A Phase 3 interventional study of Pemigatinib and Gemcitabine in Unresectable Cholangiocarcinoma and Metastatic Cholangiocarcinoma, sponsored by Incyte Corporation. Terminated at 215 sites in 19 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-11.
Sponsored by Incyte Corporation · Phase 3, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy and safety of pemigatinib versus gemcitabine plus cisplatin chemotherapy in first-line treatment of participants with unresectable or metastatic cholangiocarcinoma with FGFR2 rearrangement.
914 studies on the registry are indexed under Cholangiocarcinoma; 286 are open to participants now.
This study's enrollment of 167 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:
Drug: Pemigatinib
Participants who experience disease progression while receiving gemcitabine + cisplatin or during the follow-up period and before starting a new anticancer therapy will be eligible to cross over and receive pemigatinib.
Drug: Gemcitabine · Drug: Cisplatin
Pemigatinib at the protocol-defined dose administered orally once daily as continuous therapy schedule (a cycle is 3 weeks).
Also known as: INCB054828
Gemcitabine 1000 mg/m\^2 administered as an intravenous infusion on Days 1 and 8 of every 3-week cycle for up to 8 cycles.
Cisplatin 25 mg/m\^2 administered as an intravenous infusion on Days 1 and 8 of every 3-week cycle for up to 8 cycles.
Randomized Treatment Period: Progression-free Survival (PFS)
PFS was defined as the time from the date of randomization until the date of disease progression (according to Response Evaluation Criteria in Solid Tumors version 1.1 \[RECIST v1.1\] and assessed by an Independent Central Review \[ICR\]) or death, whichever occurs first.
Time frame: up to 1422 days
Randomized Treatment Period: Kaplan-Meier Estimates of PFS of the Indicated Lengths of Time
PFS was defined as the time from the date of randomization until the date of disease progression (according to RECIST v1.1 and assessed by an ICR) or death, whichever occurs first. Kaplan-Meier estimates indicate the percent probability of a participant being alive at the indicated time after treatment start.
Time frame: up to 1422 days
Transition Period: PFS
PFS was defined as the time from the date of the first dose of study drug after transition until the earliest date of progressive disease or death due to any cause.
Time frame: up to 1496 days
Transition Period: Kaplan-Meier Estimates of PFS of the Indicated Lengths of Time
PFS was defined as the time from the date of the first dose of study drug after transition until the earliest date of progressive disease or death due to any cause. Kaplan-Meier estimates indicate the percent probability of a participant being alive at the indicated time after treatment start.
Time frame: up to 1496 days
Randomized Treatment Period: Objective Response Rate (ORR)
ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) per RECIST v1.1 as assessed by an ICR. 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.
Time frame: up to 1422 days
Transition Period: ORR
ORR was defined as the percentage of participants with a best overall response of CR or PR per RECIST v1.1 as assessed by an ICR. 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.
Time frame: up to 1496 days
Randomized Treatment Period: Overall Survival
Overall survival was defined as the time from the date of randomization until death due to any cause.
Time frame: up to 1422 days
Randomized Treatment Period: Kaplan-Meier Estimates of Overall Survival of the Indicated Lengths of Time
Overall survival was defined as the time from the date of randomization until death due to any cause. Kaplan-Meier estimates indicate the percent probability of a participant being alive at the indicated time after treatment start.
Time frame: up to 1422 days
Randomized Treatment Period: Duration of Response (DOR)
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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.
Time frame: up to 1422 days
Randomized Treatment Period: Kaplan-Meier Estimates of DOR of the Indicated Lengths of Time
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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. Kaplan-Meier estimates indicate the percent probability of a participant still having a CR or PR at the indicated time after treatment start.
Time frame: up to 1422 days
Transition Period: DOR
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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.
Time frame: up to 1496 days
Transition Period: Kaplan-Meier Estimates of DOR of the Indicated Lengths of Time
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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. Kaplan-Meier estimates indicate the percent probability of a participant still having a CR or PR at the indicated time after treatment start.
