Pemigatinib in Treating Patients With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Including FGFR2 Rearrangement

A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Subjects With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Including FGFR2 Rearrangement Who Failed at Least One Previous Therapy

This is a phase 2 study to investigate the efficacy and safety of Pemigatinib in treating patients with advanced/metastatic or surgically unresectable cholangiocarcinoma with FGFR2 rearrangement who have failed at least 1 previous therapy

Study Overview

• Status: Active, not recruiting
• Conditions: Cholangiocarcinoma
• Intervention / Treatment: Drug: Pemigatinib

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Zhongshan Hospital Affiliated to Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Men and women, aged 18 or older.
2. Histologically or cytologically confirmed cholangiocarcinoma which was considered to be advanced/metastatic or surgically unresectable by the investigator through image examination.
3. Radiographically measurable disease per RECIST v 1.1
4. Documentation of FGFR2 rearrangement.
5. Documented disease progression after at least 1 line of prior systemic therapy.
6. ECOG performance status of 0~1.
7. Life expectancy ≥12 weeks.

Exclusion Criteria:

1. Prior receipt of a selective FGFR inhibitor.
2. 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).
3. Currently evidence of clinically significant corneal or retinal disorder confirmed by ophthalmologic examination.
4. 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pemigatinib in patients with advanced/metastatic or surgically; Patients with advanced/metastatic or surgically unresectable cholangiocarcinoma	Drug: Pemigatinib; Pemigatinib will be self-administered at 9mg Or 13.5mg as a QD oral treatment on a 2-week-on therapy and 1-week-off therapy schedule

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Objective response rate per RECIST 1.1	Every 6 weeks for the first 2 cycles and every 9 weeks thereafter through end of treatment, up to 24 months

Secondary Outcome Measures

Outcome Measure	Time Frame
PFS（PFS= first dose to progressive disease or death）	Time from first treatment to the date of first documented progression or date of death from any cause, whichever came first, assessed up to 24 months
DOR（DOR= time from the date of CR or PR until PD）	Every 6 weeks for the first 2 cycles and every 9 weeks thereafter through end of treatment, up to 24 months
DCR（DCR=CR + PR + stable disease）	Every 6 weeks for the first 2 cycles and every 9 weeks thereafter through end of treatment, up to 24 months
OS（OS= first dose to death of any cause）	Time from first treatment to the date of death from any cause, up to 24 months

Collaborators and Investigators

• Sponsor: Innovent Biologics (Suzhou) Co. Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): March 3, 2020
• Primary Completion (Actual): June 27, 2022
• Study Completion (Anticipated): December 30, 2023

Study Registration Dates

• First Submitted: January 20, 2020
• First Submitted That Met QC Criteria: February 4, 2020
• First Posted (Actual): February 5, 2020

Study Record Updates

• Last Update Posted (Actual): October 4, 2022
• Last Update Submitted That Met QC Criteria: October 3, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: CCA; FGFR2; Pemigatinib
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Cholangiocarcinoma
• Other Study ID Numbers: CIBI375A201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No