Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Leukemia

Hematologic Neoplasms

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

BKM120 100 mg (oral gelatine capsules) was administered orally once daily starting from cycle 1 day 1 and will be dosed continuously every day for each 28- day cycle

BKM120 100 mg (oral gelatine capsules) was administered orally once daily starting from cycle 1 day 1 and will be dosed continuously every day for each 28-day cycle

BKM120

Novartis Pharmaceuticals

The purpose of this signal seeking study was is to determine whether treatment with BKM120 demonstrates sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study.

BKM120 for Patients With PI3K-activated Tumors

Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 1 - BKM120 for Patients With PI3K-activated Tumors

Clinical benefit rate for patients with solid tumors will be assessed using RECIST 1.1 and will include responses of Complete Response (CR) or Partial Response (PR) or Stable Disease (SD) at \>=16 weeks. For hematologic tumors other appropriate hematological response criteria was applied. Response criteria: CR=Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm., PR=At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters, SD=Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study, PD= At least a 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

Overall Response (OR) of Partial Response (PR) or greater is based on local investigator assessment. For patients with solid tumors, the assessment criteria will be RECIST 1.1 and will include responses of CR and/or PR. For hematologic tumors other appropriate hematological response criteria apply

Progression free survival (PFS) is defined as the time from the date of first dose to the date of first documented disease progression or relapse or death due to any cause

Progression free survival (PFS) is defined as the time from the date of first dose to the date of first documented disease progression or relapse or death due to any cause.

Overall survival (OS) is defined as the time from the date of first dose to the date of death due to any cause. If a patient is not known to have died, survival time will be censored at the date of the last contact

Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field "number of deaths resulting from adverse events" all those deaths resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.

Age, Categorical

Age, Continuous

Sex: Female, Male

Caucasian

Black

Asian

Other

Race/Ethnicity, Customized

Grade 0

Grade 1

Grade 2

ECOG Scoring: 0=Fully active, able to carry on all pre-disease performance without restriction, 1=Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g. light housework, office work, 2=Ambulatory and capable of all self-care but unable to carry out any work activities.

Up and about more than 50% of waking hours, 3=Capable of only limited self-care, confined to bed or chair more than 50% of waking hours, 4=Completely disabled, Cannot carry on any self-care. Totally confined to bed or chair, 5= Dead

ECOG Grade

PIK3CA gene mutation

PTEN gene mutation

PIK3CA amplification

PIK3R1 gene mutation

Protocol pre-defined gene mutation (by local labs)

Prior Lines of Antineoplastic Medications

Secondary objective: Duration of response was not analyzed due to low response rate observed

Study Director

Participant Clinical Benefit Response Rate

Overall Response of Partial Response (PR) or Greater. PR=at Least a 30% Decrease in the Sum of Diameters of Target Lesions, Taking as Reference the Baseline Sum Diameters

participants with an event

participants censored

Progression-Free Survival - Number of Participants With an Event

Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Timing in Months

1 month

2 months

3 months

4 months

5 months

6 months

9 months

12 months

18 months

24 months

Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Rate in Percentages

Spots Global Cancer Trial Database for p13k activated tumors