Rare malignant epithelial tumor of liver and intrahepatic biliary tract

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1FDA treatments19Active trials8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Rare malignant epithelial tumors of the liver and intrahepatic biliary tract are a group of uncommon cancers that start in the cells lining the liver or the bile ducts located inside the liver. This broad category includes cancers such as intrahepatic cholangiocarcinoma (bile duct cancer inside the liver), combined hepatocellular-cholangiocarcinoma, and other rare epithelial cancers that do not fit neatly into the more common liver cancer types. These tumors are different from the most common liver cancer, hepatocellular carcinoma, though they share the same organ. These cancers affect the liver's ability to filter blood, produce bile, and carry out hundreds of chemical processes the body depends on. As the tumor grows, it can block bile flow, damage liver tissue, and spread to nearby lymph nodes or other organs. Common symptoms include yellowing of the skin and eyes (jaundice), abdominal pain, unexplained weight loss, fatigue, and changes in stool or urine color. Treatment depends on the specific tumor type, its size, and whether it has spread. Options may include surgery to remove the tumor, liver transplantation in select cases, chemotherapy, targeted therapy, immunotherapy, and radiation. Because these tumors are rare and often diagnosed at a late stage, treatment can be challenging. Research into new therapies, including targeted drugs based on genetic mutations in the tumor, is ongoing and offering new hope to patients.

Also known as:

Key symptoms:

Yellowing of the skin and whites of the eyes (jaundice)Pain or discomfort in the upper right side of the abdomenUnexplained weight lossPersistent fatigue and weaknessLoss of appetiteDark-colored urinePale or greasy stoolsItchy skinFever or night sweatsNausea or vomitingSwelling of the abdomen due to fluid buildup (ascites)A lump or mass felt in the abdomen

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Dec 2025Outpatient Versus Inpatient Care Pathway for Intra-arterial Treatment of Primary Liver Cancer (CHOC)

University Hospital, Angers — NA

TrialRECRUITING
Dec 2025Trifluridine/Tipiracil + Oxaliplatin in Participants With Advanced or Metastatic Biliary Tract Cancer

Case Comprehensive Cancer Center — PHASE2

TrialRECRUITING
Oct 2025Testing Ivonescimab Versus FOLFOX in Advanced Biliary Tract Cancer Patients

UNICANCER — PHASE2

TrialRECRUITING
Sep 2025Impact of Comprehensive Geriatric Management on Morbidity and Quality of Life in Elderly Patients Undergoing Major Hepatectomy and Pancreaticoduodenectomy for Cancer

Assistance Publique - Hôpitaux de Paris — PHASE2, PHASE3

TrialNOT YET RECRUITING
Sep 2025ctDNA in Genetic Profiling and Clinical Outcomes of Advanced Biliary Tract Cancer

CHA University

TrialRECRUITING
Sep 2025Role of ctDNA in Genetic Profiling & Outcomes for Advanced BTC

CHA University

TrialRECRUITING
Aug 2025A Single-arm, Multicenter, Exploratory Study of Adebrelimab Combined With Apatinib and Systemic Chemotherapy for Initially Unresectable Biliary Tract Cancer

Yongjun Chen — PHASE2

TrialRECRUITING
Jul 2025A Post-marketing Observational Study of Tasfygo in Participants With Unresectable Biliary Tract Cancer With Fibroblast Growth Factor Receptor 2 (FGFR2) Fusion Gene Positivity Who Progressed After Chemotherapy

Eisai Co., Ltd.

TrialRECRUITING
Jan 2025HX009+ IN10018 with or Without Standard Chemotherapy for Advanced Solid Tumours

Hangzhou Hanx Biopharmaceuticals, Ltd. — PHASE1, PHASE2

TrialRECRUITING
Nov 2024Circulating Tumor Mitochondrial DNA (ct-mtDNA) As a Biomarker for Biliary Tract Cancer Recurrence Surveillance

Tongji Hospital

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

Dyural 80-Lm

METHYLPREDNISOLONE ACETATE, LIDOCAINE HYDROCHLORIDE, BUPIVACAINE HYDROCHLORIDE, POVIDINE IODINE, ISOPROPYL ALCOHOL· Advanced Rx Pharmacy of Tennessee, LLC■ Boxed Warning

indicated in adults for the production of local or regional anesthesia or analgesia for diagnostic and therapeutic procedures

