Advanced CholangiocarcinomaPhase 2NCT02150967
A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma
A Phase 2 study testing BGJ398 (infigratinib) in people with Advanced Cholangiocarcinoma and FGFR2 Gene Mutation, run by QED Therapeutics, a BridgeBio company at 37 sites in 11 countries.
Trial summary
This is a multi-center, open label, single arm phase II study evaluating BGJ398 (infigratinib) anti-tumor activity in advanced or metastatic cholangiocarcinoma patients with fibroblast growth factor receptor (FGFR) genetic alterations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
- What is being tested?
- BGJ398 (infigratinib)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 143 participants
- Where
- 37 sitesin 11 countries
- Study dates
- 23 Jul 2014to 7 Feb 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Adult patients with histologically or cytologically confirmed cholangiocarcinoma at the time of diagnosis.
- Patients with cancers of the gallbladder or ampulla of Vater are not eligible.
- Patients must have received at least one prior regimen containing gemcitabine with or without cisplatin for advanced/ metastatic disease. Patient should have evidence of progressive disease following prior regimen, or if prior treatment discontinued due to toxicity must have continued evidence of measurable or evaluable disease.
Who cannot join
- Prior or current treatment with a MEK inhibitor (all Cohorts), BGJ398 (infigratinib) (all Cohorts), or selective FGFR inhibitor (Cohorts 1 and 2 only).
- insufficient organ function
- Absolute Neutrophil Count (ANC) < 1,000/mm3 [1.0 x 10^9/L]
- Platelets < 75,000/mm3 [75 x 10^9/L]
- Hemoglobin < 109.0 g/dL
- Total bilirubin > 1.5x upper limit of normal (ULN)
Show all 11Show fewer
- Aspartate aminotransferase/glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/glutamic pyruvic transaminase (ALT/SGPT) > 2.5x ULN (AST and ALT > 5x ULN in the presence of liver metastases)
- Serum creatinine > 1.5x ULN and a calculated or measured creatinine clearance < 45 mL/min
- Inorganic phosphorus outside of normal limits
- Total and ionized serum calcium outside of normal limits
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
37 sites in 11 countries.
37 sites in 11 countries
United States11 sites
QED Investigative Site
Boston, Massachusetts, 02114
QED Investigative Site
Columbus, Ohio, 43221
QED Investigative Site
Detroit, Michigan, 48201
QED Investigative Site
Houston, Texas, 77030-4009
QED Investigative Site
Los Angeles, California, 90033
QED Investigative Site
Los Angeles, California, 90095
QED Investigative Site
New York, New York, 10016
QED Investigative Site
New York, New York, 10029
QED Investigative Site
New York, New York, 10065
QED Investigative Site
Phoenix, Arizona, 85054
QED Investigative Site
San Francisco, California, 94158
United Kingdom4 sites
QED Investigative Site
Bebington, CH63 4JY
QED Investigative Site
Birmingham, B15 2TH
QED Investigative Site
Manchester, M20 4BX
QED Investigative Site
Nottingham, NG5 1PB
Germany3 sites
QED Investigative Site
Cologne, North Rhine-Westphalia, 50937
QED Investigative Site
Heidelberg, 69120
QED Investigative Site
Tübingen
Italy3 sites
QED Investigative Site
Ancona, AN, 60126
QED Investigative Site
Milan, MI, 20132
QED Investigative Site
Roma, RM, 00168
Spain3 sites
QED Investigative Site
Barcelona, 8035
QED Investigative Site
Barcelona, 8908
QED Investigative Site
Madrid, 28050
Thailand3 sites
QED Investigative Site
Bangkok, 10330
QED Investigative Site
Bangkok, 10400
QED Investigative Site
Khon Kaen, THA, 40002
Belgium2 sites
QED Investigative Site
Brussels, 1200
QED Investigative Site
Leuven, 3000
Russia2 sites
QED Investigative Site
Moscow, 125367
QED Investigative Site
Volzhskiy, 404133
Singapore2 sites
QED Investigative Site
Singapore, 119228
QED Investigative Site
Singapore, 169610
South Korea2 sites
QED Investigative Site
Seoul, Korea, 03080
QED Investigative Site
Seoul, Korea, 06351
Taiwan2 sites
QED Investigative Site
Taipei, Taiwan ROC, 10041
QED Investigative Site
Zhunan, 35053
About this study
Official title: A Phase II Multicenter, Single Arm Study of Oral BGJ398 in Adult Patients With Advanced or Metastatic Cholangiocarcinoma With FGFR2 Gene Fusions or Other FGFR Genetic Alterations Who Failed or Are Intolerant to Platinum-based Chemotherapy
This is a multi-center, open label, single arm phase II study evaluating BGJ398 (infigratinib) anti-tumor activity in advanced or metastatic cholangiocarcinoma patients with fibroblast growth factor receptor (FGFR) genetic alterations.