Time frame: up to 1496 days
Randomized Treatment Period: Disease Control Rate (DCR)
DCR was defined as the percentage of participants who achieved a best overall response of CR, PR, or SD per RECIST v1.1 as assessed by an ICR. 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 1422 days
Transition Period: DCR
DCR was defined as the percentage of participants who achieved a best overall response of CR, PR, or SD per RECIST v1.1 as assessed by an ICR. 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 1496 days
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug until 30 days after the last dose of study drug for participants who did not transition to pemigatinib. For participants who transitioned to pemigatinib, the end date was 30 days after the last dose date of gemcitabine plus cisplatin or the day before the first dose of pemigatinib after transition, whichever occurred first.
Time frame: up to 1457 days
Number of Participants With Any Treatment-related TEAE
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug until 30 days after the last dose of study drug for participants who did not transition to pemigatinib. For participants who transitioned to pemigatinib, the end date was 30 days after the last dose date of gemcitabine plus cisplatin or the day before the first dose of pemigatinib after transition, whichever occurred first.
Time frame: up to 1457 days
Transition Period: Number of Participants With Any TEAE
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE for the Crossover Evaluable Population was any AE either reported for the first time or for a worsened pre-existing event after the first dose of pemigatinib after switch and until 30 days after the last dose of pemigatinib.
Time frame: up to 1531 days
Transition Period: Number of Participants With Any Treatment-related TEAE
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE for the Crossover Evaluable Population was any AE either reported for the first time or for a worsened pre-existing event after the first dose of pemigatinib after switch and until 30 days after the last dose of pemigatinib.
Time frame: up to 1531 days
Randomized Treatment Period: Change From Baseline (CFB) in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-30) Scores at Early Termination
The EORTC QLQ-C30 is a 30-item scale composed of both multi-item scales and single-item measures. These include 5 functional scales (physical functioning, role, cognitive functioning, emotional functioning, and social functioning), 3 symptom scales (fatigue, pain, and nausea/vomiting), a global health status scale, and 6 single items (constipation, diarrhea, sleep, dyspnea, appetite, financial). All scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Therefore, a high score for a functional scale represents a high/healthy level of functioning and a high score for the global health status/QoL represents a high QoL. A high score for a symptom scale/item represents a high level of symptomatology/problems. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1422 days
Randomized Treatment Period: Change From Baseline (CFB) in the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Including 21 Questions Specific to the Biliary System (EORTC QLQ-BIL21) at Early Termination
The EORTC QLQ-BIL21 assessed QoL across 8 scales: eating, jaundice, tiredness, pain, anxiety, treatment side effects, drains, and weight loss. The raw score of each scale is the mean of the item values that contribute to the scale, and the standardized score is a linear transformation of the raw score so that the range is from 0 to 100. A high score for a symptom scale represents a high level of symptomatology/problems. The BIL21 questionnaire was only be administered to participants for whom the questionnaire is validated in that language. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; up to 1422 days
Randomized Treatment Period: Number of Participants With the Indicated Responses on the European Quality of Life 5 Dimensions (3 Levels) Questionnaire (EQ-5D [3L])
The EQ-5D (3L) is the descriptive system that comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 3 levels: no problems, some problems, extreme problems.
