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Completed

NCT Number: NCT01833169

BKM120 for Patients With PI3K-activated Tumors

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.

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Key information

Conditions

PI3K Pathway Activated Tumors Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type Neoplasms by Site

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Arizona Oncology Associates HOPE Division, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patient had a confirmed diagnosis of a solid tumor or hematological malignancy with the exception of endometrial cancer, glioblastoma, nonsmall cell lung cancer, prostate cancer or breast cancer.
  • Patient's tumor was evaluated and pre-identified to have activation of the PI3K pathway, at a CLIA certified laboratory
  • Patient must have received at least one prior treatment for recurrent metastatic and /or locally advanced disease and for whom no standard therapy options was anticipated to result in a durable remission.
  • Patient must have had progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines
  • Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

Exclusion criteria

Patient had received previous treatment with BKM120 Patient had symptomatic CNS metastases Patient had mood disorder as outlined in Section 5 Patient had received chemotherapy or other anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.

Treatment and study plan

BKM120

Drug

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

Primary outcomes

  1. Participant Clinical Benefit Response Rate

    Time frame: Week 16

    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

Secondary outcomes

  1. 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

    Time frame: baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months

    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

  2. Progression-Free Survival - Number of Participants With an Event

    Time frame: Every 8 Weeks until death, assessed up to 24 months

    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

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

    Time frame: baseline up to 24 months

    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

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

    Time frame: baseline up to 24 months

    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.

  5. Overall Survival - Number of Participants With an Event

    Time frame: Every 8 Weeks until death, assessed up to 24 months

    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

  6. Overall Survival (OS)- Kaplan-Meier Estimates of OS Timing in Months

    Time frame: baseline up to 24 months

    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

  7. Overall Survival (OS)- Kaplan-Meier Estimates of OS Rate in Percentages

    Time frame: baseline up to 30 months

    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

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: SIGNATURE

Important dates

Study start
2013
Primary completion
2016
Study completion
2016
First posted
Apr 16, 2013
Registry last updated
Oct 19, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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