A Phase 2 interventional study of Pemigatinib in Cholangiocarcinoma, sponsored by Incyte Corporation. Completed at 120 sites in 12 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-08-14.
Sponsored by Incyte Corporation · Phase 2, Interventional, and Treatment
The purpose of this study is evaluate the efficacy of pemigatinib in subjects with advanced/metastatic or surgically unresectable cholangiocarcinoma with FGFR2 translocation who have failed at least 1 previous treatment.
914 studies on the registry are indexed under Cholangiocarcinoma; 286 are open to participants now.
This study's enrollment of 147 is above the median of 50 across 687 interventional studies indexed under Cholangiocarcinoma.
Browse Cholangiocarcinoma studies →Incyte Corporation is the lead sponsor of 286 studies on the registry; 37 are open to participants now.
Of its 144 completed or terminated interventional studies of FDA-regulated products, 93 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Pemigatinib in subjects with FGFR2 translocation with a documented fusion partner in central laboratory report
Drug: Pemigatinib
Pemigatinibin subjects with other FGF/FGFR alterations
Drug: Pemigatinib
Pemigatinib in subjects negative for FGF/FGFR alteration
Drug: Pemigatinib
Pemigatinibonce a day by mouth for 2 consecutive weeks and 1 week off therapy
Also known as: INCB054828
Objective Response Rate (ORR) in Participants With FGFR2 Rearrangements or Fusions
ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) at any post-Baseline visit prior to first progressive disease (PD), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1). CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
Time frame: up to 1527 days
ORR in Participants FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions
ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
Time frame: up to 424 days
ORR in All Participants With FGF/FGFR Alterations
ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
Time frame: up to 1527 days
ORR in Participants Negative for FGF/FGFR Alterations
ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
Time frame: up to 143 days
Progression-free Survival (PFS)
PFS was defined as the length of time from the first dose of study drug (Day 1) to the earlier of death or disease progression by RECIST v1.1, as assessed by the independent centralized radiological review committee.
Time frame: up to 50.17 months
Duration of Response (DOR)
DOR was defined as the time from the first overall response contributing to an objective response (CR or PR) as assessed by an independent centralized radiological review committee to the earlier of death or first overall response of PD occurring after the first overall response contributing to the objective response. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion.
Time frame: up to 47.11 months
Disease Control Rate (DCR)
DCR was defined as the proportion of participants with an overall response of CR, PR, or stable disease (SD), per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion. SD: no change in target lesions to qualify for CR, PR, or PD.
Time frame: up to 1527 days
Overall Survival
Overall survival was defined as the length of time from the first dose of study drug (Day 1) until the date of death due to any cause.
Time frame: up to 51.32 months
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy (e.g., hematologic abnormality that required transfusion), or required changes in the study drug(s). A TEAE was defined as any adverse event either reported for the first time or the worsening of a pre-existing event after the first dose of study drug and within 30 days of the last dose of study drug.
Time frame: up to 1584 days
First-order Absorption Rate Constant (ka) of Pemigatinib
First-order absorption rate constant is defined as the rate at which a drug enters into the system.
Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose
CL/F of Pemigatinib
CL/F is defined as apparent oral clearance.
Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose
Vc/F of Pemigatinib
Vc/F is defined as the apparent volume of distribution for the central compartment of pemigatinib.
Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose
Vp/F of Pemigatinib
Vp/F is defined as the apparent volume of distribution for the tissue (peripheral) compartment.
Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose
| Milestone | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Started | 108 | 20 | 17 | 2 |
| Completed | 0 | 0 | 0 | 0 |
| Not completed | 108 | 20 | 17 | 2 |
| Withdrew: Progressive disease | 4 | 0 | 1 | 0 |
| Withdrew: Death | 73 | 18 | 15 | 2 |
| Withdrew: Lost to follow-up | 3 | 0 | 0 | 0 |
| Withdrew: Study terminated by sponsor | 19 | 0 | 0 | 0 |
| Withdrew: Physician decision | 1 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 7 | 2 | 1 | 0 |
| Withdrew: Rolled over to another study | 1 | 0 | 0 | 0 |
ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) at any post-Baseline visit prior to first progressive disease (PD), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1). CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
| percentage of participants | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Objective Response Rate (ORR) in Participants With FGFR2 Rearrangements or Fusions | 37.0 (27.94 to 46.86) | — | — | — |
ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
| percentage of participants | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| ORR in Participants FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | — | 0.0 (0.0 to 16.84) | — | — |
ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
| percentage of participants | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Cohort A + Cohort B |
|---|---|---|---|---|---|
| ORR in All Participants With FGF/FGFR Alterations | — | — | — | — | 31.3 (23.35 to 40.04) |
ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.