Time frame: Baseline; up to 1422 days
| Milestone | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Started | 83 | 84 |
| Completed | 0 | 0 |
| Not completed | 83 | 84 |
| Withdrew: Death | 51 | 20 |
| Withdrew: Study terminated by sponsor | 26 | 9 |
| Withdrew: Withdrawal by subject | 5 | 12 |
| Withdrew: Disease progression | 1 | 0 |
| Withdrew: Physician decision: worsening of clinical condition | 0 | 1 |
| Withdrew: Transitioned to pemigatinib | 0 | 42 |
| Milestone | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Started | 0 | 42 |
| Completed | 0 | 0 |
| Not completed | 0 | 42 |
| Withdrew: Death | 0 | 27 |
| Withdrew: Lost to follow-up | 0 | 2 |
| Withdrew: Study terminated by sponsor | 0 | 11 |
| Withdrew: Withdrawal by subject | 0 | 2 |
PFS was defined as the time from the date of randomization until the date of disease progression (according to Response Evaluation Criteria in Solid Tumors version 1.1 \[RECIST v1.1\] and assessed by an Independent Central Review \[ICR\]) or death, whichever occurs first.
| months | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Randomized Treatment Period: Progression-free Survival (PFS) | 8.34 (6.51 to 12.22) | 6.80 (6.05 to 8.25) |
PFS was defined as the time from the date of randomization until the date of disease progression (according to RECIST v1.1 and assessed by an ICR) or death, whichever occurs first. Kaplan-Meier estimates indicate the percent probability of a participant being alive at the indicated time after treatment start.
| percent probability | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| 3 months | 90.0 (81.0 to 94.9) | 77.8 (66.3 to 85.7) |
| 6 months | 68.8 (57.1 to 77.9) | 64.3 (51.9 to 74.3) |
| 9 months | 44.6 (32.7 to 55.8) | 26.5 (16.0 to 38.2) |
| 12 months | 39.1 (27.3 to 50.6) | 17.1 (8.6 to 27.9) |
| 24 months | 20.9 (10.8 to 33.2) | 8.5 (2.2 to 20.2) |
| 36 months | 11.6 (3.1 to 26.4) | 8.5 (2.2 to 20.2) |
PFS was defined as the time from the date of the first dose of study drug after transition until the earliest date of progressive disease or death due to any cause.
| months | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| Transition Period: PFS | 8.08 (4.44 to 9.89) |
PFS was defined as the time from the date of the first dose of study drug after transition until the earliest date of progressive disease or death due to any cause. Kaplan-Meier estimates indicate the percent probability of a participant being alive at the indicated time after treatment start.
| percent probability | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| 3 months | 87.6 (72.6 to 94.6) |
| 6 months | 62.4 (45.6 to 75.4) |
| 9 months | 39.7 (24.2 to 54.9) |
| 12 months | 12.6 (3.6 to 27.3) |
| 24 months | 4.2 (0.3 to 17.2) |
| 36 months | 0.0 (NA to NA) |
ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) per RECIST v1.1 as assessed by an ICR. 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.
| percentage of participants | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Randomized Treatment Period: Objective Response Rate (ORR) | 47.0 (35.93 to 58.26) | 15.5 (8.51 to 25.01) |
ORR was defined as the percentage of participants with a best overall response of CR or PR per RECIST v1.1 as assessed by an ICR. 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.
| percentage of participants | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| Transition Period: ORR | 38.1 (23.57 to 54.36) |
Overall survival was defined as the time from the date of randomization until death due to any cause.
| months | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Randomized Treatment Period: Overall Survival | 24.38 (18.63 to 35.91) | 25.00 (18.66 to 34.20) |
Overall survival was defined as the time from the date of randomization until death due to any cause. Kaplan-Meier estimates indicate the percent probability of a participant being alive at the indicated time after treatment start.
| percent probability | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| 3 months | 95.2 (87.7 to 98.2) | 100.0 (100.0 to 100.0) |
| 6 months | 89.1 (80.1 to 94.2) | 97.2 (89.4 to 99.3) |
| 9 months | 81.7 (71.4 to 88.5) | 91.7 (82.4 to 96.2) |
| 12 months | 74.2 (63.3 to 82.4) | 84.7 (74.1 to 91.2) |
| 24 months | 51.0 (38.9 to 61.8) | 55.8 (43.0 to 66.7) |
| 36 months | 36.6 (24.9 to 48.3) | 34.1 (22.0 to 46.5) |
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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.
