Trial Outcomes & Findings for BKM120 as Second-line Therapy for Advanced Endometrial Cancer (NCT NCT01289041)
NCT ID: NCT01289041
Last Updated: 2019-05-30
Results Overview
BOR was determined based on investigator assessment of overall lesion response using RECIST criteria guidelines. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
70 participants
Primary outcome timeframe
24 months
Results posted on
2019-05-30
Participant Flow
Participant milestones
| Measure |
All Patients
|
|---|---|
|
Overall Study
STARTED
|
70
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
70
|
Reasons for withdrawal
| Measure |
All Patients
|
|---|---|
|
Overall Study
Adverse Event
|
24
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Progressive Disease
|
41
|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Death
|
1
|
Baseline Characteristics
BKM120 as Second-line Therapy for Advanced Endometrial Cancer
Baseline characteristics by cohort
| Measure |
All Patients
n=70 Participants
|
|---|---|
|
Age, Continuous
|
63 years
STANDARD_DEVIATION 9.04 • n=5 Participants
|
|
Sex/Gender, Customized
Female
|
70 participants
n=5 Participants
|
|
Sex/Gender, Customized
Male
|
0 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 monthsPopulation: Full analysis set includes all patients who received at least one dose of study drug.
BOR was determined based on investigator assessment of overall lesion response using RECIST criteria guidelines. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Outcome measures
| Measure |
Activated Pl3K
n=49 Participants
|
Non-Activated Pl3K
n=21 Participants
|
All Patients
n=70 Participants
|
|---|---|---|---|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Overall Response Rate (CR + PR)
|
1 number of participants
|
1 number of participants
|
2 number of participants
|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Patients with Measurable disease at baseline
|
49 number of participants
|
21 number of participants
|
70 number of participants
|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Complete Response (CR)
|
1 number of participants
|
0 number of participants
|
1 number of participants
|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Partial Response (PR)
|
0 number of participants
|
1 number of participants
|
1 number of participants
|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Stable Disease (SD)
|
19 number of participants
|
7 number of participants
|
26 number of participants
|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Progressive disease (PD)
|
20 number of participants
|
9 number of participants
|
29 number of participants
|
|
Best Overall Response Rate (BORR) According to PI3K Activation Pathway Status
Unknown (UNK)
|
9 number of participants
|
4 number of participants
|
13 number of participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Full analysis set includes all patients who received at least one dose of study drug.
PFS is defined as the time from start of treatment to the date of first documented progression or death due to any cause. If a patient has not had an event, PFS will be censored at the date of last adequate tumor assessment. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Outcome measures
| Measure |
Activated Pl3K
n=49 Participants
|
Non-Activated Pl3K
n=21 Participants
|
All Patients
n=70 Participants
|
|---|---|---|---|
|
Progression Free Survival (PFS) According to PI3K Activation Pathway Status
|
1.9 months
Interval 1.8 to 3.2
|
1.9 months
Interval 1.6 to 3.3
|
1.9 months
Interval 1.8 to 2.8
|
SECONDARY outcome
Timeframe: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 10 monthsPopulation: Full analysis set includes all patients who received at least one dose of study drug.
Overall survival (OS) was defined as the time from start of treatment to the date of death due to any cause. If a patient is not known to have died, survival was censored at the last date of contact. From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 10 months
Outcome measures
| Measure |
Activated Pl3K
n=49 Participants
|
Non-Activated Pl3K
n=21 Participants
|
All Patients
n=70 Participants
|
|---|---|---|---|
|
Overall Survival (OS) According to PI3K Activation Pathway Status
|
8.9 months
Interval 6.3 to 16.2
|
14.2 months
Interval 8.6 to 24.0
|
9.9 months
Interval 7.4 to 16.2
|
Adverse Events
All Patients
Serious events: 33 serious events
Other events: 67 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Patients
n=70 participants at risk
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
1.4%
1/70
|
|
Cardiac disorders
Acute myocardial infarction
|
1.4%
1/70
|
|
Cardiac disorders
Cardio-respiratory arrest
|
1.4%
1/70
|
|
Gastrointestinal disorders
Abdominal pain
|
2.9%
2/70
|
|
Gastrointestinal disorders
Ascites
|
1.4%
1/70
|
|
Gastrointestinal disorders
Colonic obstruction
|
1.4%
1/70
|
|
Gastrointestinal disorders
Erosive oesophagitis
|
1.4%
1/70
|
|
Gastrointestinal disorders
Intestinal obstruction
|
1.4%
1/70
|
|
Gastrointestinal disorders
Nausea
|
2.9%
2/70
|
|
Gastrointestinal disorders
Stomatitis
