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Intake of 13.5 mg pemigatinib once daily per oral

Intake of up to 3 tablets of pemigatinib (4,5 mg each) daily per oral for 14 days in a 21-day cycle (maximum of 18 cycles in total)

Pemigatinib

Thorsten Goetze, Prof. Dr.

Doerthe Vortmeyer, Dr.

Dominik Modest, Prof. Dr.

Henning Wege, Prof. Dr.

Stefan Angermeier, Dr.

Salah-Eddin Al-Batran, Prof. Dr.

The aim of this phase II study is to determine whether pemigatinib is clinically efficious after curative local therapy such as surgery/ SBRT or ablation in iCCA patients harboring FGFR2 fusion/rearrangement and to assess the safety profile to support the continuation of the concept in a large, randomized trial for further development.

This is a prospective, exploratory, single-arm, non-randomized, open-label phase II study to investigate whether pemigatinib is clinically efficacious after curative local treatment including surgery/ SBRT or ablation in iCCA patients with FGFR2 fusion/rearrangements.

Patients will receive pemigatinib 13.5 mg oral once daily (21-day cycle; two weeks on, one week off) until disease recurrence, unacceptable toxicity, withdrawal of consent, or investigator decision, but no longer than 12 months (max. 18 cycles).

The primary objective is to assess the efficacy of pemigatinib administered after curative local therapy in treatment-naïve patients with resectable intrahepatic biliary tract cancer (recurrence free survival rate at 12 months, RFS@12).

Secondary objectives are to assess the efficacy by overall survival (OS) and recurrence free survival (RFS); to assess safety of the treatment (AEs, impact on liver function, use of subsequent therapies); to assess quality of life (QoL).

In addition, tissue samples will be analyzed for biomarkers predictive for RFS and OS.

20 patients are to be enrolled in this trial.

Pemigatinib After Curative Local Therapy in Advanced iCCA With FGFR2 Fusion/Rearrangements

A Phase II Study of Pemigatinib After Curative Local Therapy in Locally Advanced Intrahepatic Cholangiocarcinoma (iCCA) Harboring FGFR2 Fusions/Rearrangements.

Objective response rate according to investigator-based RECIST 1.1 assessment, defined as the proportion of allocated subjects with best response of complete or partial response within 12 months after the date of first administration of study treatment. Patients who receive anti-cancer treatment other than the study medication for any reason before reaching a complete or partial response will be identified as nonresponders in the assessment of ORR.

Measurement of quality of life by questionaires filld in by the patients.

Safety and tolerability will by measured by evaluation of incidence, treatment relationship, seriousness, and severity of all AEs, SAEs according to CTCAE V5.0.

Spots Global Cancer Trial Database for fgfr2 gene rearrangement