Neoplasms

All Conditions

Digestive System Diseases

Rare Diseases

Cholangiocarcinoma

Liver Neoplasms

Adenocarcinoma

Carcinoma

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Bile Duct Cancer

Biliary Tract Cancer

Intrahepatic Cholangiocarcinoma

Hematinics

All Drugs and Chemicals

Liver Extracts

derazantinib

Manuel Häckl, MD

Marc Engelhardt, MD

Basilea is providing expanded access to derazantinib for patients with locally advanced, inoperable or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR genomic alterations on a patient by patient basis while clinical development of derazantinib is ongoing.

Expanded Access Use of Derazantinib for Advanced Intrahepatic Cholangiocarcinoma (iCCA) With FGFR Genomic Alterations

Expanded Access Use of Derazantinib for Patients With Locally Advanced, Inoperable or Metastatic iCCA Harbouring FGFR Genomic Alterations

Basilea Pharmaceutica

Crouzon Syndrome

Jackson-Weiss Syndrome

Pfeiffer Syndrome

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.

Incyte Biosciences International Sàrl

Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest

Biliary Tract Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Biliary Tract Diseases

Gastrointestinal Diseases

Antineoplastic Agents

Immunomodulating Agents

Durvalumab

Tremelimumab

Antineoplastic Agents, Immunological

Durvalumab in combination with Tremelimumab

Arndt Vogel, Professor

Doerthe Vortmeyer, Doctor

Gunnar Folprecht

Ken Herrmann

Petra Buechner-Steudel

Arndt Vogel

Udo Lindig

Martin Fuchs

Jens Ricke

Salah-Eddin Al-Batran, Professor

A multicenter Phase II, randomized, prospective, open-label Trial investigating the clinical impact on combining Specific Internal Radiotherapy (SIRT) with the PD1-L Inhibitor Durvalumab and the CTLA-4 Inhibitor Tremelimumab in patients with intrahepatic Biliary Tract Cancer

IMMUWHY Phase II Clinical Trial will test the Addition of the immunotherapeutic agents Durvalumab and Tremelimumab after an initial Standard of Care SIRT in patients suffering from non-resectable intrahepatic Biliary Tract Cancer. Patients will be randomized into two experimental arms, one receiving Durvalumab only, the other one receiving Durvalumab + Tremelimumab. Clinical Outcomes will be compared vs. historical datasets.

Immunotherapy Combined With Y-90 SIRT Therapy in Advanced Stage Intrahepatic Biliary Tract Cancer (BTC)

Phase II Study of Immunotherapy With Durvalumab (MEDI4736) or Durvalumab and Tremelimumab, Both Combined With Y-90 SIRT Therapy in Advanced Stage Intrahepatic Biliary Tract Cancer (BTC)

Exploratory: Predictive biomarkers that with a potential effect on Objective Response Rate, Duration of Response, Progression Free Survival and Overall Survival via blood sample collection + genetic subset analysis. The following translational research is currently planned, but may be adapted taking into account new research data: Tumors will be tested for mRNA expression of PD-1, PD-L1 and PD-L2 expression, Immune cell infiltrates (IGHM, CD3, CD8, FOXP3, CD68, CD205), Chemokines (CXCL9. CXCL10, CXCL13) and invasion markers (MMP7, MMP9)

Proportion of allocated subjects with best response of complete or partial response

Type, incidence and severity of AEs and SAEs graded according to NCI CTCAE v5.0

Time from that the measurement criteria are first met for CR or PR (whichever is first recorded) until the first date that the PD is objectively documented or death

Time from the date of randomization to the date of first observed disease progression (investigator assessment according to RECIST 1.1) or death from any cause

time from the date of treatment allocation to the date of death.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Expanded Access Use of Derazantinib for Advanced Intrahepatic Cholangiocarcinoma (iCCA) With FGFR Genomic Alterations	NCT04087876	Intrahepatic Ch...	derazantinib	18 Years -	Basilea Pharmaceutica
Pemigatinib After Curative Local Therapy in Advanced iCCA With FGFR2 Fusion/Rearrangements	NCT05565794	Intrahepatic Ch... FGFR2 Gene Muta... FGFR2 Gene Rear... FGFR2 Gene Tran...	Pemigatinib	18 Years -	Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest
Immunotherapy Combined With Y-90 SIRT Therapy in Advanced Stage Intrahepatic Biliary Tract Cancer (BTC)	NCT04238637	Intrahepatic Ch...	Durvalumab Tremelimumab	18 Years - 99 Years	Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest

Spots Global Cancer Trial Database for icca

Every month we try and update this database with for icca cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for icca

Every month we try and update this database with for icca cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for icca cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.