Phase 1 Study of Pemigatinib in Patients With Advanced Malignancies With FGF/FGFR Alterations

Study Overview
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Brief Summary
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This is a phase 1 study to investigate the characteristics of PK, PD and safety in subjects with advanced malignancies with FGF/FGFR alterations.

Official Title
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A Phase 1, Open-Label, Pharmacokinetic(PK), Pharmacodynamics(PD) and Safety Study of Pemigatinib in Patients With Advanced Malignancies With FGF/FGFR Alterations

Conditions
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Solid Tumor

Intervention / Treatment
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* Drug: Pemigatinib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Men and women, aged 18 or older. Histologically or cytologically confirmed malignancy which was considered to be surgically unresectable advanced, relapse or metastatic . Radiographically measurable disease per RECIST v 1.1 Documentation of FGF/FGFR alteration.. Documented disease progression after standard therapy ,or no standard therapy available. ECOG performance status of 0 1. Life expectancy ≥12 weeks Exclusion Criteria: Prior receipt of a selective FGFR inhibitor. History of calcium and phosphate hemostasis disorder or systemic mineral imbalance with ectopic calcification of softy tissues ( exception: skin, kidney, tendons or vessels due to injury, disease, and aging, in the absence of systemic mineral imbalance). Currently 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patients with advanced malignancies with FGF/FGFR alterations<br> | Drug: Pemigatinib<br>* Pemigatinib will be self-administered as at 13.5mg a QD oral treatment on a 2-week-on therapy and 1-week-off therapy schedule.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum observed plasma concentration (Cmax) during the dosing interval and Cmin of Pemigatinib as monotherapy |  | Day 1 to Day 16 | 
| Time to maximum plasma concentration (Tmax) of Pemigatinib as monotherapy |  | Day 1 to Day 16 | 
| Area under the single-dose plasma concentration-time curve (AUC0-t) of Pemigatinib as monotherapy |  | Day 1 to Day 16 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of pemigatinib as monotherapy as assessed by the frequency, duration, and severity of adverse events |  | From screening through 30-35 days after end of treatment, up to 6 months |