration in the Global Health Status/Quality of Life Per EORTC-QLQ-C30	7.10 Months Interval 6.01 to 9.82	11.50 Months Interval 8.8 to 14.32

Adverse Events

BKM120 100 mg + Fulvestrant

Serious events: 146 serious events

Other events: 560 other events

Deaths: 12 deaths

Placebo + Fulvestrant

Serious events: 101 serious events

Other events: 485 other events

Deaths: 13 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER