Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3Phase 1NCT01004224
A Dose Escalation Study in Adult Patients With Advanced Solid Malignancies
A Phase 1 study testing BGJ398 in people with Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3 and Squamous Lung Cancer With FGFR1 Amplification, run by Novartis Pharmaceuticals at 50 sites in 14 countries.
Trial summary
The study will determine the maximum tolerated dose and thus the recommended phase II dose and schedule of the compound and characterize the safety. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3, Squamous Lung Cancer With FGFR1 Amplification, Bladder Cancer With FGFR3 Mutation or Fusion
- What is being tested?
- BGJ398
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 208 participants
- Where
- 50 sitesin 14 countries
- Study dates
- 11 Dec 2009to 8 Oct 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
50 sites in 14 countries.
50 sites in 14 countries
United States16 sites
University of Colorado Dept. of Anschutz Cancer (3)
Aurora, Colorado, 80045
Novartis Investigative Site
Boston, Massachusetts, 02114
Novartis Investigative Site
Columbus, Ohio, 43221
Novartis Investigative Site
Detroit, Michigan, 48201
Novartis Investigative Site
Duarte, California, 91010 3000
Novartis Investigative Site
Los Angeles, California, 90033
Novartis Investigative Site
Los Angeles, California, 90095
Novartis Investigative Site
Memphis, Tennessee, 38120
Novartis Investigative Site
Nashville, Tennessee, 37203
Novartis Investigative Site
New Haven, Connecticut, 06520
Memorial Sloan Kettering Cancer Center Onc. Dept..
New York, New York, 10021
Novartis Investigative Site
New York, New York, 10029
Novartis Investigative Site
Philadelphia, Pennsylvania, 19104
Thomas Jefferson University Hospital Onc Dept
Philadelphia, Pennsylvania, 19107-5098
Novartis Investigative Site
Pittsburgh, Pennsylvania, 15232
Novartis Investigative Site
Salt Lake City, Utah, 84103
France9 sites
Novartis Investigative Site
Bordeaux, 33075
Novartis Investigative Site
Lyon, 69373
Novartis Investigative Site
Marseille, 13273
Novartis Investigative Site
Montpellier, 34298
Novartis Investigative Site
Paris, 75015
Novartis Investigative Site
Saint-Herblain Cédex, 44805
Novartis Investigative Site
Suresnes, 92150
Novartis Investigative Site
Toulouse, 31059
Novartis Investigative Site
Villejuif, 94805
Spain7 sites
Novartis Investigative Site
Barcelona, Catalonia, 08035
Novartis Investigative Site
Barcelona, 08041
Novartis Investigative Site
Madrid, 28009
Novartis Investigative Site
Madrid, 28041
Novartis Investigative Site
Madrid, 28050
Novartis Investigative Site
Seville, Andalusia, 41013
Novartis Investigative Site
Valencia, Valencia, 46009
Germany4 sites
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Hanover, 30625
Novartis Investigative Site
Marburg, 35039
Israel2 sites
Novartis Investigative Site
Ramat Gan, 5265601
Novartis Investigative Site
Tel Aviv, 6423906
Netherlands2 sites
Novartis Investigative Site
Amsterdam, 1066 CX
Novartis Investigative Site
Amsterdam, 1081 HV
South Korea2 sites
Novartis Investigative Site
Seoul, Korea, 05505
Novartis Investigative Site
Seoul, 03080
Thailand2 sites
Novartis Investigative Site
Bangkok, 10330
Novartis Investigative Site
Chiang Mai, 50200
About this study
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
The study will determine the maximum tolerated dose and thus the recommended phase II dose and schedule of the compound and characterize the safety.
- Conditions
- 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
- Being tested
- BGJ398
- Keywords
- advanced solid tumors, lung cancer, bladder cancer, BGJ398, FGFR, kinase inhibitor, advanced solid malignancies
- Medical subject headings
- Lung Neoplasms, Urinary Bladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BGJ398Drug
| Study group | Role | What they receive |
|---|---|---|
| BGJ398 | Experimental | Not described Receives: Drug: BGJ398 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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
Measured: 23 months
Secondary outcomes (4)
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)
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
Measured: 23 months
To determine the pharmacokinetic (PK) profiles of oral BGJ398
Time vs. concentration profiles, PK parameters of BGJ398 and known active metabolite(s).
Measured: 23 months
To evaluate the pharmacodynamic effect of the drug.
Pre- vs. post treatment serial changes in FGF23 plasma levels (not done for patients enrolled to expansion Arm 4)
Measured: 23 months
Assess preliminary anti-tumor activity for patients not in Arm 4.
Overall tumor response rate (ORR) and PFS assessed by investigator per RECIST
Measured: 23 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2009 (estimated)
- Study start11 Dec 2009
- Primary completion8 Oct 2018
- Study completion8 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 208 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01004224
- Sponsor's protocol ID
- CBGJ398X2101
- Other IDs
- 2009-010876-73, 2009
- Status last verified
- Oct 2019
- First submitted
- 27 Oct 2009
- Last updated
- 4 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01004224
- Acronym / sponsor ID
- CBGJ398X2101
- Conditions
- 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
- Keywords
- advanced solid tumors, lung cancer, bladder cancer, BGJ398, FGFR, kinase inhibitor, advanced solid malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 208 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 11 Dec 2009 (actual)
- Primary completion
- 8 Oct 2018 (actual)
- Study completion
- 8 Oct 2018 (actual)
- First posted
- 29 Oct 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will determine the maximum tolerated dose and thus the recommended phase II dose and schedule of the compound and characterize the safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BGJ398 | Experimental | Drug: BGJ398 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BGJ398 | Drug | BGJ398 |
Primary outcome
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
4 secondary outcomes
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
To determine the pharmacokinetic (PK) profiles of oral BGJ398
Time frame: 23 months
To evaluate the pharmacodynamic effect of the drug.
Time frame: 23 months
Assess preliminary anti-tumor activity for patients not in Arm 4.
Time frame: 23 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2019.
- Individual participant dataSharing planned: No
Related trials for Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3
Active, not recruitingPhase 2
Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
50 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BGJ398 in people with Advanced Solid Tumors With Alterations of FGFR1, 2 and or 3 and Squamous Lung Cancer With FGFR1 Amplification, run by Novartis Pharmaceuticals at 50 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, France, Spain, Germany, Israel and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2019.