Clinical trial NCT04256980: Pemigatinib in Treating Patients With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Including FGFR2 Rearrangement
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cholangiocarcinoma
Keywords: CCA
FGFR2
Pemigatinib
Lead sponsor: Innovent Biologics (Suzhou) Co. Ltd.
Sponsor class: INDUSTRY
First posted: 2020-02-05
Enrollment: 34
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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


Eligibility criteria: 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.

Primary outcomes:
measure: Objective response rate per RECIST 1.1
timeFrame: Every 6 weeks for the first 2 cycles and every 9 weeks thereafter through end of treatment, up to 24 months
Secondary outcomes:
measure: PFS（PFS= first dose to progressive disease or death）
timeFrame: 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
measure: DOR（DOR= time from the date of CR or PR until PD）
timeFrame: Every 6 weeks for the first 2 cycles and every 9 weeks thereafter through end of treatment, up to 24 months
measure: DCR（DCR=CR + PR + stable disease）
timeFrame: Every 6 weeks for the first 2 cycles and every 9 weeks thereafter through end of treatment, up to 24 months
measure: OS（OS= first dose to death of any cause）
timeFrame: Time from first treatment to the date of death from any cause, up to 24 months
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Zhongshan Hospital Affiliated to Fudan University
state: Shanghai
Study officials:
References: