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Efficacy and Safety of Pemigatinib in Subjects With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Who Failed Previous Therapy - (FIGHT-202)

Phase 2
Completed
Conditions
Cholangiocarcinoma
Interventions
Registration Number
NCT02924376
Lead Sponsor
Incyte Corporation
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
147
Inclusion Criteria
  • Histologically or cytologically confirmed cholangiocarcinoma.
  • Radiographically measurable or evaluable disease per RECIST v1.1.
  • Tumor assessment for FGF/FGFR gene alteration status.
  • Documented disease progression after at least 1 line of prior systemic therapy.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
  • Life expectancy ≥ 12 weeks.
Exclusion Criteria
  • Prior receipt of a selective FGFR inhibitor.
  • History of and/or current evidence of ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, myocardia, or lung, excepting calcified lymph nodes and asymptomatic arterial or cartilage/tendon calcifications.
  • Current evidence of clinically significant corneal or retinal disorder confirmed by ophthalmologic examination.
  • Use of any potent CYP3A4 inhibitors or inducers within 14 days or 5 half-lives, whichever is shorter, before the first dose of study drug. Topical ketoconazole will be allowed.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Cohort A PemigatinibPemigatinibPemigatinib in subjects with FGFR2 translocation with a documented fusion partner in central laboratory report
Cohort B PemigatinibPemigatinibPemigatinibin subjects with other FGF/FGFR alterations
Cohort C PemigatinibPemigatinibPemigatinib in subjects negative for FGF/FGFR alteration
Primary Outcome Measures
NameTimeMethod
Objective Response Rate (ORR) in Participants With FGFR2 Rearrangements or Fusionsup to 1527 days

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.

Secondary Outcome Measures
NameTimeMethod
ORR in All Participants With FGF/FGFR Alterationsup to 1527 days

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.

ORR in Participants Negative for FGF/FGFR Alterationsup to 143 days

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.

Number of Participants With Any Treatment-emergent Adverse Event (TEAE)up to 1584 days

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.

Overall Survivalup to 51.32 months

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.

Vc/F of PemigatinibPredose; 1-2 hours post-dose; 4-12 hours post-dose

Vc/F is defined as the apparent volume of distribution for the central compartment of pemigatinib.

Vp/F of PemigatinibPredose; 1-2 hours post-dose; 4-12 hours post-dose

Vp/F is defined as the apparent volume of distribution for the tissue (peripheral) compartment.

Progression-free Survival (PFS)up to 50.17 months

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.

Disease Control Rate (DCR)up to 1527 days

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.

First-order Absorption Rate Constant (ka) of PemigatinibPredose; 1-2 hours post-dose; 4-12 hours post-dose

First-order absorption rate constant is defined as the rate at which a drug enters into the system.

CL/F of PemigatinibPredose; 1-2 hours post-dose; 4-12 hours post-dose

CL/F is defined as apparent oral clearance.

ORR in Participants FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusionsup to 424 days

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.

Duration of Response (DOR)up to 47.11 months

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.

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