Clinical Trials

Numerous clinical trials evaluate pemigatinib as monotherapy or combination therapy across Phase 1, Phase 2, and Phase 3 studies targeting advanced solid tumors, cholangiocarcinoma, non-muscle invasive bladder cancer, urothelial carcinoma, endometrial cancer, and glioblastoma. Supported by industry sponsors including Incyte Corporation and Innovent Biologics alongside academic centers such as Massachusetts General Hospital, these investigations encompass actively recruiting, completed, terminated, and withdrawn protocols to establish efficacy.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07434843 RECRUITING
SDH Gene Mutation; Gastrointestinal Stromal Tumor
Dana-Farber Cancer Institute
 2026-05-19 PHASE2
NCT06439485 RECRUITING
Advanced Cholangiocarcinoma; FGFR2 Fusion
M.D. Anderson Cancer Center
 2024-11-19 PHASE1; PHASE2
NCT07780838 RECRUITING
Locally Advanced Ampulla of Vater Carcinoma; Locally Advanced Biliary Tract Carcinoma; Locally Advanced Extrahepatic Cholangiocarcinoma; Locally Advanced Gallbladder Carcinoma; Locally Advanced Intrahepatic Cholangiocarcinoma; Metastatic Ampulla of Vater Carcinoma; Metastatic Biliary Tract Carcinoma; Metastatic Extrahepatic Cholangiocarcinoma; Metastatic Gallbladder Carcinoma; Metastatic Intrahepatic Cholangiocarcinoma; Stage III Ampulla of Vater Cancer AJCC v8; Stage III Distal Bile Duct Cancer AJCC v8; Stage III Gallbladder Cancer AJCC v8; Stage III Intrahepatic Cholangiocarcinoma AJCC v8; Stage IV Ampulla of Vater Cancer AJCC v8; Stage IV Distal Bile Duct Cancer AJCC v8; Stage IV Gallbladder Cancer AJCC v8; Stage IV Intrahepatic Cholangiocarcinoma AJCC v8; Unresectable Ampulla of Vater Carcinoma; Unresectable Biliary Tract Carcinoma; Unresectable Extrahepatic Cholangiocarcinoma; Unresectable Gallbladder Carcinoma; Unresectable Intrahepatic Cholangiocarcinoma
Ohio State University Comprehensive Cancer Center
 2026-11-01 PHASE2
NCT06389799 RECRUITING
Dedifferentiated Liposarcoma
Lund University Hospital
 2024-06-20 PHASE2
NCT07786766 RECRUITING
Biliary Tract Cancer (BTC)
Haitao Zhao, MD
 2026-08-20 PHASE2
NCT06302621 RECRUITING
Advanced Solid Tumor; Unresectable Solid Tumor; Metastatic Solid Tumor; Cholangiocarcinoma
Massachusetts General Hospital
 2024-04-17 PHASE1
NCT02925234 RECRUITING
Cancer; Tumors; Neoplasm; Neoplasia
The Netherlands Cancer Institute
 2016-08 PHASE2
NCT06728410 RECRUITING
Intrahepatic Cholangiocarcinoma; FGFR2 Gene Rearrangement; FGFR2 Gene Mutation
Mehmet Akce
 2026-01-27 PHASE2
NCT06653777 RECRUITING
Solid Tumor, Miscellaneous
UNICANCER
 2025-03-27 PHASE2
NCT06906562 RECRUITING
Advanced Pancreatic Carcinoma; Metastatic Pancreatic Carcinoma; Stage II Pancreatic Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8
Sameek Roychowdhury
 2025-08-26 PHASE2
NCT04659616 RECRUITING
Acute Myeloid Leukemia
OHSU Knight Cancer Institute
 2021-01-14 PHASE1
NCT05159245 RECRUITING
Advanced Cancer; Solid Tumor; Haematological Malignancy
Helsinki University Central Hospital
 2021-12-10 PHASE2
NCT06530823 NOT_YET_RECRUITING
Biliary Tract Carcinoma; Cholangiocarcinoma
Eastern Hepatobiliary Surgery Hospital
 2024-08-01 PHASE2
NCT06551896 NOT_YET_RECRUITING
Urothelial Carcinoma; Breast Neoplasms; Lung Cancer; Gastric Cancer; Soft Tissue Sarcoma; Other Carcinoma
