Trial Outcomes & Findings for Buparlisib (BKM120) In Patients With Recurrent/Refractory Primary Central Nervous System Lymphoma (PCNSL) and Recurrent/Refractory Secondary Central Nervous System Lymphoma (SCNSL) (NCT NCT02301364)

NCT ID: NCT02301364

Last Updated: 2017-10-19

Results Overview

Progression-free survival (PFS) is defined as the time from the date of treatment start to the date of the first documented PD or death due to any cause. PFS will be based on the investigator's assessment of MRI, CSF studies and clinical presentation.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

2 years

Results posted on

2017-10-19

Participant Flow

Protocol Open to Accrual 11/20/2014 Protocol Closed to Accrual 03/22/2/016 Primary Completion Date 10/11/2016 Recruitment Location is the medical clinic

Participant milestones

Participant milestones
Measure
Buparlisib (BKM120)
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Overall Study
STARTED
4
Overall Study
COMPLETED
4
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Buparlisib (BKM120) In Patients With Recurrent/Refractory Primary Central Nervous System Lymphoma (PCNSL) and Recurrent/Refractory Secondary Central Nervous System Lymphoma (SCNSL)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Buparlisib (BKM120)
n=4 Participants
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Age, Continuous
64 years
n=5 Participants
Sex: Female, Male
Female
1 Participants
n=5 Participants
Sex: Female, Male
Male
3 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
4 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 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
0 Participants
n=5 Participants
Race (NIH/OMB)
White
4 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
4 participants
n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Progression-free survival (PFS) is defined as the time from the date of treatment start to the date of the first documented PD or death due to any cause. PFS will be based on the investigator's assessment of MRI, CSF studies and clinical presentation.

Outcome measures

Outcome measures
Measure
Buparlisib (BKM120)
n=4 Participants
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Progression Free Survival
39 days
Interval 30.0 to 78.0

SECONDARY outcome

Timeframe: 2 years

Adverse events be summarized based on the Common Toxicity Criteria version 4.0.

Outcome measures

Outcome measures
Measure
Buparlisib (BKM120)
n=4 Participants
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Number of Participants With Adverse Events
4 Participants

SECONDARY outcome

Timeframe: 2 years

Overall survival time is defined as the time from treatment start to the date of death due to any cause.

Outcome measures

Outcome measures
Measure
Buparlisib (BKM120)
n=4 Participants
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Overall Survival
196 days
Interval 54.0 to 284.0

SECONDARY outcome

Timeframe: 2 years

This study will use the Macdonald criteria. Specific lesions must be evaluated serially, and comparative analysis of changes in the area of contrast enhancement, as well as the non-enhancing component, should be performed. Complete Response: Complete disappearance of all measurable and non-measurable disease. No new lesions. Partial Response: Great than or equal to 50% decrease over the baseline in the sum of products of perpendicular diameters of all measurable lesions. no progression of non-measurable disease. No new lesions. Stable/No Response: Does not qualify for CT, PR, or progression. Progressive Disease: 25% increase in the sum of products of all measureable lesions over smallest sum observes (or baseline if no decrease), OR clear clinical worsening of any non-measurable disease, OR appearance of any new lesion/site, OR clear clinical worsening or failure to return for evaluation due to death or deteriorating condition (unless clearly unrelated to this cancer).

Outcome measures

Outcome measures
Measure
Buparlisib (BKM120)
n=4 Participants
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Overall Response Rate
Partial Response
1 Participants
Overall Response Rate
Progressive Disease
3 Participants

Adverse Events

Buparlisib (BKM120)

Serious events: 4 serious events
Other events: 4 other events
Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure
Buparlisib (BKM120)
n=4 participants at risk
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Psychiatric disorders
Confusion
25.0%
1/4 • Up to 30 days post treatment
General disorders
Death NOS
25.0%
1/4 • Up to 30 days post treatment
Psychiatric disorders
Depression
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Dysphasia
50.0%
2/4 • Up to 30 days post treatment
Nervous system disorders
Encephalopathy
25.0%
1/4 • Up to 30 days post treatment
Psychiatric disorders
Hallucinations
25.0%
1/4 • Up to 30 days post treatment
Infections and infestations
Infections and infestations - Other, specify
25.0%
1/4 • Up to 30 days post treatment
Musculoskeletal and connective tissue disorders
Muscle weakness right-sided
25.0%
1/4 • Up to 30 days post treatment
Skin and subcutaneous tissue disorders
Rash maculo-papular
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Seizure
25.0%
1/4 • Up to 30 days post treatment

Other adverse events

Other adverse events
Measure
Buparlisib (BKM120)
n=4 participants at risk
This is an open-label, phase II trial of the pan-PI3K inhibitor buparlisib (BKM120) for patients with recurrent or refractory primary central nervous lymphoma (PCNSL) and recurrent or refractory secondary central nervous lymphoma (SCNSL).
Blood and lymphatic system disorders
Anemia
100.0%
4/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hyperglycemia
100.0%
4/4 • Up to 30 days post treatment
Investigations
Platelet count decreased
100.0%
4/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hypoalbuminemia
75.0%
3/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hypocalcemia
75.0%
3/4 • Up to 30 days post treatment
Investigations
INR increased
75.0%
3/4 • Up to 30 days post treatment
Investigations
Alanine aminotransferase increased
50.0%
2/4 • Up to 30 days post treatment
Investigations
Blood bilirubin increased
50.0%
2/4 • Up to 30 days post treatment
Nervous system disorders
Cognitive disturbance
50.0%
2/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hypokalemia
50.0%
2/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hyponatremia
50.0%
2/4 • Up to 30 days post treatment
Investigations
Lymphocyte count decreased
50.0%
2/4 • Up to 30 days post treatment
Skin and subcutaneous tissue disorders
Rash maculo-papular
50.0%
2/4 • Up to 30 days post treatment
Investigations
White blood cell decreased
50.0%
2/4 • Up to 30 days post treatment
Investigations
Alkaline phosphatase increased
25.0%
1/4 • Up to 30 days post treatment
Investigations
Aspartate aminotransferase increased
25.0%
1/4 • Up to 30 days post treatment
Investigations
Cholesterol high
25.0%
1/4 • Up to 30 days post treatment
Psychiatric disorders
Confusion
25.0%
1/4 • Up to 30 days post treatment
Psychiatric disorders
Depression
25.0%
1/4 • Up to 30 days post treatment
Skin and subcutaneous tissue disorders
Dry skin
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Dysphasia
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Encephalopathy
25.0%
1/4 • Up to 30 days post treatment
General disorders
Fatigue
25.0%
1/4 • Up to 30 days post treatment
Psychiatric disorders
Hallucinations
25.0%
1/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hypernatremia
25.0%
1/4 • Up to 30 days post treatment
Metabolism and nutrition disorders
Hypoglycemia
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Lethargy
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Memory impairment
25.0%
1/4 • Up to 30 days post treatment
Investigations
Neutrophil count decreased
25.0%
1/4 • Up to 30 days post treatment
Skin and subcutaneous tissue disorders
Pruritus
25.0%
1/4 • Up to 30 days post treatment
Nervous system disorders
Seizure
25.0%
1/4 • Up to 30 days post treatment
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
25.0%
1/4 • Up to 30 days post treatment
Musculoskeletal and connective tissue disorders
Muscle weakness right-sided
25.0%
1/4 • Up to 30 days post treatment

Additional Information

Christian Grommes, MD

Memorial Sloan Kettering Cancer Center

Phone: 212-639-4058

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place