| percentage of participants | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| ORR in Participants Negative for FGF/FGFR Alterations | — | — | 0.0 (0.0 to 19.51) | — |
PFS was defined as the length of time from the first dose of study drug (Day 1) to the earlier of death or disease progression by RECIST v1.1, as assessed by the independent centralized radiological review committee.
| months | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Progression-free Survival (PFS) | 7.03 (6.08 to 10.48) | 2.10 (1.18 to 4.86) | 1.51 (1.38 to 1.84) | — |
DOR was defined as the time from the first overall response contributing to an objective response (CR or PR) as assessed by an independent centralized radiological review committee to the earlier of death or first overall response of PD occurring after the first overall response contributing to the objective response. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion.
| months | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Duration of Response (DOR) | 9.13 (6.01 to 14.49) | — | — | — |
DCR was defined as the proportion of participants with an overall response of CR, PR, or stable disease (SD), per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion. SD: no change in target lesions to qualify for CR, PR, or PD.
| percentage of participants | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Disease Control Rate (DCR) | 82.4 (73.9 to 89.1) | 40.0 (19.1 to 63.9) | 17.6 (3.8 to 43.4) | — |
Overall survival was defined as the length of time from the first dose of study drug (Day 1) until the date of death due to any cause.
| months | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Overall Survival | 17.48 (14.36 to 22.93) | 6.70 (2.10 to 10.55) | 3.98 (1.97 to 4.60) | — |
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy (e.g., hematologic abnormality that required transfusion), or required changes in the study drug(s). A TEAE was defined as any adverse event either reported for the first time or the worsening of a pre-existing event after the first dose of study drug and within 30 days of the last dose of study drug.
| Participants | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other |
|---|---|---|---|---|
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | 108 | 20 | 17 | 2 |
First-order absorption rate constant is defined as the rate at which a drug enters into the system.
| 1/hour | All Cohorts |
|---|---|
| First-order Absorption Rate Constant (ka) of Pemigatinib | 1.29 ± 0.827 |
CL/F is defined as apparent oral clearance.
| Liters/hour | All Cohorts |
|---|---|
| CL/F of Pemigatinib | 12.2 ± 5.28 |
Vc/F is defined as the apparent volume of distribution for the central compartment of pemigatinib.
| Liters | All Cohorts |
|---|---|
| Vc/F of Pemigatinib | 144 ± 55.7 |
Vp/F is defined as the apparent volume of distribution for the tissue (peripheral) compartment.
