Cholangiocarcinoma
Conditions
Keywords
CCA, FGFR2, Pemigatinib
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
Interventions
DRUGPemigatinib
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
Sponsors
Innovent Biologics (Suzhou) Co. Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| 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
| 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 |
Countries
China
Outcome results
None listed