BGJ398 for Patients With Tumors With FGFR Genetic Alterations

Solid Tumor Clinical Trial

— CBGJ398XUS04  

Official title:

Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 6 - BGJ398 for Patients With Tumors With FGFR Genetic Alterations

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02160041
• Other study ID #: CBGJ398XUS04
• Status: Terminated
• Phase: Phase 2
• Start date: July 24, 2014
• Est. completion date: April 30, 2018

Study information

• Verified date: May 2019
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 84
• Est. completion date: April 30, 2018
• Est. primary completion date: April 30, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

Patient has a confirmed diagnosis of a select solid tumor (except with a primary diagnosis of Urothelial cell carcinoma, Cholangiocarcinoma, Endometrial cancer, and Glioblastoma multiforme) or hematologic malignancies and is in need of treatment because of progression or relapse.

Patient's tumor has been evaluated and pre-identified as having a tumor with a FGFR genetic alteration. The qualifying alteration must be assessed and reported by 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 are anticipated to result in a durable remission.

Patient must have progressive and measurable disease per RECIST 1.1. or other appropriate hematological response criteria.

Patient has an Eastern Cooperative Oncology Group (ECOG) performance status = 1

Exclusion Criteria:

Patient has received prior treatment with BGJ398

Patients with Central Nervous System (CNS) metastasis or leptomeningeal carcinomatosis

Patient has received chemotherapy or other anticancer therapy = 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.

Patients with acute or chronic pancreatitis

Patients with impaired cardiac function or clinically significant cardiac diseases

History and/or current evidence of extensive tissue calcification

Use of medications that increase serum levels of phosphorus and/or calcium

Current evidence of corneal or retinal disorder/keratopathy

History and/or current evidence of renal or endocrine alterations of calcium/phosphate homeostasis

Patients with another primary malignancy within 3 years prior to starting study treatment, with the exception of adequately treated basal cell carcinoma, squamous cell carcinoma or other non-melanomatous skin cancer, or in-situ carcinoma of the uterine cervix

Related Conditions & MeSH terms

• Hematologic Malignancies
• Neoplasms
• Solid Tumor

Intervention

Drug:
• BGJ398
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.

