Trial Outcomes & Findings for A Phase 2 Study of BGJ398 in Patients With Recurrent GBM (NCT NCT01975701)
NCT ID: NCT01975701
Last Updated: 2019-12-04
Results Overview
To assess the anti-tumor activity of BGJ398 for patients with GBM and/or other glioma subtypes that harbor FGFR1-TACC1, FGFR3-TACC3 fusion and/or activating mutation in FGFR1, 2 or 3 based on PFS6 (PFS rate at 6 months as defined by RANO criteria as assessed by the investigator)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
26 participants
Primary outcome timeframe
6 months
Results posted on
2019-12-04
Participant Flow
All 26 patients included were enrolled in the non-Surgical group, and were treated with BGJ398 125 mg once daily in 28-day cycles, on a 3 weeks on/1 week off schedule (dosing days 1 to 21 of every 28-day cycle). No patients were enrolled in the surgical group.
Patients were planned to be enrolled in two groups: Group 1 (non-surgical group) targeted patients with response assessment in neuro-oncology (RANO) defined tumor progression not eligible for surgical resection. Group 2 (surgical group) targeted patients with recurrent disease eligible for cytoreductive surgery.
Participant milestones
| Measure |
BGJ398X
125 mg BGJ398 non surgical
|
|---|---|
|
Overall Study
STARTED
|
26
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
25
|
Reasons for withdrawal
| Measure |
BGJ398X
125 mg BGJ398 non surgical
|
|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Death
|
23
|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
A Phase 2 Study of BGJ398 in Patients With Recurrent GBM
Baseline characteristics by cohort
| Measure |
BGJ398X
n=26 Participants
125 mg BGJ398 non surgical
|
|---|---|
|
Age, Continuous
|
53.7 years
STANDARD_DEVIATION 13.59 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
caucasian
|
26 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: full analysis set, patients censored
To assess the anti-tumor activity of BGJ398 for patients with GBM and/or other glioma subtypes that harbor FGFR1-TACC1, FGFR3-TACC3 fusion and/or activating mutation in FGFR1, 2 or 3 based on PFS6 (PFS rate at 6 months as defined by RANO criteria as assessed by the investigator)
Outcome measures
| Measure |
BGJ398X
n=24 Participants
125 mg BGJ398 non surgical
|
|---|---|
|
Progression Free Survival
|
1.7 months
Interval 1.05 to 2.8
|
SECONDARY outcome
Timeframe: 5 yearsPopulation: full analysis set
To further assess the anti-tumor activity of BGJ398 for patients with GBM with an amplification, translocation, or activating mutation in FGFR1,2,3 or 4, based on Objective Response Rate (ORR - patients with measurable disease - as defined by RANO criteria as assessed by the investigator
Outcome measures
| Measure |
BGJ398X
n=26 Participants
125 mg BGJ398 non surgical
|
|---|---|
|
Overall Response Rate
partial response
|
2 Participants
|
|
Overall Response Rate
stable disease
|
7 Participants
|
|
Overall Response Rate
progressive disease
|
13 Participants
|
|
Overall Response Rate
unknown
|
3 Participants
|
|
Overall Response Rate
missing
|
1 Participants
|
SECONDARY outcome
Timeframe: 5 yearsPopulation: full analysis set
To further assess the anti-tumor activity of BGJ398 for patients with GBM and/or other glioma subtypes that harbor FGFR1-TACC1, FGFR3-TACC3 fusion and/or activating mutation in FGFR1, 2 and 3 based on Overall Survival
Outcome measures
| Measure |
BGJ398X
n=26 Participants
125 mg BGJ398 non surgical
|
|---|---|
|
Overall Survival
|
6.74 months
Interval 4.17 to 11.73
|
SECONDARY outcome
Timeframe: 5 yearsPopulation: Safety analysis set
Safety: type, frequency, and severity of AEs and SAEs; Tolerability: dose interruptions, reductions and dose intensity, and evaluations of laboratory values
Outcome measures
| Measure |
BGJ398X
n=26 Participants
125 mg BGJ398 non surgical
|
|---|---|
|
Safety and Tolerability
participants with dose interruptions
|
13 Participants
|
|
Safety and Tolerability
participants with dose reductions
|
4 Participants
|
Adverse Events
Non Surg BGJ398 125 mg
Serious events: 9 serious events
Other events: 26 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Non Surg BGJ398 125 mg
n=26 participants at risk
Non Surg BGJ398 125 mg
|
|---|---|
|
Eye disorders
Cataract
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Gastrointestinal disorders
Vomiting
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
General disorders
General physical health deterioration
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Dehydration
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Ataxia
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Epilepsy
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Hemiparesis
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Hydrocephalus
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Neurological decompensation
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Neurological symptom
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Seizure
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Surgical and medical procedures
Cataract operation
|
3.8%
1/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
Other adverse events
| Measure |
Non Surg BGJ398 125 mg
n=26 participants at risk
Non Surg BGJ398 125 mg
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Eye disorders
Dry eye
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Eye disorders
Eyelid ptosis
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Gastrointestinal disorders
Constipation
|
26.9%
7/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Gastrointestinal disorders
Diarrhoea
|
30.8%
8/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Gastrointestinal disorders
Dyspepsia
|
26.9%
7/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Gastrointestinal disorders
Stomatitis
|
19.2%
5/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
General disorders
Asthenia
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
General disorders
Fatigue
|
34.6%
9/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
General disorders
Gait disturbance
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
General disorders
Mucosal inflammation
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
General disorders
Oedema peripheral
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Infections and infestations
Conjunctivitis
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Infections and infestations
Oral candidiasis
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Injury, poisoning and procedural complications
Fall
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Investigations
Alanine aminotransferase increased
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Investigations
Aspartate aminotransferase increased
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Investigations
Blood alkaline phosphatase increased
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Hyperlipasaemia
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
76.9%
20/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Aphasia
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Dizziness
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Headache
|
23.1%
6/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Hemiparesis
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Memory impairment
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Seizure
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Somnolence
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Nervous system disorders
Tremor
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Psychiatric disorders
Anxiety
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Psychiatric disorders
Confusional state
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Psychiatric disorders
Depression
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Psychiatric disorders
Insomnia
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Renal and urinary disorders
Urinary incontinence
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
15.4%
4/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Nail disorder
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Onycholysis
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysaesthesia syndrome
|
11.5%
3/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
7.7%
2/26 • All AEs reported in this record are treatment emergent AEs, collected from date of First Patient First Treatment until the completion of the safety follow-up ( 30 days after the Last Patient Last Treatment ) up to approximately 5 years.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER