Intractable diarrhea of infancy

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ORPHA:73014
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1Active trials1Specialists8Treatment centers

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

Intractable diarrhea of infancy (IDI) is a rare and serious condition in which babies develop severe, persistent diarrhea that does not respond to standard treatments. The diarrhea typically begins in the first few months of life and can be life-threatening if not managed quickly. Unlike ordinary diarrhea caused by infections, IDI is caused by problems with how the intestinal lining develops or functions, meaning the gut simply cannot absorb nutrients and fluids properly. IDI is actually an umbrella term that covers several distinct underlying conditions. These include microvillus inclusion disease (also called Davidson disease), tufting enteropathy (also called intestinal epithelial dysplasia), and syndromic diarrhea (also called tricho-hepato-enteric syndrome or Phenotypic diarrhea of infancy). Each of these has a different genetic cause, but they all share the hallmark of severe, watery diarrhea starting in early infancy. Babies with IDI typically cannot absorb enough nutrition through their gut and require intravenous feeding (called total parenteral nutrition or TPN) to survive. Key symptoms include large volumes of watery diarrhea, poor weight gain, dehydration, and malnutrition. Some forms also affect the liver, immune system, or physical appearance. Treatment is largely supportive, and intestinal transplantation may be considered in severe cases. Long-term outcomes depend on the specific underlying cause.

Also known as:

Key symptoms:

Severe, watery diarrhea that does not stop with standard treatmentsPoor weight gain or weight loss in a young babyDehydration (dry mouth, sunken eyes, no tears, reduced wet diapers)Swollen bellyMalnutrition and failure to thriveVomitingAbnormal hair texture or sparse, brittle hair (in syndromic forms)Liver problems such as jaundice or enlarged liver (in syndromic forms)Immune system problems leading to frequent infections (in some forms)Facial features that look different from other family members (in syndromic forms)Low blood protein levelsElectrolyte imbalances (low sodium, potassium, or other minerals in the blood)

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Apr 2026Design, Development and Evaluation of a Digital Health Assistant for Paediatric Asthma

University Children's Hospital Basel

TrialNOT YET RECRUITING
May 2025Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)

Napo Pharmaceuticals, Inc. — PHASE2

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Intractable diarrhea of infancy.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Phase 21 trial
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Phase 2
Actively Recruiting
PI: Lissette Jimenez, MD, MPH (Boston Children's Hospital) · Sites: Boston, Massachusetts; Rome +1 more · Age: 017 yrs

Specialists

1 foundView all specialists →
LM
Lissette Jimenez, MD, MPH
Specialist
PI on 1 active trial

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 Intractable diarrhea of infancy.

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Community

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Latest news about Intractable diarrhea of infancy

Disease timeline:

New recruiting trial: Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)

A new clinical trial is recruiting patients for Intractable diarrhea of infancy

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.Which specific subtype of intractable diarrhea of infancy does my child have, and what gene is involved?,What is the long-term plan for nutrition — will my child always need TPN, or is there a chance they can eat by mouth?,What are the signs of a central line infection, and what should I do if I suspect one?,Is intestinal transplantation an option for my child, and when would you consider it?,Are there any clinical trials or new treatments we should know about?,What specialists should be part of my child's care team, and how often should we see each one?,What support services are available for our family, including home nursing, social work, and mental health support?

Common questions about Intractable diarrhea of infancy

What is Intractable diarrhea of infancy?

Intractable diarrhea of infancy (IDI) is a rare and serious condition in which babies develop severe, persistent diarrhea that does not respond to standard treatments. The diarrhea typically begins in the first few months of life and can be life-threatening if not managed quickly. Unlike ordinary diarrhea caused by infections, IDI is caused by problems with how the intestinal lining develops or functions, meaning the gut simply cannot absorb nutrients and fluids properly. IDI is actually an umbrella term that covers several distinct underlying conditions. These include microvillus inclusion d

How is Intractable diarrhea of infancy inherited?

Intractable diarrhea of infancy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Intractable diarrhea of infancy typically begin?

Typical onset of Intractable diarrhea of infancy is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Intractable diarrhea of infancy?

Yes — 1 recruiting clinical trial is currently listed for Intractable diarrhea of infancy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Intractable diarrhea of infancy?

1 specialists and care centers treating Intractable diarrhea of infancy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.