Understanding the progression of Noncirrhotic Portal Hypertension

Natural History of Noncirrhotic Portal Hypertension

This study is trying to learn more about how Noncirrhotic Portal Hypertension develops and changes over time by looking at the health of up to 400 people aged 12 and older who have or are at risk for this condition.

Quick facts

• Study type: Observational
• Enrollment: 400 (estimated)
• Ages: 12 Years to 100 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (NIH)
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT02417740 on ClinicalTrials.gov

What this trial studies

This observational study aims to investigate the natural history of Noncirrhotic Portal Hypertension (NCPH) by enrolling up to 400 participants aged 12 and older who have NCPH or are at risk for developing it. Participants will undergo a series of assessments including medical history, physical exams, imaging studies, and blood tests to gather data on the disease's progression and biological mechanisms. The study seeks to identify novel biomarkers and establish guidelines for monitoring and managing NCPH effectively.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 12 and older diagnosed with NCPH or at risk due to underlying conditions.

Not a fit: Patients with other liver diseases that typically result in cirrhosis or active hepatitis infections may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved monitoring and management strategies for patients with Noncirrhotic Portal Hypertension.

How similar studies have performed: While studies on liver diseases exist, this specific focus on NCPH and its natural history is relatively novel and has not been extensively tested.

Eligibility criteria

* INCLUSION CRITERIA:

In order to be eligible to participate in this study, an individual must meet all of the following criteria:

* Male or female, aged \>= 18 years of age, and minors 12-17 years of age.
* Women of childbearing potential must agree to use birth control unless they are menopausal or had hysterectomy.
* Known diagnosis of NCPH, or to be at the risk for NCPH by virtue of underlying disease processes such as but not limited to; CGD, SCD, Mastocytosis, CVID, CF, and CHF.

EXCLUSION CRITERIA:

An individual who meets any of the following criteria will be excluded from participation in this study:

* Pregnancy.
* Evidence of other forms of liver disease that typically result in cirrhosis.
* Evidence of active Chronic Hepatitis B infection as defined by the presence of hepatitis B surface antigen (HBsAg) in serum and elevated HBV DNA (\>10,000 IU/mL).
* Hepatitis C as defined by the presence of hepatitis C RNA in serum.
* Evidence of other liver disease such as primary sclerosing cholangitis, primary biliary cirrhosis, Wilson s disease, autoimmune hepatitis as defined by either liver histology or laboratory abnormalities.
* Hemochromatosis as defined by presence of 3+ or 4+ stainable iron on liver biopsy or homozygosity for C282Y. Patients with iron saturation indices of \>45% and serum ferritin levels of \>300 ng/ml for men and \>250 ng/ml for women will undergo genetic testing for hemochromatosis.
* Bile duct obstruction as suggested by imaging studies done within the previous six months.
* The presence of cirrhosis confirmed by liver biopsy.
* Active substance abuse, such as alcohol, inhaled or injection drugs within the previous one year (assessed during subject interviews by subject self-report).
* Evidence of hepatocellular carcinoma; either alpha-fetoprotein (AFP) levels greater than 50 ng/ml (normal \<6.6ng/ml) and/or ultrasound (or other imaging study) demonstrating a mass suggestive of liver cancer.
* Evidence of cholangiocarcinoma as suggested by liver histology.
* Any other severe condition, which in the opinion of the investigators would impede the patient s participation or compliance in the study.
* Inability to comply or give written informed consent.

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: Theo Heller, M.D. — National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Study coordinator: Jaha F Norman-Wheeler

Conditions Cystic Fibrosis; Immunologic Deficiency Syndrome; Turner Syndrome; Congenital Hepatic Fibrosis; Idiopathic Non-Cirrhotic Portal Hypertension; Portal Hypertension; Portal Fibrosis; Liver Disease