18 and older, any sex, with Advanced Cholangiocarcinoma or FGFR2 Gene Mutation. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Overall Response Rate (ORR) as Assessed by Blinded Independent Central Imaging Review (BICR)Primary· 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 pr
Group
Value
95% CI
Cohort 1: FGFR2 Fusions
23.1
15.6 – 32.2
Overall Response Rate (ORR) as Assessed by the InvestigatorSecondary· 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.
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 additi
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
32.4
23.7 – 42.1
Best Overall Response (BOR)Secondary· 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.
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 th
BOR by BICR
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
1
Cohort 1: FGFR2 Fusion/Rearrangements
24
Cohort 1: FGFR2 Fusion/Rearrangements
66
Cohort 1: FGFR2 Fusion/Rearrangements
11
BOR by Investigator
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
0
Cohort 1: FGFR2 Fusion/Rearrangements
35
Cohort 1: FGFR2 Fusion/Rearrangements
56
Cohort 1: FGFR2 Fusion/Rearrangements
11
Disease Control Rate (DCR)Secondary· 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.
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 dia
DCR by BICR
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
84.3
76.0 – 90.6
DCR by Investigator
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
84.3
76.0 – 90.6
Progression-Free Survival (PFS)Secondary· 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.
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 a
PFS by BICR
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
7.29
5.59 – 7.56
PFS by Investigator
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
6.74
5.55 – 7.56
Overall Survival (OS)Secondary· 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.
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.
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
11.86
10.68 – 14.85
Duration of Response (DOR)Secondary· 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.
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 lesion
DOR by BICR
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
5.55
3.78 – 7.66
DOR by Investigator
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
7.23
5.16 – 9.00
Response OnsetSecondary· 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 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 presp
Response onset by BICR
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
3.61
1.38 – 7.36
Response onset by Investigator
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
1.94
1.38 – 18.76
Growth Modulation Index (GMI)Secondary· 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.
The GMI is defined as the ratio of PFS (months) during treatment with infigratinib relative to the time (months) to progression (TTP) during treatment with last prior line of therapy.
Subjects served as their own control.
Results are provided for both BICR and 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.
PFS by BICR
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
1.22
0.00 – 120
PFS by Investigator
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
1.24
0.00 – 120
Retrospective Analysis of Post-second-line Antineoplastic Treatment Outcomes on ORRSecondary· 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.
Post-hoc subgroup assessment of efficacy in those subjects who were receiving infigratinib as a third or later line of treatment.
Investigator-assessed ORR was obtained from subjects' medical histories to provide a baseline evaluation of their response after historical second-line antineoplastic treatment prior to infigratinib treatment. Investigator-assessed ORR was then calculated in the same subjects after third- or later-line infigratinib therapy.
ORR is defined as the percentage (%) of patients with CR or PR, per RECIST (v1.1).
RECIST (v1.1) response criteria were as follows:
CR: disa
After Second-Line Therapy (Prior to Infigratinib Treatment)
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
0
0.0 – 6.1
Third or Later-Line Therapy (Infigratinib)
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
28.8
17.8 – 42.1
Retrospective Analysis of Post-second-line Antineoplastic Treatment Outcomes on PFSSecondary· 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.
Post-hoc subgroup assessment of efficacy in those subjects who were receiving infigratinib as a third or later line of treatment.
Investigator-assessed PFS was obtained from subjects' medical histories to provide a baseline evaluation of their response after historical second-line antineoplastic treatment prior to infigratinib treatment. Investigator-assessed PFS was then calculated in the same subjects after third- or later-line infigratinib therapy.
Note: The primary efficacy outcome measures were prespecified only for Cohort 1 (FGFR fusions) (disclosed 22 June 2022).
Due to early termina
After Second-Line Therapy (Prior to Infigratinib Treatment)
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
5.36
3.25 – 8.15
Third or Later-Line Therapy (Infigratinib)
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
6.93
4.76 – 7.59
Retrospective Analysis of Post-second-line Antineoplastic Treatment Outcomes on BORSecondary· 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.
Post-hoc subgroup assessment of efficacy in those subjects who were receiving infigratinib as a third or later line of treatment.
Investigator-assessed BOR was obtained from subjects' medical histories to provide a baseline evaluation of their response after historical second-line antineoplastic treatment prior to infigratinib treatment. Investigator-assessed BOR was then calculated in the same subjects after third- or later-line infigratinib therapy.
The endpoint is summarized for the rate of BOR of PD, SD, PR, and CR.
Note: The primary efficacy outcome measures were prespecified only for
After Second-Line Therapy (Prior to Infigratinib Treatment)
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
0
Cohort 1: FGFR2 Fusion/Rearrangements
0
Cohort 1: FGFR2 Fusion/Rearrangements
19
Cohort 1: FGFR2 Fusion/Rearrangements
22
Third or Later-Line Therapy (Infigratinib)
Group
Value
95% CI
Cohort 1: FGFR2 Fusion/Rearrangements
0
Cohort 1: FGFR2 Fusion/Rearrangements
17
Cohort 1: FGFR2 Fusion/Rearrangements
31
Cohort 1: FGFR2 Fusion/Rearrangements
7
Adverse events — posted to ClinicalTrials.gov
Time frame: From first administration of infigratinib with follow-up of at least 10 months after initial exposure. Data cutoff for Cohort 1 (primary analysis population) was 01 March 2021 (disclosed 22 June 2022). Data cutoff for Cohorts 2 and 3 (exploratory analysis only) was 07 February 2022..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Cohort 1: FGFR2 Fusions
Serious: 35/108 (32%)
Deaths: 93/108
Cohort 2: Other FGFR Genetic Alterations
Serious: 11/25 (44%)
Deaths: 19/25
Cohort 3: FGFR2 Fusions and Prior FGFR Inhibitor
Serious: 2/10 (20%)
Deaths: 7/10
Serious adverse events (66 terms)
Reaction
System
Cohort 1: FGFR2 Fusions
Cohort 2: Other FGFR Genet…
Cohort 3: FGFR2 Fusions an…
Anaemia
Blood and lymphatic system disorders
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—
—
Pyrexia
General disorders
—
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—
Hypercalcaemia
Metabolism and nutrition disorders
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Sepsis
Infections and infestations
—
—
—
Abdominal pain
Gastrointestinal disorders
—
—
—
Abdominal pain upper
Gastrointestinal disorders
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Ascites
Gastrointestinal disorders
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Constipation
Gastrointestinal disorders
—
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—
Cholangitis
Hepatobiliary disorders
—
—
—
Cellulitis
Infections and infestations
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—
Infection
Infections and infestations
—
—
—
Hypophosphataemia
Metabolism and nutrition disorders
—
—
—
Acute kidney injury
Renal and urinary disorders
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Atrial fibrillation
Cardiac disorders
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Tachycardia
Cardiac disorders
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Hypothyroidism
Endocrine disorders
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Diarrhoea
Gastrointestinal disorders
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Gastric ulcer haemorrhage
Gastrointestinal disorders
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Gastrointestinal haemorrhage
Gastrointestinal disorders
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Noninfective sialoadenitis
Gastrointestinal disorders
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Pancreatitis
Gastrointestinal disorders
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—
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Small intestinal obstruction
Gastrointestinal disorders
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—
—
Stomatitis
Gastrointestinal disorders
—
—
—
Intestinal ischaemia
Gastrointestinal disorders
—
—
—
Asthenia
General disorders
—
—
—
Other adverse events (119 terms — click to expand)
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by QED Therapeutics, a BridgeBio company
Last refreshed: 3 July 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02150967.