| months | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Randomized Treatment Period: Duration of Response (DOR) | 14.19 (8.74 to 24.74) | 6.31 (4.30 to NA) |
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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. Kaplan-Meier estimates indicate the percent probability of a participant still having a CR or PR at the indicated time after treatment start.
| percent probability | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| 3 months | 100.0 (100.0 to 100.0) | 100.0 (100.0 to 100.0) |
| 6 months | 80.1 (62.8 to 90.0) | 55.0 (23.2 to 78.3) |
| 9 months | 66.3 (47.1 to 79.9) | 45.8 (16.9 to 71.0) |
| 12 months | 50.4 (31.0 to 67.0) | 34.4 (9.2 to 61.9) |
| 24 months | 33.0 (14.0 to 53.6) | NA (NA to NA) |
| 36 months | 24.8 (7.7 to 46.8) | NA (NA to NA) |
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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.
| months | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| Transition Period: DOR | 6.21 (4.17 to 12.45) |
DOR was defined as the time from the date of the first assessment of CR or PR until the date of the first disease progression by an ICR per RECIST v1.1 or death, whichever occurred first. 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. Kaplan-Meier estimates indicate the percent probability of a participant still having a CR or PR at the indicated time after treatment start.
| percent probability | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| 3 months | 100.0 (100.0 to 100.0) |
| 6 months | 61.4 (33.3 to 80.5) |
| 9 months | 29.8 (8.6 to 55.0) |
| 12 months | 29.8 (8.6 to 55.0) |
| 24 months | 0.0 (NA to NA) |
| 36 months | 0.0 (NA to NA) |
DCR was defined as the percentage of participants who achieved a best overall response of CR, PR, or SD per RECIST v1.1 as assessed by an ICR. 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 | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Randomized Treatment Period: Disease Control Rate (DCR) | 89.2 (80.41 to 94.92) | 67.9 (56.78 to 77.64) |
DCR was defined as the percentage of participants who achieved a best overall response of CR, PR, or SD per RECIST v1.1 as assessed by an ICR. 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 | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| Transition Period: DCR | 83.3 (68.64 to 93.03) |
An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug until 30 days after the last dose of study drug for participants who did not transition to pemigatinib. For participants who transitioned to pemigatinib, the end date was 30 days after the last dose date of gemcitabine plus cisplatin or the day before the first dose of pemigatinib after transition, whichever occurred first.
| Participants | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | 83 | 73 |
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug until 30 days after the last dose of study drug for participants who did not transition to pemigatinib. For participants who transitioned to pemigatinib, the end date was 30 days after the last dose date of gemcitabine plus cisplatin or the day before the first dose of pemigatinib after transition, whichever occurred first.
| Participants | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Number of Participants With Any Treatment-related TEAE | 80 | 70 |
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE for the Crossover Evaluable Population was any AE either reported for the first time or for a worsened pre-existing event after the first dose of pemigatinib after switch and until 30 days after the last dose of pemigatinib.
| Participants | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| Transition Period: Number of Participants With Any TEAE | 42 |
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE for the Crossover Evaluable Population was any AE either reported for the first time or for a worsened pre-existing event after the first dose of pemigatinib after switch and until 30 days after the last dose of pemigatinib.
