Trial Outcomes & Findings for A Study of BKM120 (Buparlisib) in Relapsed or Refractory Thymomas (NCT NCT02220855)
NCT ID: NCT02220855
Last Updated: 2022-04-11
Results Overview
Percent of patients with Objective response and the Binomial Exact 95% confidence interval. Objective response is defined as having a best response of Complete Response (defined as disappearance of all target lesions and any pathological lymph nodes must have reduction in short axis to \<10mm) or Partial Response (defined as at least a 30% decrease in the sum of diameters of target lesions from the baseline sum diameters) by RECIST v1.1 criteria.
COMPLETED
PHASE2
14 participants
up to three years
2022-04-11
Participant Flow
This protocol was based on enrolling at least 16 patients. The protocol enrolled 14 patients due to lack of funding.
Participant milestones
| Measure |
BKM120
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
14
|
Reasons for withdrawal
| Measure |
BKM120
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Disease Progression
|
5
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
A Study of BKM120 (Buparlisib) in Relapsed or Refractory Thymomas
Baseline characteristics by cohort
| Measure |
BKM120
n=14 Participants
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
53.5 years
STANDARD_DEVIATION 12.81 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to three yearsPopulation: All patients receiving at least one dose of study drug and having at least one evaluable post-baseline visit
Percent of patients with Objective response and the Binomial Exact 95% confidence interval. Objective response is defined as having a best response of Complete Response (defined as disappearance of all target lesions and any pathological lymph nodes must have reduction in short axis to \<10mm) or Partial Response (defined as at least a 30% decrease in the sum of diameters of target lesions from the baseline sum diameters) by RECIST v1.1 criteria.
Outcome measures
| Measure |
BKM120
n=14 Participants
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Percent of Patients With Objective Response
|
7.1 percentage of participants
Interval 0.2 to 33.9
|
SECONDARY outcome
Timeframe: up to three yearsPopulation: All patients enrolled and received treatment.
Number of unique patients who had a treatment related (possible, probable or definite) adverse events with grade \>= 3.
Outcome measures
| Measure |
BKM120
n=14 Participants
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Treatment Related Adverse Events Grade 3 or Above
|
7 Participants
|
SECONDARY outcome
Timeframe: up to three yearsPopulation: All patients enrolled and received treatment.
Duration of time from the start of treatment to time of documented progression or death. Patients who do not progress or die will be censored on their last evaluation date. Kaplan-Meier methods will be used and the median and 95% confidence intervals will be calculated.
Outcome measures
| Measure |
BKM120
n=14 Participants
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Progression-free Survival Rate
|
11.1 months
Interval 2.9 to 18.8
|
SECONDARY outcome
Timeframe: up to three yearsPopulation: All patients enrolled and received treatment.
Percent of patients achieving disease control and the Binomial Exact 95% confidence interval. Disease control is defined as having a best response of Complete Response (defined as disappearance of all target lesions and any pathological lymph nodes must have reduction in short axis to \<10mm) or Partial Response (defined as at least a 30% decrease in the sum of diameters of target lesions from the baseline sum diameters) or Stable Disease for at least 4 months (defined by neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progression) by RECIST v1.1 criteria.
Outcome measures
| Measure |
BKM120
n=14 Participants
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Percent of Patients Achieving Disease Control
|
50.0 percentage of participants
Interval 23.0 to 77.0
|
SECONDARY outcome
Timeframe: up to three yearsPopulation: All patients enrolled and received treatment.
Duration of time from the start of treatment to time of death due to any causes. Patients who do not die will be censored on their last known alive date. Kaplan-Meier methods will be used and the median and 95% confidence intervals will be calculated.
Outcome measures
| Measure |
BKM120
n=14 Participants
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Overall Survival Rate
|
22.5 months
Interval 10.7 to 31.3
|
Adverse Events
BKM120
Serious adverse events
| Measure |
BKM120
n=14 participants at risk
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
7.1%
1/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
7.1%
1/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
7.1%
1/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
7.1%
1/14 • Up to 3 years
|
Other adverse events
| Measure |
BKM120
n=14 participants at risk
BKM120, 100mg capsule for oral use, taken once daily for two or more months for a maximum of one year. Each cycle is 28 days.
