The purpose of this signal seeking study was to determine whether treatment with BGJ398 demonstrates sufficient efficacy in select FGFR pathway-regulated solid tumors and/or hematologic malignancies to warrant further study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
84
BGJ398 was dosed on a flat scale of 125 mg (e.g., 1 x 100 mg and 1 x 25 mg capsules) once daily for the first 21 days of the 28-day cycle (3 weeks on, 1 week off in a cycle). A complete treatment cycle is defined as 28 days.
Clinical Benefit Rate (CBR) Associated With BGJ398 Treatment
Tumor Response: Overall response rate (ORR) and clinical benefit rate (CBR) for solid tumor (non-lymphoma) which excludes 3 TIO and 1 Lymphoma patients (hence 80 patients and not 84) Clinical benefit rate for patients with solid tumors were assessed using RECIST 1.1 and include responses of CR or PR or SD. For hematologic tumors other appropriate hematological response criteria may apply 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
Time frame: 16 weeks
Overall Response (OR) or Partial Response (PR) or Greater
The key secondary endpoint, OR, was determined by Investigator assessment for each tumor assessment and defined as responses of CR and PR per RECIST version 1.1. 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
Time frame: baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months
Progression-Free Survival (PFS)
Kaplan-Meier estimates of PFS timing, 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
Time frame: every 8 weeks until death, assessed up to 24 months
Kaplan-Meier Estimates of PFS Rate, % (95% CI)
Time frame: Months 1, 2, 3, 4, 5, 6, 12, 18, 24
Overall Survival (OS)
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Alabama Oncology St. Vincent's Birmingham
Birmingham, Alabama, United States
North County Oncology Medical Clinic Inc
Oceanside, California, United States
San Francisco General Hospital San Francisco Gen Hosp (7)
San Francisco, California, United States
Rocky Mountain Cancer Centers Rocky Mountain Cancer Ctr (50)
Greenwood Village, Colorado, United States
Norwalk Hospital
Norwalk, Connecticut, United States
Florida Cancer Specialists Florida Cancer Specialists 36
Fort Myers, Florida, United States
University of Miami Sylvester Comprehensive Cancer
Miami, Florida, United States
NorthWest Georgia Oncology Centers NW Georgia Oncology
Marietta, Georgia, United States
Harbin Clinic Medical Oncology Clin. Res.
Rome, Georgia, United States
Illinois Cancer Specialists
Arlington Heights, Illinois, United States
...and 46 more locations
Overall survival (OS) is defined as the time from the date of first dose to the date of death due to any cause
Time frame: every 8 weeks until death, assessed up to 36 months
Kaplan-Meier Estimates of Survival Rate, % (95% CI)
Overall survival (OS) is the time from the date of start of treatment to date of death due to any cause. If a patient was not known to have died, survival was censored at the date of last contact.
Time frame: months 3, 6, 9, 12, 24
Number of Participants With 99 Day Minimum Duration of Response (DOR)
The duration of response (PR or greater) applies only to patients whose best response was PR or greater. It is defined as the Ttime from the first documented response to the date first documented disease progression or relapse or death due to any cause
Time frame: baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months