| Participants | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|
| Transition Period: Number of Participants With Any Treatment-related TEAE | 41 |
The EORTC QLQ-C30 is a 30-item scale composed of both multi-item scales and single-item measures. These include 5 functional scales (physical functioning, role, cognitive functioning, emotional functioning, and social functioning), 3 symptom scales (fatigue, pain, and nausea/vomiting), a global health status scale, and 6 single items (constipation, diarrhea, sleep, dyspnea, appetite, financial). All scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Therefore, a high score for a functional scale represents a high/healthy level of functioning and a high score for the global health status/QoL represents a high QoL. A high score for a symptom scale/item represents a high level of symptomatology/problems. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
| scores on a scale | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Baseline, Appetite loss | 17.1 ± 27.56 | 22.4 ± 31.46 |
| CFB at Early Termination, Appetite loss | 10.2 ± 26.71 | -3.7 ± 32.64 |
| Baseline, Constipation | 12.9 ± 18.75 | 14.9 ± 23.42 |
| CFB at Early Termination, Constipation | 11.8 ± 27.06 | 0.9 ± 30.33 |
| Baseline, Cognitive functioning | 86.4 ± 15.16 | 91.2 ± 13.15 |
| CFB at Early Termination, Cognitive functioning | -5.4 ± 18.05 | -1.9 ± 23.14 |
| Baseline, Diarrhea | 11.1 ± 22.36 | 6.5 ± 15.61 |
| CFB at Early Termination, Diarrhea | 0.5 ± 20.46 | 0.9 ± 12.56 |
| Baseline, Dyspnea | 16.9 ± 24.22 | 14.9 ± 21.15 |
| CFB at Early Termination, Dyspnea | 2.2 ± 24.05 | 8.3 ± 26.87 |
| Baseline, Emotional functioning | 75.9 ± 19.21 | 78.2 ± 20.15 |
| CFB at Early Termination, Emotional functioning | -5.6 ± 20.90 | -9.3 ± 19.60 |
| Baseline, Fatigue | 31.0 ± 25.38 | 26.2 ± 22.53 |
| CFB at Early Termination, Fatigue | 6.8 ± 20.94 | 10.6 ± 22.16 |
| Baseline, Financial difficulties | 14.0 ± 24.64 | 20.4 ± 31.76 |
| CFB at Early Termination, Financial difficulties | 8.1 ± 29.37 | 7.4 ± 26.56 |
| Baseline, Global health status/QoL | 66.7 ± 22.82 | 67.5 ± 20.00 |
| CFB at Early Termination, Global health status/QoL | -8.3 ± 20.36 | -6.0 ± 22.85 |
| Baseline, Insomnia | 30.5 ± 31.27 | 24.9 ± 28.63 |
| CFB at Early Termination, Insomnia | -6.5 ± 29.47 | -3.7 ± 23.61 |
| Baseline, Nausea and vomiting | 8.6 ± 16.27 | 11.1 ± 21.94 |
| CFB at Early Termination, Nausea and vomiting | -1.9 ± 18.13 | 1.4 ± 21.91 |
| Baseline, Pain | 24.6 ± 28.56 | 20.1 ± 23.31 |
| CFB at Early Termination, Pain | 6.6 ± 29.07 | 2.3 ± 22.24 |
| Baseline, Physical functioning | 82.3 ± 21.45 | 82.7 ± 19.21 |
| CFB at Early Termination, Physical functioning | -11.6 ± 20.36 | -5.6 ± 22.79 |
| Baseline, Role functioning | 76.7 ± 31.14 | 79.1 ± 24.51 |
| CFB at Early Termination, Role functioning | -18.0 ± 31.26 | -13.0 ± 26.16 |
| Baseline, Social functioning | 77.4 ± 26.00 | 77.4 ± 24.91 |
| CFB at Early termination, Social functioning | -12.4 ± 27.65 | -11.1 ± 21.82 |
The EORTC QLQ-BIL21 assessed QoL across 8 scales: eating, jaundice, tiredness, pain, anxiety, treatment side effects, drains, and weight loss. The raw score of each scale is the mean of the item values that contribute to the scale, and the standardized score is a linear transformation of the raw score so that the range is from 0 to 100. A high score for a symptom scale represents a high level of symptomatology/problems. The BIL21 questionnaire was only be administered to participants for whom the questionnaire is validated in that language. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