|
2.9%
2/70
|
|
Gastrointestinal disorders
Vomiting
|
4.3%
3/70
|
|
General disorders
Asthenia
|
2.9%
2/70
|
|
General disorders
Face oedema
|
1.4%
1/70
|
|
Immune system disorders
Hypersensitivity
|
4.3%
3/70
|
|
Infections and infestations
Bacteraemia
|
1.4%
1/70
|
|
Infections and infestations
Cellulitis
|
1.4%
1/70
|
|
Infections and infestations
Lung infection
|
1.4%
1/70
|
|
Infections and infestations
Sepsis
|
2.9%
2/70
|
|
Infections and infestations
Urinary tract infection
|
1.4%
1/70
|
|
Infections and infestations
Urosepsis
|
1.4%
1/70
|
|
Infections and infestations
Uterine infection
|
1.4%
1/70
|
|
Infections and infestations
Vulval abscess
|
1.4%
1/70
|
|
Investigations
International normalised ratio increased
|
1.4%
1/70
|
|
Investigations
Transaminases increased
|
1.4%
1/70
|
|
Metabolism and nutrition disorders
Decreased appetite
|
2.9%
2/70
|
|
Metabolism and nutrition disorders
Dehydration
|
5.7%
4/70
|
|
Metabolism and nutrition disorders
Hypercreatininaemia
|
1.4%
1/70
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
5.7%
4/70
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
1.4%
1/70
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
1.4%
1/70
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour pain
|
1.4%
1/70
|
|
Nervous system disorders
Cerebrovascular accident
|
1.4%
1/70
|
|
Nervous system disorders
Convulsion
|
1.4%
1/70
|
|
Nervous system disorders
Encephalopathy
|
1.4%
1/70
|
|
Nervous system disorders
Leukoencephalopathy
|
1.4%
1/70
|
|
Psychiatric disorders
Anxiety
|
1.4%
1/70
|
|
Psychiatric disorders
Confusional state
|
1.4%
1/70
|
|
Psychiatric disorders
Delirium
|
2.9%
2/70
|
|
Psychiatric disorders
Mental status changes
|
2.9%
2/70
|
|
Renal and urinary disorders
Hydronephrosis
|
4.3%
3/70
|
|
Renal and urinary disorders
Renal failure
|
1.4%
1/70
|
|
Renal and urinary disorders
Renal failure acute
|
1.4%
1/70
|
|
Renal and urinary disorders
Urinary incontinence
|
1.4%
1/70
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
1.4%
1/70
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.9%
2/70
|
|
Skin and subcutaneous tissue disorders
Drug reaction with eosinophilia and systemic symptoms
|
1.4%
1/70
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.4%
1/70
|
|
Vascular disorders
Hypotension
|
1.4%
1/70
|
Other adverse events
| Measure |
All Patients
n=70 participants at risk
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
18.6%
13/70
|
|
Gastrointestinal disorders
Abdominal pain
|
21.4%
15/70
|
|
Gastrointestinal disorders
Constipation
|
17.1%
12/70
|
|
Gastrointestinal disorders
Diarrhoea
|
24.3%
17/70
|
|
Gastrointestinal disorders
Dyspepsia
|
12.9%
9/70
|
|
Gastrointestinal disorders
Dysphagia
|
5.7%
4/70
|
|
Gastrointestinal disorders
Nausea
|
44.3%
31/70
|
|
Gastrointestinal disorders
Stomatitis
|
14.3%
10/70
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
14/70
|
|
General disorders
Asthenia
|
8.6%
6/70
|
|
General disorders
Fatigue
|
34.3%
24/70
|
|
General disorders
Oedema peripheral
|
12.9%
9/70
|
|
General disorders
Pain
|
5.7%
4/70
|
|
General disorders
Pyrexia
|
8.6%
6/70
|
|
Infections and infestations
Urinary tract infection
|
14.3%
10/70
|
|
Investigations
Alanine aminotransferase increased
|
24.3%
17/70
|
|
Investigations
Aspartate aminotransferase increased
|
25.7%
18/70
|
|
Investigations
Blood alkaline phosphatase increased
|
5.7%
4/70
|
|
Investigations
Blood creatinine increased
|
8.6%
6/70
|
|
Investigations
Gamma-glutamyltransferase increased
|
15.7%
11/70
|
|
Investigations
Weight decreased
|
20.0%
14/70
|
|
Metabolism and nutrition disorders
Decreased appetite
|
40.0%
28/70
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
52.9%
37/70
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
5.7%
4/70
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
17.1%
12/70
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
7.1%
5/70
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.6%
6/70
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.7%
4/70
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.7%
4/70
|
|
Nervous system disorders
Dizziness
|
15.7%
11/70
|
|
Nervous system disorders
Dysgeusia
|
14.3%
10/70
|
|
Nervous system disorders
Headache
|
11.4%
8/70
|
|
Nervous system disorders
Memory impairment
|
7.1%
5/70
|
|
Nervous system disorders
Tremor
|
10.0%
7/70
|
|
Psychiatric disorders
Anxiety
|
24.3%
17/70
|
|
Psychiatric disorders
Confusional state
|
12.9%
9/70
|
|
Psychiatric disorders
Depression
|
22.9%
16/70
|
|
Psychiatric disorders
Insomnia
|
11.4%
8/70
|
|
Psychiatric disorders
Mood altered
|
8.6%
6/70
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.4%
8/70
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
11.4%
8/70
|
|
Skin and subcutaneous tissue disorders
Erythema
|
8.6%
6/70
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
14.3%
10/70
|
|
Skin and subcutaneous tissue disorders
Rash
|
30.0%
21/70
|
|
Vascular disorders
Hypertension
|
7.1%
5/70
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
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