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BGJ398 for Patients With Tumors With FGFR Genetic Alterations (CBGJ398XUS04)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02160041
Recruitment Status : Terminated
First Posted : June 10, 2014
Results First Posted : June 18, 2019
Last Update Posted : June 18, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Solid Tumor
Hematologic Malignancies
Intervention Drug: BGJ398
Enrollment 84
Recruitment Details  
Pre-assignment Details  
Arm/Group Title BGJ398
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 84
Completed 0
Not Completed 84
Reason Not Completed
Adverse Event             11
Death             2
disease progression             57
Protocol Violation             1
non-compliance with study             1
Physician Decision             3
Withdrawal by Subject             8
study terminated by by sponsor             1
Arm/Group Title BGJ398
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 84
Hide Baseline Analysis Population Description
Full Analysis Set (FAS): The FAS included all enrolled patients who received at least 1 dose of study drug. The FAS was the primary set for efficacy analyses.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 84 participants
60.2  (12.08)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 84 participants
<65 years
54
  64.3%
>=65-<75 years
21
  25.0%
>=75 years
9
  10.7%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 84 participants
Female
47
  56.0%
Male
37
  44.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 84 participants
Caucasian
74
  88.1%
Black
6
   7.1%
Asian
1
   1.2%
Other
3
   3.6%
1.Primary Outcome
Title Clinical Benefit Rate (CBR) Associated With BGJ398 Treatment
Hide Description

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
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): The FAS included all enrolled patients who received at least 1 dose of study drug. The FAS was the primary set for efficacy analyses.
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 80
Measure Type: Count of Participants
Unit of Measure: Participants
Complete response (CR)
0
   0.0%
Partial response (PR)
6
   7.5%
Progressive disease (PD)
54
  67.5%
Stable disease (SD)
7
   8.8%
Non-evaluable (NE)
13
  16.3%
Overall response rate (ORR: CR + PR)
6
   7.5%
Clinical benefit rate (CBR: CR+PR+SD)
12
  15.0%
2.Secondary Outcome
Title Overall Response (OR) or Partial Response (PR) or Greater
Hide Description

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
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 84
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: participants
6
(2.8 to 15.6)
3.Secondary Outcome
Title Progression-Free Survival (PFS)
Hide Description

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
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 82
Median (95% Confidence Interval)
Unit of Measure: months
1.8
(1.8 to 2.0)
4.Secondary Outcome
Title Kaplan-Meier Estimates of PFS Rate, % (95% CI)
Hide Description [Not Specified]
Time Frame Months 1, 2, 3, 4, 5, 6, 12, 18, 24
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 82
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percent of participants
Month 1
88.0
(78.2 to 93.6)
Month 2
38.0
(27.0 to 48.9)
Month 3
32.3
(21.9 to 43.0)
Month 4
20.0
(11.5 to 30.1)
Month 5
15.4
(8.0 to 24.9)
Month 6
12.3
(5.8 to 21.3)
Month 12
10.2
(4.3 to 19.2)
Month 18
6.1
(1.8 to 14.5)
Month 24
0.0
(0.0 to 0.0)
5.Secondary Outcome
Title Overall Survival (OS)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 84
Median (95% Confidence Interval)
Unit of Measure: months
6.2
(4.4 to 9.8)
6.Secondary Outcome
Title Kaplan-Meier Estimates of Survival Rate, % (95% CI)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 84
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percent of participants
Month 3
75.9
(64.9 to 83.9)
Month 6
50.1
(38.6 to 60.6)
Month 9
39.4
(28.5 to 50.1)
Month 12
35.1
(24.6 to 45.8)
Month 24
19.5
(10.5 to 30.5)
7.Secondary Outcome
Title Number of Participants With 99 Day Minimum Duration of Response (DOR)
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title BGJ398
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 84
Measure Type: Count of Participants
Unit of Measure: Participants
6
   7.1%
Time Frame Adverse Events (AEs) were collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit) up to maximum of 26.6 months
Adverse Event Reporting Description All cause mortality (deaths) was collected for as long as participants could be contacted from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) up to a maximum of 26.6 months
 