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
 2024-08-15 PHASE2
NCT04463771 COMPLETED
Endometrial Cancer
Incyte Corporation
 2021-01-26 PHASE2
NCT06300528 ACTIVE_NOT_RECRUITING
Relapsed or Refractory B-cell Non-Hodgkin Lymphoma; Mantle Cell Lymphoma; Marginal Zone Lymphoma
University of Utah
 2025-10-03 PHASE2
NCT03914794 COMPLETED
Bladder Cancer; NMIBC; Non-Muscle Invasive Bladder Cancer; Urothelial Carcinoma Recurrent
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2020-10-02 PHASE2
NCT03656536 TERMINATED
Unresectable Cholangiocarcinoma; Metastatic Cholangiocarcinoma
Incyte Corporation
 2019-06-03 PHASE3
NCT06022289 UNKNOWN
Solid Tumor; FGF Receptor Gene Mutation; FGF Amplification; FGF Receptor Gene Family Rearrangement; FGF Receptor Gene Translocation
Tianjin Medical University Second Hospital
 2023-05-01 PHASE2
NCT05565794 TERMINATED
Intrahepatic Cholangiocarcinoma; FGFR2 Gene Mutation; FGFR2 Gene Rearrangement; FGFR2 Gene Translocation
Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest
 2022-11-11 PHASE2
NCT05997459 UNKNOWN
Locally Advanced Unresectable Gastric Cancer
Sun Yat-sen University
 2023-08-25 PHASE2
NCT05267106 TERMINATED
Glioblastoma; Adult-type Diffuse Gliomas
Incyte Corporation
 2022-05-20 PHASE2
NCT05651672 RECRUITING
Gastrointestinal Cancer
Tianjin Medical University Cancer Institute and Hospital
 2022-12-02 PHASE2
NCT04591431 UNKNOWN
Breast Cancer; Gastrointestinal Cancer; Non Small Cell Lung Cancer; Other Cancer
Fondazione per la Medicina Personalizzata
 2020-10-07 PHASE2
NCT05913661 UNKNOWN
Carcinoma; Intrahepatic Cholangiocarcinoma; Digestive System Neoplasms; PD-1 Inhibitor; First-line Treatment
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
 2023-07 PHASE2
NCT03011372 COMPLETED
MPN (Myeloproliferative Neoplasms)
Incyte Corporation
 2017-04-25 PHASE2
NCT05560334 UNKNOWN
Breast Cancer
Tianjin Medical University Cancer Institute and Hospital
 2022-09-08 PHASE2
NCT05216120 WITHDRAWN
Pancreas Cancer
HonorHealth Research Institute
 2022-06-14 PHASE2
NCT05559775 UNKNOWN
Gastrointestinal Cancer
Zhejiang Cancer Hospital
 2022-08-10 PHASE2
NCT06302621 Recruiting
Advanced Solid Tumor|Unresectable Solid Tumor|Metastatic Solid Tumor|Cholangiocarcinoma
Massachusetts General Hospital|Boehringer Ingelheim|Incyte Corporation
 2024-07-01 Phase 1
NCT06389799 Not yet recruiting
Dedifferentiated Liposarcoma
Lund University Hospital
 2024-05-07 Phase 2
NCT04949191 TERMINATED
Advanced Malignancies
Incyte Corporation
 2021-07-08 PHASE2
NCT05287386 UNKNOWN
Advanced Non-Small Cell Lung Cancer
The First Affiliated Hospital of Xiamen University
 2022-01-21 PHASE2
NCT05210946 UNKNOWN
Non-Small Cell Lung Cancer
The First Affiliated Hospital of Soochow University
 2021-12-06
NCT04088188 TERMINATED
Advanced Cholangiocarcinoma; Metastatic Cholangiocarcinoma; Unresectable Cholangiocarcinoma
Academic and Community Cancer Research United
 2021-01-25 PHASE1
NCT05202236 UNKNOWN
Gastric and Colorectal Cancer
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
 2021-10-13 PHASE2
NCT05004974 UNKNOWN
Advanced Non Small Cell Lung Cancer
The Fourth Affiliated Hospital of Zhejiang University School of Medicine
 2022-04-26 PHASE2