Read the detailed description
Adult patients with histologically or cytologically confirmed advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations or other FGFR genetic alterations have been enrolled. Subjects must have received at least one prior regimen containing gemcitabine with or without cisplatin for advanced/metastatic disease. Subjects should have had evidence of progressive disease following their prior regimen or if prior treatment was discontinued due to toxicity must have continued evidence of measurable disease. Up to approximately 160 adult patients over age 18, both male and female were planned for enrollment.
Three cohorts of subjects comprise the study population:
Cohort 1: subjects with FGFR2 gene fusions (ie, fusions or rearrangements [formerly translocations]).
Cohort 2: subjects with FGFR genetic alterations other than FGFR2 gene fusions or rearrangements.
Cohort 3: subjects with FGFR2 gene fusions or rearrangements who have received a prior FGFR inhibitor.
All subjects received oral BGJ398 (infigratinib), once-daily, on a three weeks on (21 days), one week off (7 days) schedule. One treatment cycle consists of 28 days.
Notes:
Cohort 1 was pre-specified as the primary analysis population. Results of these analyses were previously disclosed (posted 22 June 2022). There were no additional efficacy or safety endpoints to assess in Cohort 1 after primary completion (01 March 2021).
Cohorts 2 and 3 were added at protocol amendment (PA) 4 to support only exploratory efficacy objectives of the study. These cohorts were ongoing the time of primary completion (01 March 2021). After interim review of the data from these cohorts (as permitted by the protocol) only limited efficacy was observed and the sponsor terminated the study early. Therefore, a formal efficacy analysis was not performed for Cohorts 2 and 3. However, baseline characteristics and safety data were analyzed.
- Conditions
- Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
- Being tested
- BGJ398 (infigratinib)
- Keywords
- cholangiocarcinoma,, FGFR2 gene fusion,, FGFR genetic alteration
- Medical subject headings
- Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BGJ398 (infigratinib)Drug
Capsule for oral use
| Study group | Role | What they receive |
|---|---|---|
| BGJ398 (infigratinib) | Experimental | To estimate the anti-tumor activity of BGJ398 (infigratinib) Receives: Drug: BGJ398 (infigratinib) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR) as Assessed by Blinded Independent Central Imaging Review (BICR)
ORR is defined as the percentage (%) of subjects with a best overall response of Complete Response (CR) or Partial Response (PR), as per Response Evaluation Criteria in Solid Tumors (RECIST), Version 1.1, evaluated by computed tomography (CT) or magnetic resonance imaging (MRI) scans every 28 days.
RECIST (v1.1) response criteria were as follows:
CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm.
PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions). Results were previously disclosed (22 June 2022). There are no additional efficacy endpoints to assess for Cohort 1, thus efficacy data were not re-analyzed for the final analysis.
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis was conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Secondary outcomes (7)
Overall Response Rate (ORR) as Assessed by the Investigator
ORR is defined as the percentage (%) of subjects with a best overall response of CR or PR, evaluated by CT or MRI scans every 28 days.
RECIST (v1.1) response criteria were as follows:
CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm.
PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) only. These results were previously disclosed (22 June 2022).
There are no additional efficacy endpoints to assess for Cohort 1, thus efficacy data were not re-analyzed for the final analysis.
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff for the primary analysis was 01 March 2021.
Best Overall Response (BOR)
BOR is defined as the best overall response a subject achieved during the study before any subsequent antineoplastic therapy. The endpoint is summarized for the rate of BOR of CR, PR, progressive disease (PD), and stable disease (SD), evaluated by CT or MRI scans every 28 days.
