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A cancer that involves the intrahepatic bile duct.
Biomarker and diagnostic research for intrahepatic bile duct cancer has been reported in the published literature.
No approved treatments are currently available for intrahepatic bile duct cancer. An additional 4 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for intrahepatic bile duct cancer, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for intrahepatic bile duct cancer. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
5 clinical trials registered, 2 recruiting. Interventions under study include drug therapy, other interventions, medical devices, and procedural interventions. Pipeline includes 2 PHASE2, 2 PHASE1, 1 NA. Research is primarily sponsored by academic and government institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT05286814](https://clinicaltrials.gov/study/NCT05286814) |
Data assembled from 4 of 12 sources · Last updated Sep 20, 2026, 5:51 AM UTC
Designated
Exclusivity End |
|---|
Designation Status |
|---|
3-bromopyruvate | 3-bromopyruvate | Cage Pharmaceuticals, Inc. | 2025 | — | Designated |
monoclonal antibody targeting CD36 | monoclonal antibody targeting CD36 | Pilatus Biosciences Inc. | 2024 | — | Designated |
pentamidine | pentamidine | Verlyx Pharma, Inc. | 2013 | — | Withdrawn |
opioid growth factor | opioid growth factor | Primocure Pharma, Inc. | 2013 | — | Designated |
Gene therapy approaches for intrahepatic bile duct cancer have been reported in the published literature.
5 trials found
PDS01ADC in Combination With Hepatic Artery Infusion Pump (HAIP) and Systemic Therapy for Subjects With Metastatic Colorectal Cancer, Intrahepatic Cholangiocarcinoma, or Metastatic Adrenocortical Carcinoma |
PHASE2 |
National Cancer Institute (NCI) |
UNKNOWN |
[NCT04491942](https://clinicaltrials.gov/study/NCT04491942) | Testing the Addition of an Anti-cancer Drug, BAY 1895344, to the Usual Chemotherapy Treatment (Cisplatin, or Cisplatin and Gemcitabine) for Advanced Solid Tumors With Emphasis on Urothelial Cancer | PHASE1 | National Cancer Institute (NCI) | ACTIVE_NOT_RECRUITING |
[NCT06888063](https://clinicaltrials.gov/study/NCT06888063) | Additional Chemotherapy Administered Directly Into the Liver Using a Chemo Pump in Patients With Bile Duct Cancer Inside the Liver Treatable by Surgery | PHASE2 | Erasmus Medical Center | RECRUITING |
[NCT05285358](https://clinicaltrials.gov/study/NCT05285358) | Pressurized Intraperitoneal Aerosolized Nab-Paclitaxel in Combination With Gemcitabine and Cisplatin for the Treatment of Biliary Tract Cancer Patients With Peritoneal Metastases | PHASE1 | City of Hope Medical Center | RECRUITING |
[NCT07573514](https://clinicaltrials.gov/study/NCT07573514) | Patients With Primary Biliary Cholangitis | NA | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | ACTIVE_NOT_RECRUITING |
185 publications have been identified in PubMed for intrahepatic bile duct cancer. Research spans Basic Science / Preclinical (36%), Review / Meta-Analysis (26%), and Epidemiology / Natural History (14%).
Research Type | Count | % of Total |
|---|---|---|
Laboratory research | 67 | 36% |
Research summaries | 48 | 26% |
Disease patterns and progression | 25 | 14% |
Testing and diagnosis research | 17 | 9% |
New treatment approaches | 12 | 6% |
Clinical study results | 8 | 4% |
Patient case studies | 6 | 3% |
Other research | 2 | 1% |
Unknown (2026). [PMID: 42127237](https://pubmed.ncbi.nlm.nih.gov/42127237/). *Unknown Journal*. [Review / Meta-Analysis]
Itano O (2026). [PMID: 41488837](https://pubmed.ncbi.nlm.nih.gov/41488837/). *Ann Gastroenterol Surg*. [Review / Meta-Analysis]
El Homsi M (2026). [PMID: 40944876](https://pubmed.ncbi.nlm.nih.gov/40944876/). *Abdom Radiol (NY)*. [Review / Meta-Analysis]
Tao J (2026). [PMID: 41817522](https://pubmed.ncbi.nlm.nih.gov/41817522/). *JAMA Oncol*. [Epidemiology / Natural History]
Li F (2026). [PMID: 40489753](https://pubmed.ncbi.nlm.nih.gov/40489753/). *Hepatology*. [Basic Science / Preclinical]
Hong L (2026). [PMID: 39999448](https://pubmed.ncbi.nlm.nih.gov/39999448/). *Hepatology*. [Basic Science / Preclinical]
Zhou T (2026). [PMID: 40324630](https://pubmed.ncbi.nlm.nih.gov/40324630/). *J Adv Res*. [Basic Science / Preclinical]
Malik S (2026). [PMID: 30725866](https://pubmed.ncbi.nlm.nih.gov/30725866/). *Unknown Journal*. [Basic Science / Preclinical]
Yasinzai AQK (2026). [PMID: 41104650](https://pubmed.ncbi.nlm.nih.gov/41104650/). *J Natl Cancer Inst*. [Epidemiology / Natural History]
Sang C (2026). [PMID: 41839437](https://pubmed.ncbi.nlm.nih.gov/41839437/). *Cancer Lett*. [Basic Science / Preclinical]