NCT05253807 COMPLETED
Non-Small Cell Lung Cancer (NSCLC)
Incyte Corporation
 2022-04-29 PHASE2
NCT03498521 COMPLETED
Cancer of Unknown Primary Site
Hoffmann-La Roche
 2018-07-10 PHASE2
NCT05216120 Withdrawn
Pancreas Cancer
HonorHealth Research Institute|Incyte Corporation
 2022-06-14 Phase 2
NCT04096417 UNKNOWN
FGFR1 Gene Amplification; FGFR1 Gene Mutation; FGFR1 Gene Translocation; FGFR2 Gene Amplification; FGFR2 Gene Mutation; FGFR2 Gene Translocation; FGFR3 Gene Amplification; FGFR3 Gene Mutation; FGFR3 Gene Translocation; Metastatic Colorectal Carcinoma; Stage III Colorectal Cancer AJCC v8; Stage IIIA Colorectal Cancer AJCC v8; Stage IIIB Colorectal Cancer AJCC v8; Stage IIIC Colorectal Cancer AJCC v8; Stage IV Colorectal Cancer AJCC v8; Stage IVA Colorectal Cancer AJCC v8; Stage IVB Colorectal Cancer AJCC v8; Stage IVC Colorectal Cancer AJCC v8; Unresectable Colorectal Carcinoma
Academic and Community Cancer Research United
 2020-08-31 PHASE2
NCT03822117 TERMINATED
Solid Tumor Malignancy
Incyte Corporation
 2019-10-17 PHASE2
NCT02872714 COMPLETED
UC (Urothelial Cancer)
Incyte Corporation
 2017-01-12 PHASE2
NCT02924376 COMPLETED
Cholangiocarcinoma
Incyte Corporation
 2017-01-16 PHASE2
NCT02393248 TERMINATED
Lung Cancer; Solid Tumor; Gastric Cancer; Urothelial Cancer; Endometrial Cancer; Multiple Myeloma; Myeloproliferative Neoplasms; Breast Cancer; Cholangiocarcinoma; UC; MPN
Incyte Corporation
 2015-02-27 PHASE1; PHASE2
NCT04294277 TERMINATED
Urothelial Cancer
European Association of Urology Research Foundation
 2020-07-13 PHASE2
NCT04003610 TERMINATED
Metastatic Urothelial Carcinoma; Unresectable Urothelial Carcinoma
Incyte Corporation
 2020-05-14 PHASE2
NCT04256980 COMPLETED
Cholangiocarcinoma
Innovent Biologics (Suzhou) Co. Ltd.
 2020-03-03 PHASE2
NCT03914794 Recruiting
Bladder Cancer|NMIBC|Non-Muscle Invasive Bladder Cancer|Urothelial Carcinoma Recurrent
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins|Incyte Corporation
 2020-10-02 Phase 2
NCT04258527 COMPLETED
Solid Tumor
Innovent Biologics (Suzhou) Co. Ltd.
 2020-03-26 PHASE1
NCT04003623 TERMINATED
Advanced or Metastatic Solid Tumors; FGFR Mutations; FGFR Translocations
Incyte Corporation
 2019-10-31 PHASE2
NCT03235570 COMPLETED
Solid Tumors
Incyte Corporation
 2017-08-01 PHASE1
NCT04258527 Completed
Solid Tumor
Innovent Biologics (Suzhou) Co. Ltd.
 2020-03-26 Phase 1
NCT03235570 Completed
Solid Tumors
Incyte Corporation
 2017-08-01 Phase 1

(data from https://clinicaltrials.gov, updated on 2026-05-22)

Check the Pemigatinib (INCB054828) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Pemigatinib is a potent inhibitor that selectively targets fibroblast growth factor receptors FGFR1, FGFR2, and FGFR3 with IC50 values of 0.4 nM, 0.5 nM, and 1.2 nM, respectively, thereby blocking receptor autophosphorylation and suppressing downstream intracellular signaling cascades to inhibit tumor cell proliferation and induce apoptosis. This targeted inhibition directly suppresses cell survival in FGFR-aberrant malignancies, providing the mechanistic rationale for its clinical evaluation in FGFR-driven cancers such as cholangiocarcinoma, urothelial carcinoma, and advanced solid tumors.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.