RECIST (v1.1) response criteria were as follows:
CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm.
PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions.
PD: at least a 20% increase in the sum of diameters of all target lesions from that of the smallest sum on study and an absolute increase in target lesion of at least 5mm.
SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters while on study.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusion)
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Disease Control Rate (DCR)
DCR is the percentage (%) of subjects with a BOR of CR, PR, or SD, evaluated by CT or MRI scans every 28 days.
Results are based on both BICR and on Investigator assessment.
RECIST (v1.1) response criteria were as follows:
CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm.
PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions.
SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters while on study.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) (disclosed 22 June 2022).
There are no additional efficacy endpoints to assess for Cohort 1, thus efficacy data were not re-analyzed for the final analysis.
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Progression-Free Survival (PFS)
PFS was calculated as the number of months from the first dose of study drug to the first documented progression or death due to any cause, whichever occurred earlier. Subjects without an assessment of progression or death were censored at the last adequate tumor assessment. For subjects who had an event after ≥2 missed visits, the subject was censored at the last adequate tumor assessment before the missing visit.
Disease progression was assessed per RECIST (v1.1) and defined as at least a 20% increase in the sum of diameters of all target lesions from that of the smallest sum on study and an absolute increase in target lesion of at least 5mm.
Results are based on both BICR and on Investigator assessment.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) (disclosed 22 June 2022).
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Overall Survival (OS)
OS was defined as the time (months) from the date of start of treatment to the date of death due to any cause.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) (disclosed 22 June 2022).
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Duration of Response (DOR)
DOR is defined as the time (months) from the initial response to the time of the event; defined as the first documented progression or death due to any cause, whichever was earlier.
Note that results are based on a subgroup of subjects with confirmed responses (CR or PR) as assessed by BICR or by the Investigator.
RECIST (v1.1) response criteria was as follows:
CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm.
PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) (disclosed 22 June 2022).
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Response Onset
Response onset was defined as the time (months) from the first study treatment administration date to the initial response.
Note that results are based on a subgroup of subjects with confirmed responses (CR or PR) as assessed by BICR or by the Investigator.
RECIST (v1.1) response criteria was as follows:
CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm.
PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) (disclosed 22 June 2022).
Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
Measured: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2014 (estimated)
- Study start23 Jul 2014
- Primary completion1 Mar 2021
- Study completion7 Feb 2022
Why it stopped: The study was terminated early due to limited efficacy in Cohorts 2 and 3 (exploratory endpoints). Termination was not due to concerns about safety and had no impact on the primary efficacy analysis (ie, results reported for Cohort 1).
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Advanced CholangiocarcinomaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 143 participants (actual)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Helsinn Healthcare SA
- Responsible party
- Sponsor
- Investigators
- QED Therapeutics, Study director, QED Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02150967
- Sponsor's protocol ID
- CBGJ398X2204
- Other IDs
- 2013-005085-19
- Status last verified
- Jun 2023
- First submitted
- 12 May 2014
- Last updated
- 3 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Javle M, Roychowdhury S, Kelley RK, Sadeghi S, Macarulla T, Weiss KH, Waldschmidt DT, Goyal L, Borbath I, El-Khoueiry A, Borad MJ, Yong WP, Philip PA, Bitzer M, Tanasanvimon S, Li A, Pande A, Soifer HS, Shepherd SP, Moran S, Zhu AX, Bekaii-Saab TS, Abou-Alfa GK. Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. Lancet Gastroenterol Hepatol. 2021 Oct;6(10):803-815. doi: 10.1016/S2468-1253(21)00196-5. Epub 2021 Aug 3.
PubMed 34358484Javle M, Lowery M, Shroff RT, Weiss KH, Springfeld C, Borad MJ, Ramanathan RK, Goyal L, Sadeghi S, Macarulla T, El-Khoueiry A, Kelley RK, Borbath I, Choo SP, Oh DY, Philip PA, Chen LT, Reungwetwattana T, Van Cutsem E, Yeh KH, Ciombor K, Finn RS, Patel A, Sen S, Porter D, Isaacs R, Zhu AX, Abou-Alfa GK, Bekaii-Saab T. Phase II Study of BGJ398 in Patients With FGFR-Altered Advanced Cholangiocarcinoma. J Clin Oncol. 2018 Jan 20;36(3):276-282. doi: 10.1200/JCO.2017.75.5009. Epub 2017 Nov 28.
