Idiopathic ductopenia

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

Idiopathic ductopenia is a rare liver condition where the small bile ducts inside the liver gradually disappear or are severely reduced in number. The word 'idiopathic' means doctors cannot find a clear underlying cause. Bile ducts are tiny tubes that carry bile — a digestive fluid made by the liver — from the liver to the small intestine. When these ducts are missing or damaged, bile builds up inside the liver, causing a condition called cholestasis. Over time, this bile buildup can damage liver cells and lead to scarring (fibrosis) or even cirrhosis. People with idiopathic ductopenia may experience yellowing of the skin and eyes (jaundice), intense itching, fatigue, and abdominal discomfort. Some people have a mild course with stable liver function for many years, while others may progress to more serious liver disease. The condition can affect both adults and children, though it is more commonly recognized in adults. Treatment is mainly focused on managing symptoms and slowing liver damage. There is no specific cure. Medications like ursodeoxycholic acid (UDCA) are often used to help bile flow and protect liver cells. In advanced cases where the liver fails, a liver transplant may be considered. Regular monitoring by a liver specialist (hepatologist) is essential to track disease progression and adjust treatment as needed.

Also known as:

Key symptoms:

Yellowing of the skin and whites of the eyes (jaundice)Intense, persistent itching all over the bodyFatigue and low energyDark-colored urinePale or clay-colored stoolsAbdominal pain or discomfort, especially in the upper right sideNauseaPoor appetite or unintended weight lossSwelling of the abdomen due to fluid buildup (ascites) in advanced casesEasy bruising or bleeding due to poor vitamin absorption

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

5 events
Jan 2026Testing a Novel Barrier Cream for Fecal Incontinence in an Ambulatory Population

Scotiaderm — NA

TrialNOT YET RECRUITING
Aug 2025Testing the Safety and Efficacy of a Novel Barrier Cream for the Treatment of Moisture Associated Skin Damage

Scotiaderm — NA

TrialNOT YET RECRUITING
May 2025Development of a Risk Assessment Tool for Incontinence-associated Dermatitis (IAD): a Cohort Study on the Predictive Performance of Risk Factors for IAD

Örebro University, Sweden

TrialENROLLING BY INVITATION
Jan 2022Study of Dextromethorphan in OCD and Related Disorders

Stanford University — PHASE2

TrialRECRUITING
Aug 2016International Intracranial Dissection Study

Insel Gruppe AG, University Hospital Bern

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Idiopathic ductopenia.

View clinical trials →

No actively recruiting trials found for Idiopathic ductopenia at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Idiopathic ductopenia community →

No specialists are currently listed for Idiopathic ductopenia.

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 Idiopathic ductopenia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Idiopathic ductopenia

Disease timeline:

New recruiting trial: International Intracranial Dissection Study

A new clinical trial is recruiting patients for Idiopathic ductopenia

New recruiting trial: Study of Dextromethorphan in OCD and Related Disorders

A new clinical trial is recruiting patients for Idiopathic ductopenia

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.How advanced is my liver disease right now, and what does that mean for my future?,Should I have genetic testing to make sure there is not an inherited cause for my condition?,Is ursodeoxycholic acid (UDCA) the right treatment for me, and how will we know if it is working?,What symptoms should prompt me to go to the emergency room immediately?,Do I need vitamin supplements, and which ones should I take?,At what point would a liver transplant be considered, and how do I get evaluated for one?,Are there any clinical trials or research studies I could participate in?

Common questions about Idiopathic ductopenia

What is Idiopathic ductopenia?

Idiopathic ductopenia is a rare liver condition where the small bile ducts inside the liver gradually disappear or are severely reduced in number. The word 'idiopathic' means doctors cannot find a clear underlying cause. Bile ducts are tiny tubes that carry bile — a digestive fluid made by the liver — from the liver to the small intestine. When these ducts are missing or damaged, bile builds up inside the liver, causing a condition called cholestasis. Over time, this bile buildup can damage liver cells and lead to scarring (fibrosis) or even cirrhosis. People with idiopathic ductopenia may ex

How is Idiopathic ductopenia inherited?

Idiopathic ductopenia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.