Locations

Country	Name	City	State
United States	New Mexico Cancer Center	Albuquerque	New Mexico
United States	Lehigh Valley Health Network	Allentown	Pennsylvania
United States	Community Clinical Research Center	Anderson	Indiana
United States	Illinois Cancer Specialists	Arlington Heights	Illinois
United States	St. Agnes Hospital St. Agnes Hospital (2)	Baltimore	Maryland
United States	Dartmouth Hitchcock Medical Center Dartmouth Hitchcock - Lebanon	Bedford	New Hampshire
United States	Bend Memorial Clinic Bend Mem. Clinic	Bend	Oregon
United States	Billings Clinic Billings Clinic (8)	Billings	Montana
United States	Alabama Oncology St. Vincent's Birmingham	Birmingham	Alabama
United States	Waverly Hematology Oncology	Cary	North Carolina
United States	University of N C at Chapel Hill Physician Office Building	Chapel Hill	North Carolina
United States	Chattanooga Oncology and Hematology Assoicates, PC Chattanooga Oncology	Chattanooga	Tennessee
United States	Lurie Children's Hospital of Chicago Developmental Therapeutics	Chicago	Illinois
United States	Oncology Hematology Care Inc Oncology Hematology Care 2	Cincinnati	Ohio
United States	Cleveland Clinic Foundation Taussig Cancer Institute	Cleveland	Ohio
United States	University Hospitals of Cleveland Seidman Cancer Center University Hospitals	Cleveland	Ohio
United States	Duke University Medical Center Seeley G. Mudd Bldg.	Durham	North Carolina
United States	Virginia Cancer Specialists Fairfax Northern Virginia	Fairfax	Virginia
United States	Southcoast Centers for Cancer Care	Fairhaven	Massachusetts
United States	Sanford Hematology Oncology	Fargo	North Dakota
United States	Florida Cancer Specialists Florida Cancer Specialists 36	Fort Myers	Florida
United States	The Center for Cancer and Blood Disorders	Fort Worth	Texas
United States	Cancer and Hematology Centers of West Michigan Dept. of Oncology	Grand Rapids	Michigan
United States	Rocky Mountain Cancer Centers Rocky Mountain Cancer Ctr (50)	Greenwood Village	Colorado
United States	Houston Methodist Cancer Center	Houston	Texas
United States	MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3)	Houston	Texas
United States	Oncology Consultants Oncology Group	Houston	Texas
United States	Indiana University Indiana Univ. - Purdue Univ.	Indianapolis	Indiana
United States	Research Medical Center Research Med Center (2)	Kansas City	Missouri
United States	University of Louisville / James Graham Brown Cancer Center SC	Louisville	Kentucky
United States	NorthWest Georgia Oncology Centers NW Georgia Oncology	Marietta	Georgia
United States	Texas Oncology	McAllen	Texas
United States	University of Miami Sylvester Comprehensive Cancer	Miami	Florida
United States	Minnesota Oncology Hematology, P.A. Minnesota Oncology Hem (27)	Minneapolis	Minnesota
United States	Intermountain Medical Center Intermountain Healthcare	Murray	Utah
United States	Tennessee Oncology Tennessee Oncology (3)	Nashville	Tennessee
United States	Rutgers Cancer Institute of New Jersey	New Brunswick	New Jersey
United States	Norwalk Hospital	Norwalk	Connecticut
United States	North County Oncology Medical Clinic Inc	Oceanside	California
United States	Northern Utah Cancer Associates Northern Utah Assoc (3)	Ogden	Utah
United States	Cancer Treatment Centers of America Eastern Regional Medical Center	Philadelphia	Pennsylvania
United States	University of Pittsburgh Cancer Institute Hillman Cancer Center (2)	Pittsburgh	Pennsylvania
United States	Northwest Cancer Specialists Northwest Cancer	Portland	Oregon
United States	Rhode Island Hospital Rhode Island Hosp. (2)	Providence	Rhode Island
United States	Harbin Clinic Medical Oncology Clin. Res.	Rome	Georgia
United States	University of Utah / Huntsman Cancer Institute SC-2	Salt Lake City	Utah
United States	Utah Cancer Specialists Utah Cancer Specialists (11)	Salt Lake City	Utah
United States	Cancer Therapy & Research Center UT Health Science Center Oncology Dept.	San Antonio	Texas
United States	San Francisco General Hospital San Francisco Gen Hosp (7)	San Francisco	California
United States	Sanford University of South Dakota Medical Center Sanford Health	Sioux Falls	South Dakota
United States	Northern Indiana Cancer Research Consortium No. Indiana Cancer Res.	South Bend	Indiana
United States	Northwest Medical Specialties NW Medical Specialties	Tacoma	Washington
United States	Texas Oncology Cancer Care & Research Center Texas Oncology	Waco	Texas
United States	Deke Slayton Cancer Center Deke Slayton Cancer Center (2)	Webster	Texas
United States	Shenandoah Oncology Shenandoah Oncology (5)	Winchester	Virginia
United States	Wake Forest Baptist Health Hem & Onc Medical Center	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	16 weeks
Secondary	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	baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months
Secondary	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	every 8 weeks until death, assessed up to 24 months
Secondary	Kaplan-Meier Estimates of PFS Rate, % (95% CI)		Months 1, 2, 3, 4, 5, 6, 12, 18, 24
Secondary	Overall Survival (OS)	Overall survival (OS) is defined as the time from the date of first dose to the date of death due to any cause	every 8 weeks until death, assessed up to 36 months
Secondary	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.	months 3, 6, 9, 12, 24
Secondary	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	baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months