|
|---|---|
|
Ear and labyrinth disorders
Middle ear inflammation
|
7.1%
1/14 • Up to 3 years
|
|
Endocrine disorders
Endocrine disorders - Other
|
14.3%
2/14 • Up to 3 years
|
|
Eye disorders
Cataract
|
7.1%
1/14 • Up to 3 years
|
|
Gastrointestinal disorders
Constipation
|
14.3%
2/14 • Up to 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
42.9%
6/14 • Up to 3 years
|
|
Gastrointestinal disorders
Dyspepsia
|
14.3%
2/14 • Up to 3 years
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
7.1%
1/14 • Up to 3 years
|
|
Gastrointestinal disorders
Mucositis oral
|
21.4%
3/14 • Up to 3 years
|
|
Gastrointestinal disorders
Nausea
|
42.9%
6/14 • Up to 3 years
|
|
Gastrointestinal disorders
Vomiting
|
21.4%
3/14 • Up to 3 years
|
|
General disorders
Edema limbs
|
14.3%
2/14 • Up to 3 years
|
|
General disorders
Fatigue
|
78.6%
11/14 • Up to 3 years
|
|
General disorders
Fever
|
21.4%
3/14 • Up to 3 years
|
|
General disorders
Non-cardiac chest pain
|
35.7%
5/14 • Up to 3 years
|
|
General disorders
Pain
|
14.3%
2/14 • Up to 3 years
|
|
Infections and infestations
Bronchial infection
|
7.1%
1/14 • Up to 3 years
|
|
Infections and infestations
Infections and infestations - Other
|
21.4%
3/14 • Up to 3 years
|
|
Infections and infestations
Lung infection
|
7.1%
1/14 • Up to 3 years
|
|
Infections and infestations
Otitis media
|
7.1%
1/14 • Up to 3 years
|
|
Infections and infestations
Sinusitis
|
7.1%
1/14 • Up to 3 years
|
|
Infections and infestations
Skin infection
|
7.1%
1/14 • Up to 3 years
|
|
Infections and infestations
Upper respiratory infection
|
7.1%
1/14 • Up to 3 years
|
|
Injury, poisoning and procedural complications
Fracture
|
7.1%
1/14 • Up to 3 years
|
|
Investigations
Alanine aminotransferase increased
|
7.1%
1/14 • Up to 3 years
|
|
Investigations
Aspartate aminotransferase increased
|
7.1%
1/14 • Up to 3 years
|
|
Investigations
Platelet count decreased
|
7.1%
1/14 • Up to 3 years
|
|
Investigations
Weight loss
|
14.3%
2/14 • Up to 3 years
|
|
Investigations
White blood cell decreased
|
7.1%
1/14 • Up to 3 years
|
|
Metabolism and nutrition disorders
Anorexia
|
78.6%
11/14 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
21.4%
3/14 • Up to 3 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
14.3%
2/14 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.3%
2/14 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
14.3%
2/14 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other
|
21.4%
3/14 • Up to 3 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.1%
1/14 • Up to 3 years
|
|
Nervous system disorders
Dizziness
|
14.3%
2/14 • Up to 3 years
|
|
Nervous system disorders
Dysgeusia
|
21.4%
3/14 • Up to 3 years
|
|
Nervous system disorders
Headache
|
7.1%
1/14 • Up to 3 years
|
|
Nervous system disorders
Peripheral motor neuropathy
|
7.1%
1/14 • Up to 3 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
21.4%
3/14 • Up to 3 years
|
|
Nervous system disorders
Tremor
|
42.9%
6/14 • Up to 3 years
|
|
Psychiatric disorders
Anxiety
|
57.1%
8/14 • Up to 3 years
|
|
Psychiatric disorders
Confusion
|
7.1%
1/14 • Up to 3 years
|
|
Psychiatric disorders
Depression
|
28.6%
4/14 • Up to 3 years
|
|
Psychiatric disorders
Insomnia
|
28.6%
4/14 • Up to 3 years
|
|
Psychiatric disorders
Psychiatric disorders - Other
|
7.1%
1/14 • Up to 3 years
|
|
Renal and urinary disorders
Urinary tract pain
|
7.1%
1/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
7.1%
1/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
64.3%
9/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
57.1%
8/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
7.1%
1/14 • Up to 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
35.7%
5/14 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
14.3%
2/14 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
71.4%
10/14 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
64.3%
9/14 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
14.3%
2/14 • Up to 3 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
21.4%
3/14 • Up to 3 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place