Clinical Trial: Natural History of Noncirrhotic Portal Hypertension

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Natural History of Noncirrhotic Portal Hypertension

Brief Summary:

Background:

- Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start slowly and not have many warning signs. Many people may not even know that they have a liver disease. There are no specific treatments for NCPH.

Objectives:

- To learn more about how NCPH develops over time.

Eligibility:

- People age 12 and older who have NCPH or are at risk for getting it. In the past year, they cannot have had other types of liver disease that typically result in cirrhosis, liver cancer, or active substance abuse.

Design:

• Participants will have 2 screening visits.
• Visit 1: to see if they have or may develop NCPH.
• Medical history
• Physical exam
• Urine and stool studies
• Abdominal ultrasound
• Fibroscan. Sound waves measure liver stiffness.

<TAB>- Visit 2:

• Blood tests
• Abdominal MRI
• Echocardiogram
• Questionnaire
• Liver blood vessel pressure (hepatic venous portal gradient (HVPG)) measurement. This is done with a small tube inserted in a neck vein.
• They may have a
  

  Detailed Summary: Noncirrhotic Portal Hypertension (NCPH) includes a spectrum of chronic liver diseases characterized by increased pressure within the portal circulation in the absence of cirrhosis. The complications from NCPH are similar to that of cirrhosis induced portal hypertension which includes the development of gastrointestinal varices, portal hypertensive gastropathy, splenomegaly, sepsis and ascites. However, unlike cirrhosis related portal hypertension, NCPH is characterized by well-preserved hepatic synthetic function. With increasing recognition both of patients with noncirrhotic portal hypertensive liver diseases, and mortality due to NCPH, it is clear that the specific mechanism(s) and the natural history(s) of noncirrhotic portal hypertensive liver disease have yet to be elucidated and described. At the Clinical Center of the NIH, various cohorts of patients have been identified to be at increased risk for the development of noncirrhotic portal hypertensive liver diseases such as Cystic Fibrosis (CF), common variable immunodeficiency (CVID), Turner s Syndrome (TS) and congenital hepatic fibrosis (CHF) to name a few. We propose to study individuals with NCPH, and those at risk of developing NCPH within these and other cohorts of patients known to be at risk for NCPH for an indefinite period of time. Through continued evaluation and scientific discovery, our aim is to provide a greater understanding of noncirrhotic portal hypertensive liver diseases and the different underlying biological processes that lead to the development of NCPH. We also aim to further the scant existing knowledge regarding the natural history of this disease and the global phenomenon of portal hypertension. From the data obtained from this natural history protocol, future studies will be planned to evaluate specific hypothesis in specific disease cohorts. Patients 12 years of age and older with diseases known to cause cirrhosis will be excluded. Individuals thought to be at risk for the developm
  Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  

  Current Primary Outcome: To study the natural history of non cirrhotic portal hypertension. [ Time Frame: Ongoing ]
  
  

  Original Primary Outcome: Same as current
  
  

  Current Secondary Outcome:
  
  

  Original Secondary Outcome:
  
  Information By: National Institutes of Health Clinical Center (CC)
  

  Dates:
  Date Received: April 15, 2015
  Date Started: March 30, 2015
  Date Completion: December 31, 2050
  Last Updated: April 21, 2017
  Last Verified: January 4, 2017