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A Dose Escalation Study in Adult Patients With Advanced Solid Malignancies

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: October 27, 2009
Last updated: March 23, 2017
Last verified: March 2017
The study will determine the maximum tolerated dose and thus the recommended phase II dose and schedule of the compound and characterize the safety.

Condition Intervention Phase
Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3
Squamous Lung Cancer With FGFR1 Amplification
Bladder Cancer With FGFR3 Mutation or Fusion
Advanced Solid Tumors With FGFR1 Amplication
Advanced Solid Tumors With FGFR2 Amplication
Advanced Solid Tumors With FGFR3 Mutation
Drug: BGJ398
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: A Phase I, Open-label, Multi-center, Dose Escalation Study of Oral BGJ398, a Pan FGF-R Kinase Inhibitor, in Adult Patients With Advanced Solid Malignancies

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Incidence rate and category of dose-limiting toxicities will be tabulated for patients included in the dose escalation portion of the study, to establish the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RPTD) [ Time Frame: 23 months ]
    Incidence rate and category of dose-limiting toxicities will be tabulated for patients included in the dose escalation portion of the study, to establish the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RPTD). This will be calculated using an established statistical model, based on incidence of adverse events and serious adverse events, physical examinations, vital signs, electrocardiograms, and laboratory parameters

Secondary Outcome Measures:
  • To assess preliminary anti-tumor activity of BGJ398 for patients in expansion Arm 4 (previously treated patients with advanced/metastatic UCC with FGFR3 gene alterations) [ Time Frame: 23 months ]
    overall response rate (ORR), as assessed by investigator per RECIST v 1.0; overall survival (OS), duration of response (DOR) and disease control rate (DCR) will be assessed

  • To determine the pharmacokinetic (PK) profiles of oral BGJ398 [ Time Frame: 23 months ]
    Time vs. concentration profiles, PK parameters of BGJ398 and known active metabolite(s).

  • To evaluate the pharmacodynamic effect of the drug. [ Time Frame: 23 months ]
    Pre- vs. post treatment serial changes in FGF23 plasma levels (not done for patients enrolled to expansion Arm 4)

  • Assess preliminary anti-tumor activity for patients not in Arm 4. [ Time Frame: 23 months ]
    Overall tumor response rate (ORR) and PFS assessed by investigator per RECIST

Enrollment: 208
Actual Study Start Date: December 11, 2009
Estimated Study Completion Date: August 31, 2017
Estimated Primary Completion Date: August 31, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BGJ398 Drug: BGJ398


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with histologically/cytologically confirmed advanced solid tumors with FGFR1 or FGFR2 amplification or FGFR3 mutation, for which no further effective standard anticancer treatment exists
  • Adequate bone marrow function
  • Adequate hepatic and renal function
  • Adequate cardiovascular function
  • Contraception.

    • For women: Must be surgically sterile, post-menopausal, or compliant with a medically approved contraceptive regimen during and for 3 months after the treatment period; must have a negative serum or urine pregnancy test and must not be nursing.
    • For men: Must be surgically sterile or compliant with a contraceptive regimen during and for 3 months after the treatment period

Exclusion Criteria:

  • Patients with primary CNS tumor or CNS tumor involvement
  • Patients with history and/or current evidence of endocrine alteration of calcium-phosphate homeostasis
  • History and/or current evidence of ectopic mineralization/ calcification including but not limited to the soft tissue, kidneys, intestine, myocard and lung with the exception of calcified lymphnodes and asymptomatic coronary calcification
  • Current evidence of corneal disorder/ keratopathy incl. but not limited to bullous/ band keratopathy, corneal abrasion, inflammation/ulceration, keratoconjunctivitis etc., confirmed by ophthalmologic examination.
  • History or current evidence of cardiac arrhythmia and/or conduction abnormality
  • Women who are pregnant or nursing.

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
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Please refer to this study by its identifier: NCT01004224

  Show 50 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals Identifier: NCT01004224     History of Changes
Other Study ID Numbers: CBGJ398X2101
2009-010876-73 ( EudraCT Number )
Study First Received: October 27, 2009
Last Updated: March 23, 2017

Keywords provided by Novartis:
advanced solid tumors
lung cancer
bladder cancer
kinase inhibitor
advanced solid malignancies

Additional relevant MeSH terms:
Urinary Bladder Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Urinary Bladder Diseases
Urologic Diseases processed this record on May 25, 2017