PubMed 29182496Borad MJ, Gores GJ, Roberts LR. Fibroblast growth factor receptor 2 fusions as a target for treating cholangiocarcinoma. Curr Opin Gastroenterol. 2015 May;31(3):264-8. doi: 10.1097/MOG.0000000000000171.
PubMed 25763789
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Multicenter, Single Arm Study of Oral BGJ398 in Adult Patients With Advanced or Metastatic Cholangiocarcinoma With FGFR2 Gene Fusions or Other FGFR Genetic Alterations Who Failed or Are Intolerant to Platinum-based Chemotherapy
- ClinicalTrials.gov ID
- NCT02150967
- Acronym / sponsor ID
- CBGJ398X2204
- Conditions
- Advanced Cholangiocarcinoma, FGFR2 Gene Mutation
- Keywords
- cholangiocarcinoma,, FGFR2 gene fusion,, FGFR genetic alteration
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated early due to limited efficacy in Cohorts 2 and 3 (exploratory endpoints). Termination was not due to concerns about safety and had no impact on the primary efficacy analysis (ie, results reported for Cohort 1).
- Enrolment
- 143 participants (actual)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Collaborators
- Helsinn Healthcare SA
- Study start
- 23 Jul 2014 (actual)
- Primary completion
- 1 Mar 2021 (actual)
- Study completion
- 7 Feb 2022 (actual)
- First posted
- 30 May 2014 (estimated)
- Results
- Posted 15 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multi-center, open label, single arm phase II study evaluating BGJ398 (infigratinib) anti-tumor activity in advanced or metastatic cholangiocarcinoma patients with fibroblast growth factor receptor (FGFR) genetic alterations.
Detailed description
Adult patients with histologically or cytologically confirmed advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations or other FGFR genetic alterations have been enrolled. Subjects must have received at least one prior regimen containing gemcitabine with or without cisplatin for advanced/metastatic disease. Subjects should have had evidence of progressive disease following their prior regimen or if prior treatment was discontinued due to toxicity must have continued evidence of measurable disease. Up to approximately 160 adult patients over age 18, both male and female were planned for enrollment.
Three cohorts of subjects comprise the study population:
Cohort 1: subjects with FGFR2 gene fusions (ie, fusions or rearrangements [formerly translocations]).
Cohort 2: subjects with FGFR genetic alterations other than FGFR2 gene fusions or rearrangements.
Cohort 3: subjects with FGFR2 gene fusions or rearrangements who have received a prior FGFR inhibitor.
All subjects received oral BGJ398 (infigratinib), once-daily, on a three weeks on (21 days), one week off (7 days) schedule. One treatment cycle consists of 28 days.
Notes:
Cohort 1 was pre-specified as the primary analysis population. Results of these analyses were previously disclosed (posted 22 June 2022). There were no additional efficacy or safety endpoints to assess in Cohort 1 after primary completion (01 March 2021).
Cohorts 2 and 3 were added at protocol amendment (PA) 4 to support only exploratory efficacy objectives of the study. These cohorts were ongoing the time of primary completion (01 March 2021). After interim review of the data from these cohorts (as permitted by the protocol) only limited efficacy was observed and the sponsor terminated the study early. Therefore, a formal efficacy analysis was not performed for Cohorts 2 and 3. However, baseline characteristics and safety data were analyzed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BGJ398 (infigratinib) | Experimental | Drug: BGJ398 (infigratinib)To estimate the anti-tumor activity of BGJ398 (infigratinib) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Adult patients with histologically or cytologically confirmed cholangiocarcinoma at the time of diagnosis.
- Patients with cancers of the gallbladder or ampulla of Vater are not eligible.
- Patients must have received at least one prior regimen containing gemcitabine with or without cisplatin for advanced/ metastatic disease. Patient should have evidence of progressive disease following prior regimen, or if prior treatment discontinued due to toxicity must have continued evidence of measurable or evaluable disease.