Clinical Trials

19 recruitingView all trials with filters →
Phase 33 trials
Durvalumab With Chemotherapy as First Line Treatment in Patients With Advanced Biliary Tract Cancers (aBTCs)
Phase 3
Active
· Sites: Mobile, Alabama; Orange, California +32 more · Age: 18130 yrs
Trastuzumab Plus Chemotherapy vs Chemotherapy Alone in First-line HER2 Positive Advanced Biliary Tract Cancer Patients
Phase 3
Actively Recruiting
PI: Vikas Ostwal, DM (Tata Memorial Hospital, Parel, Mumbai, Maharashtra) · Sites: Muzaffarpur, Bihar; Mumbai, Maharashtra +4 more · Age: 1899 yrs
Rilvegostomig + Chemotherapy as Adjuvant Therapy for Biliary Tract Cancer After Resection (ARTEMIDE-Biliary01)
Phase 3
Active
· Sites: Birmingham, Alabama; Phoenix, Arizona +175 more · Age: 1899 yrs
Phase 25 trials
Testing Ivonescimab Versus FOLFOX in Advanced Biliary Tract Cancer Patients
Phase 2
Actively Recruiting
PI: Julien Edeline, MD (Centre Eugène Marquis) · Sites: Paris; Rennes +1 more · Age: 1899 yrs
A Single-arm, Multicenter, Exploratory Study of Adebrelimab Combined With Apatinib and Systemic Chemotherapy for Initially Unresectable Biliary Tract Cancer
Phase 2
Actively Recruiting
· Sites: Wuhan, Hubei · Age: 1880 yrs
DEB-TACE Combined With Apatinib and PD-1 for the Treatment of Intrahepatic Cholangiocarcinoma
Phase 2
Actively Recruiting
· Sites: Chengdu, Sichuan · Age: 1880 yrs
Trifluridine/Tipiracil + Oxaliplatin in Participants With Advanced or Metastatic Biliary Tract Cancer
Phase 2
Actively Recruiting
PI: Madison Conces, MD (Case Comprehensive Cancer Center, University Hospi) · Sites: Cleveland, Ohio; Cleveland, Ohio · Age: 1999 yrs
Phase II Study of the Combination of Durvalumab (MEDI4736) (PDL1 Inhibitor) and Olaparib (PARP Inhibitor) in Advanced Cholangiocarcinoma After Initial Chemotherapy and Durvalumab (BIL-PPP)
Phase 2
Actively Recruiting
· Sites: Camperdown, New South Wales; Clayton, New South Wales +6 more · Age: 1899 yrs
Phase 12 trials
Study of LY3410738 Administered to Patients With Advanced Solid Tumors With IDH1 or IDH2 Mutations
Phase 1
Active
PI: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) (Eli Lilly and Company) · Sites: Phoenix, Arizona; Tucson, Arizona +31 more · Age: 1899 yrs
Prospective Exploratory Study of FAPi PET/CT With Histopathology Validation in Patients With Various Cancers
Phase 1
Active
PI: Jeremie Calais (UCLA / Jonsson Comprehensive Cancer Center) · Sites: Los Angeles, California · Age: 1899 yrs
N/A2 trials
Senior Adult Hepatobiliary Prehab Study
N/A
Active
PI: Mohammed Al-Jumayli, MD (Moffitt Cancer Center) · Sites: Tampa, Florida · Age: 6599 yrs
Outpatient Versus Inpatient Care Pathway for Intra-arterial Treatment of Primary Liver Cancer (CHOC)
N/A
Actively Recruiting
PI: Christophe AUBE, Pr (University Hospital, Angers) · Sites: Amiens; Angers +12 more · Age: 1899 yrs
Other4 trials
Circulating Tumor Mitochondrial DNA (ct-mtDNA) As a Biomarker for Biliary Tract Cancer Recurrence Surveillance
Actively Recruiting
PI: Ze-yang Ding, M.D. (Tongji Hospital) · Sites: Wuhan, Hubei · Age: 1899 yrs
Role of ctDNA in Genetic Profiling & Outcomes for Advanced BTC
Actively Recruiting
PI: Hong Jae Chon, MD. PhD (Principal Investigator) · Sites: Seongnam-si, Gyeonggi-do · Age: 1999 yrs
ctDNA in Genetic Profiling and Clinical Outcomes of Advanced Biliary Tract Cancer
Actively Recruiting
PI: Hong Jae Chon (Principal Investigator) · Sites: Seongnam-si, Gyeonggi-do · Age: 1999 yrs
A Post-marketing Observational Study of Tasfygo in Participants With Unresectable Biliary Tract Cancer With Fibroblast Growth Factor Receptor 2 (FGFR2) Fusion Gene Positivity Who Progressed After Chemotherapy
Actively Recruiting
· Sites: Tokyo

No specialists are currently listed for Rare malignant epithelial tumor of liver and intrahepatic biliary tract.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Rare malignant epithelial tumor of liver and intrahepatic biliary tract.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What is the exact type of tumor I have, and has it spread beyond the liver?,Has my tumor been tested for genetic mutations like FGFR2, IDH1, or others that could guide treatment?,Am I a candidate for surgery, and if not, what are my treatment options?,Are there any clinical trials I might be eligible for?,What are the expected side effects of the recommended treatment, and how will they be managed?,Should I or my family members be tested for hereditary cancer syndromes?,What support services — such as nutrition counseling, palliative care, or mental health support — are available to me?

Common questions about Rare malignant epithelial tumor of liver and intrahepatic biliary tract

What is Rare malignant epithelial tumor of liver and intrahepatic biliary tract?

Rare malignant epithelial tumors of the liver and intrahepatic biliary tract are a group of uncommon cancers that start in the cells lining the liver or the bile ducts located inside the liver. This broad category includes cancers such as intrahepatic cholangiocarcinoma (bile duct cancer inside the liver), combined hepatocellular-cholangiocarcinoma, and other rare epithelial cancers that do not fit neatly into the more common liver cancer types. These tumors are different from the most common liver cancer, hepatocellular carcinoma, though they share the same organ. These cancers affect the li

How is Rare malignant epithelial tumor of liver and intrahepatic biliary tract inherited?

Rare malignant epithelial tumor of liver and intrahepatic biliary tract follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Rare malignant epithelial tumor of liver and intrahepatic biliary tract typically begin?

Typical onset of Rare malignant epithelial tumor of liver and intrahepatic biliary tract is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Rare malignant epithelial tumor of liver and intrahepatic biliary tract?

Yes — 19 recruiting clinical trials are currently listed for Rare malignant epithelial tumor of liver and intrahepatic biliary tract on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.