Last updated on May 2017

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


Brief description of study

The primary objective is to determine if the medication Carbamazepine, can be used as a therapy for patients with severe liver disease due to Alpha-1-Antitrypsin Deficiency .

Detailed Study Description

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.

Clinical Study Identifier: NCT01379469

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David A Rudnick, MD, PhD

Washington University in St. Louis School of Medicine
Saint Louis, MO United States
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Robert H. Squires, JR., M.D.

Children's Hospital of Pittsburgh, UPMC
Pittsburgh, PA United States
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Robert H. Squires, M.D.

University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, PA United States
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