Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

The primary objective is to determine if Carbamazepine therapy in patients with severe liver
disease due to Alpha-1-Antitrypsin Deficiency leads to a significant reduction in the hepatic
accumulation of ATZ.

The other objectives are:

To determine whether Carbamazepine treatment reduces hepatic fibrosis in alpha-1-antitrypsin
deficient patients with severe liver disease. To determine whether Carbamazepine treatment
reduces portal pressure in alpha-1-antitrypsin deficient patients with severe liver disease.
To determine whether Carbamazepine treatment is safe and tolerated by patients with severe
liver disease caused by alpha-1-deficiency. To determine whether Carbamazepine treatment
leads to stabilization in disease severity as measured by the MELD scores.

Inclusion Criteria:

- Age greater than or equal to 14 years to less than or equal to 80 years of age.

- Alpha-1-Antitrypsin deficiency confirmed by ZZ or SZ phenotype & serum level

- < 83mg/dl.

- HVPG greater than or equal to 10 mmHg unless collateral vessels are visualized via
transvenous biopsy.

Exclusion Criteria:

- Child Pugh Score greater than or equal to 12. Serum total bilirubin > 5 mg/dl. INR >
2.2.