Key exclusion criteria (11 listed)
- Prior or current treatment with a MEK inhibitor (all Cohorts), BGJ398 (infigratinib) (all Cohorts), or selective FGFR inhibitor (Cohorts 1 and 2 only).
- insufficient organ function
- Other protocol-defined inclusion/exclusion criteria may apply.
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 (infigratinib)Capsule for oral use | Drug | BGJ398 (infigratinib) |
Primary outcome
Overall Response Rate (ORR) as Assessed by Blinded Independent Central Imaging Review (BICR)
Time frame: Analysis was conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
ORR is defined as the percentage (%) of subjects with a best overall response of Complete Response (CR) or Partial Response (PR), as per Response Evaluation Criteria in Solid Tumors (RECIST), Version 1.1, evaluated by computed tomography (CT) or magnetic resonance imaging (MRI) scans every 28 days. RECIST (v1.1) response criteria were as follows: CR: disappearance of all target lesions. Any pathological lymph node (whether target or non-target) must have reduction in short axis to <10 mm. PR: at least a 30% decrease from baseline in the sum of diameters of all target lesions. Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions). Results were previously disclosed (22 June 2022). There are no additional efficacy endpoints to assess for Cohort 1, thus efficacy data were not re-analyzed for the final analysis. Due to early termination of the study, no formal efficacy analyses were performed for Cohorts 2 and 3.
7 secondary outcomes
Overall Response Rate (ORR) as Assessed by the Investigator
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff for the primary analysis was 01 March 2021.
Best Overall Response (BOR)
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Disease Control Rate (DCR)
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Progression-Free Survival (PFS)
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Overall Survival (OS)
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Duration of Response (DOR)
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Response Onset
Time frame: Analysis conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34358484Javle M, Roychowdhury S, Kelley RK, Sadeghi S, Macarulla T, Weiss KH, Waldschmidt DT, Goyal L, Borbath I, El-Khoueiry A, Borad MJ, Yong WP, Philip PA, Bitzer M, Tanasanvimon S, Li A, Pande A, Soifer HS, Shepherd SP, Moran S, Zhu AX, Bekaii-Saab TS, Abou-Alfa GK. Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. Lancet Gastroenterol Hepatol. 2021 Oct;6(10):803-815. doi: 10.1016/S2468-1253(21)00196-5. Epub 2021 Aug 3.
- Derived from this study: PubMed 29182496Javle M, Lowery M, Shroff RT, Weiss KH, Springfeld C, Borad MJ, Ramanathan RK, Goyal L, Sadeghi S, Macarulla T, El-Khoueiry A, Kelley RK, Borbath I, Choo SP, Oh DY, Philip PA, Chen LT, Reungwetwattana T, Van Cutsem E, Yeh KH, Ciombor K, Finn RS, Patel A, Sen S, Porter D, Isaacs R, Zhu AX, Abou-Alfa GK, Bekaii-Saab T. Phase II Study of BGJ398 in Patients With FGFR-Altered Advanced Cholangiocarcinoma. J Clin Oncol. 2018 Jan 20;36(3):276-282. doi: 10.1200/JCO.2017.75.5009. Epub 2017 Nov 28.
- Derived from this study: PubMed 25763789Borad MJ, Gores GJ, Roberts LR. Fibroblast growth factor receptor 2 fusions as a target for treating cholangiocarcinoma. Curr Opin Gastroenterol. 2015 May;31(3):264-8. doi: 10.1097/MOG.0000000000000171.
- Individual participant dataSharing planned: No
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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.
QED Therapeutics study director
QED Therapeutics
37 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BGJ398 (infigratinib) in people with Advanced Cholangiocarcinoma and FGFR2 Gene Mutation, run by QED Therapeutics, a BridgeBio company at 37 sites in 11 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 3 inclusion and 11 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: stopped early, as of the sponsor's last update on 3 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, United Kingdom, Germany, Italy, Spain and 6 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Response Rate (ORR) as Assessed by Blinded Independent Central Imaging Review (BICR) (time frame: Analysis was conducted when all subjects in Cohort 1 had the opportunity to be followed for at least 10 months after their initial exposure to infigratinib. Data cutoff 01 March 2021.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02150967, which also lists 3 linked publications.
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 3 Jul 2023.