| scores on a scale | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Baseline, Anxiety | 35.9 ± 24.78 | 36.1 ± 25.83 |
| CFB at Early Termination, Anxiety | 5.7 ± 24.43 | 0.8 ± 18.50 |
| Baseline, Drains | 2.3 ± 10.14 | 2.9 ± 12.92 |
| CFB at Early Termination, Drains | 3.0 ± 17.15 | 7.8 ± 27.24 |
| Baseline, Eating | 14.9 ± 17.34 | 17.5 ± 19.44 |
| CFB at Early Termination, Eating | 12.2 ± 19.68 | 3.9 ± 21.22 |
| Baseline, Jaundice | 5.6 ± 10.40 | 5.6 ± 10.61 |
| CFB at Early Termination, Jaundice | -1.7 ± 9.99 | 1.5 ± 10.67 |
| Baseline, Pain | 22.5 ± 19.97 | 18.8 ± 18.62 |
| CFB at Early Termination, Pain | -7.6 ± 20.31 | 5.9 ± 15.30 |
| Baseline, Treatment side effects | 14.4 ± 25.33 | 13.0 ± 25.53 |
| CFB at Early Termination, Treatment side effects | 30.5 ± 36.56 | 11.5 ± 30.06 |
| Baseline, Tiredness | 34.8 ± 31.09 | 33.7 ± 32.32 |
| CFB at Early Termination, Tiredness | 6.9 ± 26.99 | 4.0 ± 28.87 |
| Baseline, Weight loss | 18.7 ± 25.26 | 21.0 ± 27.81 |
| CFB at Early Termination, Weight loss | 2.9 ± 22.76 | -9.8 ± 33.36 |
The EQ-5D (3L) is the descriptive system that comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 3 levels: no problems, some problems, extreme problems.
| Participants | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD |
|---|---|---|
| Baseline, Anxiety/Depression; no problems | 2 | 1 |
| Baseline, Anxiety/Depression; some problems | 60 | 52 |
| Baseline, Anxiety/Depression; extreme problems | 18 | 15 |
| Early Termination, Anxiety/Depression; no problems | 3 | 0 |
| Early Termination, Anxiety/Depression; some problems | 36 | 26 |
| Early Termination, Anxiety/Depression; extreme problems | 24 | 11 |
| Baseline, Mobility; no problems | 2 | 0 |
| Baseline, Mobility; some problems | 74 | 65 |
| Baseline, Mobility; extreme problems | 5 | 3 |
| Early Termination, Mobility; no problems | 1 | 1 |
| Early Termination, Mobility; some problems | 47 | 33 |
| Early Termination, Mobility; extreme problems | 15 | 3 |
| Baseline, Pain/Discomfort; no problems | 5 | 3 |
| Baseline, Pain/Discomfort; some problems | 52 | 43 |
| Baseline, Pain/Discomfort; extreme problems | 23 | 22 |
| Early Termination, Pain/Discomfort; no problems | 6 | 3 |
| Early Termination, Pain/Discomfort; some problems | 28 | 22 |
| Early Termination, Pain/Discomfort; extreme problems | 28 | 12 |
| Baseline, Self-care; no problems | 4 | 3 |
| Baseline, Self-care; some problems | 41 | 36 |
| Baseline, Self-care; extreme problems | 35 | 28 |
| Early Termination; Self-care; no problems | 2 | 0 |
| Early Termination; Self-care; some problems | 42 | 14 |
| Early Termination; Self-care; extreme problems | 19 | 23 |
| Baseline, Usual Activities; no problems | 2 | 1 |
| Baseline, Usual Activities; some problems | 33 | 29 |
| Baseline, Usual Activities; extreme problems | 46 | 38 |
| Early Termination, Usual Activities; no problems | 3 | 2 |
| Early Termination, Usual Activities; some problems | 30 | 17 |
| Early Termination, Usual Activities; extreme problems | 30 | 18 |