Arm/Group Title BGJ398
Hide Arm/Group Description 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.
All-Cause Mortality
BGJ398
Affected / at Risk (%)
Total   11/83 (13.25%) 
Show Serious Adverse Events Hide Serious Adverse Events
BGJ398
Affected / at Risk (%)
Total   33/83 (39.76%) 
Blood and lymphatic system disorders   
Anaemia  1  3/83 (3.61%) 
Leukocytosis  1  1/83 (1.20%) 
Cardiac disorders   
Atrial fibrillation  1  1/83 (1.20%) 
Pericardial effusion  1  1/83 (1.20%) 
Gastrointestinal disorders   
Abdominal pain  1  2/83 (2.41%) 
Constipation  1  1/83 (1.20%) 
Diarrhoea  1  2/83 (2.41%) 
Dysphagia  1  2/83 (2.41%) 
Enterovesical fistula  1  1/83 (1.20%) 
Gastritis  1  1/83 (1.20%) 
Gastrointestinal haemorrhage  1  1/83 (1.20%) 
Haematemesis  1  1/83 (1.20%) 
Large intestinal obstruction  1  2/83 (2.41%) 
Mallory-Weiss syndrome  1  1/83 (1.20%) 
Nausea  1  3/83 (3.61%) 
Vomiting  1  2/83 (2.41%) 
General disorders   
Asthenia  1  1/83 (1.20%) 
Chest pain  1  1/83 (1.20%) 
Fatigue  1  1/83 (1.20%) 
Malaise  1  1/83 (1.20%) 
Pyrexia  1  2/83 (2.41%) 
Hepatobiliary disorders   
Bile duct obstruction  1  1/83 (1.20%) 
Infections and infestations   
Pneumonia  1  2/83 (2.41%) 
Sepsis  1  4/83 (4.82%) 
Septic shock  1  2/83 (2.41%) 
Soft tissue infection  1  1/83 (1.20%) 
Urinary tract infection  1  3/83 (3.61%) 
Investigations   
Blood creatinine increased  1  1/83 (1.20%) 
Platelet count decreased  1  1/83 (1.20%) 
Metabolism and nutrition disorders   
Cachexia  1  1/83 (1.20%) 
Dehydration  1  2/83 (2.41%) 
Failure to thrive  1  1/83 (1.20%) 
Hypoglycaemia  1  2/83 (2.41%) 
Hyponatraemia  1  1/83 (1.20%) 
Musculoskeletal and connective tissue disorders   
Back pain  1  2/83 (2.41%) 
Bone pain  1  1/83 (1.20%) 
Pain in extremity  1  1/83 (1.20%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Cancer pain  1  1/83 (1.20%) 
Thyroid neoplasm  1  1/83 (1.20%) 
Vaginal neoplasm  1  1/83 (1.20%) 
Nervous system disorders   
Coma  1  1/83 (1.20%) 
Spinal cord compression  1  1/83 (1.20%) 
Syncope  1  2/83 (2.41%) 
Renal and urinary disorders   
Acute kidney injury  1  1/83 (1.20%) 
Obstructive uropathy  1  1/83 (1.20%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnoea  1  4/83 (4.82%) 
Pleural effusion  1  1/83 (1.20%) 
Pneumonia aspiration  1  1/83 (1.20%) 
Pulmonary embolism  1  2/83 (2.41%) 
Respiratory disorder  1  1/83 (1.20%) 
Vascular disorders   
Hypotension  1  1/83 (1.20%) 
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BGJ398
Affected / at Risk (%)
Total   81/83 (97.59%) 
Blood and lymphatic system disorders   
Anaemia  1  9/83 (10.84%) 
Eye disorders   
Dry eye  1  16/83 (19.28%) 
Punctate keratitis  1  5/83 (6.02%) 
Gastrointestinal disorders   
Abdominal distension  1  7/83 (8.43%) 
Abdominal pain  1  12/83 (14.46%) 
Constipation  1  26/83 (31.33%) 
Diarrhoea  1  20/83 (24.10%) 
Dry mouth  1  22/83 (26.51%) 
Dyspepsia  1  10/83 (12.05%) 
Dysphagia  1  6/83 (7.23%) 
Glossodynia  1  5/83 (6.02%) 
Nausea  1  26/83 (31.33%) 
Oral pain  1  6/83 (7.23%) 
Stomatitis  1  22/83 (26.51%) 
Vomiting  1  15/83 (18.07%) 
General disorders   
Asthenia  1  5/83 (6.02%) 
Fatigue  1  44/83 (53.01%) 
Mucosal inflammation  1  15/83 (18.07%) 
Oedema peripheral  1  6/83 (7.23%) 
Pyrexia  1  7/83 (8.43%) 
Infections and infestations   
Conjunctivitis  1  8/83 (9.64%) 
Sinusitis  1  6/83 (7.23%) 
Urinary tract infection  1  12/83 (14.46%) 
Investigations   
Alanine aminotransferase increased  1  8/83 (9.64%) 
Aspartate aminotransferase increased  1  10/83 (12.05%) 
Blood alkaline phosphatase increased  1  9/83 (10.84%) 
Blood creatinine increased  1  17/83 (20.48%) 
Blood phosphorus increased  1  14/83 (16.87%) 
Gamma-glutamyltransferase increased  1  5/83 (6.02%) 
Lipase increased  1  5/83 (6.02%) 
Weight decreased  1  15/83 (18.07%) 
Metabolism and nutrition disorders   
Decreased appetite  1  19/83 (22.89%) 
Dehydration  1  12/83 (14.46%) 
Hypercalcaemia  1  6/83 (7.23%) 
Hyperphosphataemia  1  47/83 (56.63%) 
Hypoalbuminaemia  1  5/83 (6.02%) 
Hypokalaemia  1  6/83 (7.23%) 
Hypomagnesaemia  1  12/83 (14.46%) 
Hyponatraemia  1  10/83 (12.05%) 
Hypophosphataemia  1  10/83 (12.05%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  6/83 (7.23%) 
Back pain  1  7/83 (8.43%) 
Muscle spasms  1  7/83 (8.43%) 
Myalgia  1  7/83 (8.43%) 
Pain in extremity  1  10/83 (12.05%) 
Nervous system disorders   
Dizziness  1  6/83 (7.23%) 
Dysgeusia  1  17/83 (20.48%) 
Headache  1  6/83 (7.23%) 
Neuropathy peripheral  1  5/83 (6.02%) 
Psychiatric disorders   
Insomnia  1  6/83 (7.23%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  8/83 (9.64%) 
Dyspnoea  1  9/83 (10.84%) 
Oropharyngeal pain  1  5/83 (6.02%) 
Skin and subcutaneous tissue disorders   
Alopecia  1  15/83 (18.07%) 
Dry skin  1  8/83 (9.64%) 
Nail discolouration  1  5/83 (6.02%) 
Nail disorder  1  8/83 (9.64%) 
Onychomadesis  1  5/83 (6.02%) 
Rash  1  6/83 (7.23%) 
Vascular disorders   
Hypotension  1  6/83 (7.23%) 
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: +1 (800) 778-8300
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02160041     History of Changes
Other Study ID Numbers: CBGJ398XUS04
First Submitted: June 6, 2014
First Posted: June 10, 2014
Results First Submitted: April 23, 2019
Results First Posted: June 18, 2019
Last Update Posted: June 18, 2019