| Liters | All Cohorts |
|---|---|
| Vp/F of Pemigatinib | 85.6 ± 30.5 |
Collected over up to 1584 days. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort A: FGFR2 Rearrangements or Fusions | 76/108 (70.4%) | 46/108 (42.6%) | 108/108 (100%) |
| Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | 18/20 (90%) | 10/20 (50%) | 20/20 (100%) |
| Cohort C: Negative for FGF/FGFR Alterations | 15/17 (88.2%) | 12/17 (70.6%) | 17/17 (100%) |
| Other | 2/2 (100%) | 0/2 (0%) | 2/2 (100%) |
| Total | 111/147 (75.5%) | 68/147 (46.3%) | 147/147 (100%) |
| Event | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Total |
|---|---|---|---|---|---|
| Abdominal painGastrointestinal disorders | 4/108 | 1/20 | 2/17 | 0/2 | 7/147 |
| Back painMusculoskeletal and connective tissue disorders | 0/108 | 0/20 | 2/17 | 0/2 | 2/147 |
| HyponatraemiaMetabolism and nutrition disorders | 1/108 | 2/20 | 0/17 | 0/2 | 3/147 |
| Pleural effusionRespiratory, thoracic and mediastinal disorders | 2/108 | 2/20 | 1/17 | 0/2 | 5/147 |
| Acute kidney injuryRenal and urinary disorders | 2/108 | 0/20 | 1/17 | 0/2 | 3/147 |
| AnaemiaBlood and lymphatic system disorders | 1/108 | 0/20 | 1/17 | 0/2 | 2/147 |
| Anaphylactic reactionImmune system disorders | 0/108 | 0/20 | 1/17 | 0/2 | 1/147 |
| BilomaHepatobiliary disorders | 0/108 | 0/20 | 1/17 | 0/2 | 1/147 |
| Blood creatinine increasedInvestigations | 1/108 | 0/20 | 1/17 | 0/2 | 2/147 |
| CholangitisHepatobiliary disorders | 5/108 | 0/20 | 1/17 | 0/2 | 6/147 |
| Event | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Total |
|---|---|---|---|---|---|
| AlopeciaSkin and subcutaneous tissue disorders | 64/108 | 4/20 | 3/17 | 2/2 | 73/147 |
| Dry mouthGastrointestinal disorders | 42/108 | 5/20 | 1/17 | 2/2 | 50/147 |
| Dry skinSkin and subcutaneous tissue disorders | 30/108 | 0/20 | 0/17 | 2/2 | 32/147 |
| DysgeusiaNervous system disorders | 45/108 | 3/20 | 3/17 | 2/2 | 53/147 |
| HyperphosphataemiaMetabolism and nutrition disorders | 60/108 | 13/20 | 12/17 | 1/2 | 86/147 |
| DiarrhoeaGastrointestinal disorders | 58/108 | 5/20 | 6/17 | 1/2 | 70/147 |
| FatigueGeneral disorders | 50/108 | 5/20 | 9/17 | 0/2 | 64/147 |
| Abdominal painGastrointestinal disorders | 22/108 | 3/20 | 3/17 | 1/2 | 29/147 |
| Activated partial thromboplastin time prolongedInvestigations | 3/108 | 0/20 | 0/17 | 1/2 | 4/147 |
| ConstipationGastrointestinal disorders | 46/108 | 5/20 | 2/17 | 1/2 | 54/147 |
| Age, Continuous(years) | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Total |
|---|---|---|---|---|---|
| Mean | 55.2 ± 12.00 | 61.9 ± 10.99 | 65.6 ± 7.12 | 41.0 ± 14.14 | 57.1 ± 12.08 |
| Sex: Female, Male(Participants) | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Total |
|---|---|---|---|---|---|
| Female | 66 | 11 | 7 | 1 | 85 |
| Male | 42 | 9 | 10 | 1 | 62 |
| Race/Ethnicity, Customized(Participants) | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Total |
|---|---|---|---|---|---|
| White | 79 | 9 | 14 | 2 | 104 |
| Black or African American | 7 | 0 | 1 | 0 | 8 |
| Asian | 12 | 11 | 0 | 0 | 23 |
| American-Indian/Alaska Native | 0 | 0 | 1 | 0 | 1 |
| Captured as "Other" | 4 | 0 | 1 | 0 | 5 |
| Missing | 6 | 0 | 0 | 0 | 6 |
| Race/Ethnicity, Customized(Participants) | Cohort A: FGFR2 Rearrangements or Fusions | Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions | Cohort C: Negative for FGF/FGFR Alterations | Other | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 2 | 0 | 4 | 0 | 6 |
| Not Hispanic or Latino | 88 | 18 | 13 | 2 | 121 |
| Not Reported | 15 | 0 | 0 | 0 | 15 |
| Unknown | 1 | 0 | 0 | 0 | 1 |
| Captured as "Other" | 2 | 2 | 0 | 0 | 4 |
Showing the first 100 of 120 sites across 12 countries.
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency
Supporting information: Study protocol, Sap
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