Collected over up to approximately 6 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Randomized Period: Pemigatinib 13.5 mg QD | 51/83 (61.4%) | 23/83 (27.7%) | 83/83 (100%) |
| Randomized Period: Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 | 47/84 (56%) | 17/73 (23.3%) | 72/73 (98.6%) |
| Transition Period: Pemigatinib 13.5 mg QD | 27/42 (64.3%) | 9/42 (21.4%) | 42/42 (100%) |
| Event | Randomized Period: Pemigatinib 13.5 mg QD | Randomized Period: Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 | Transition Period: Pemigatinib 13.5 mg QD |
|---|---|---|---|
| CholangitisHepatobiliary disorders | 1/83 | 2/73 | 2/42 |
| SepsisInfections and infestations | 3/83 | 0/73 | 1/42 |
| Platelet count decreasedInvestigations | 0/83 | 2/73 | 0/42 |
| Jaundice cholestaticHepatobiliary disorders | 2/83 | 0/73 | 0/42 |
| Abdominal painGastrointestinal disorders | 1/83 | 0/73 | 1/42 |
| AnaemiaBlood and lymphatic system disorders | 0/83 | 1/73 | 1/42 |
| AscitesGastrointestinal disorders | 0/83 | 1/73 | 1/42 |
| Back painMusculoskeletal and connective tissue disorders | 0/83 | 1/73 | 1/42 |
| MalaiseGeneral disorders | 0/83 | 1/73 | 1/42 |
| NauseaGastrointestinal disorders | 0/83 | 0/73 | 1/42 |
| Event | Randomized Period: Pemigatinib 13.5 mg QD | Randomized Period: Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 | Transition Period: Pemigatinib 13.5 mg QD |
|---|---|---|---|
| HyperphosphataemiaMetabolism and nutrition disorders | 68/83 | 2/73 | 37/42 |
| AlopeciaSkin and subcutaneous tissue disorders | 47/83 | 13/73 | 21/42 |
| AnaemiaBlood and lymphatic system disorders | 12/83 | 37/73 | 5/42 |
| NauseaGastrointestinal disorders | 17/83 | 36/73 | 6/42 |
| StomatitisGastrointestinal disorders | 38/83 | 12/73 | 20/42 |
| Palmar-plantar erythrodysaesthesia syndromeSkin and subcutaneous tissue disorders | 39/83 | 2/73 | 16/42 |
| ConstipationGastrointestinal disorders | 37/83 | 26/73 | 13/42 |
| DiarrhoeaGastrointestinal disorders | 37/83 | 12/73 | 13/42 |
| FatigueGeneral disorders | 17/83 | 31/73 | 10/42 |
| DysgeusiaNervous system disorders | 31/83 | 8/73 | 15/42 |
| Age, Customized(years) | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD | Total |
|---|---|---|---|
| Mean | 59.4 ± 13.05 | 57.2 ± 12.45 | 58.3 ± 12.76 |
| Sex: Female, Male(Participants) | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD | Total |
|---|---|---|---|
| Female | 46 | 51 | 97 |
| Male | 37 | 33 | 70 |
| Race/Ethnicity, Customized(Participants) | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD | Total |
|---|---|---|---|
| White | 60 | 57 | 117 |
| Black or African American | 5 | 3 | 8 |
| Asian | 11 | 17 | 28 |
| Not Reported | 6 | 5 | 11 |
| Captured as "Hispanic" in the Database | 1 | 0 | 1 |
| Asian - Indian | 0 | 1 | 1 |
| Egyptian | 0 | 1 | 1 |
| Race/Ethnicity, Customized(Participants) | Pemigatinib 13.5 mg QD | Gemcitabine 1000 mg/m^2 Plus Cisplatin 25 mg/m^2 to Pemigatinib 13.5 mg QD | Total |
|---|---|---|---|
| Hispanic or Latino | 4 | 3 | 7 |
| Not Hispanic or Latino | 66 | 61 | 127 |
| Not Reported | 10 | 14 | 24 |
| Unknown | 2 | 0 | 2 |
| Captured as "Other" in Database | 1 | 6 | 7 |
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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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