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

The Washington University

Status and phase: Terminated; Phase 2

Conditions: Liver Cirrhosis; Alpha-1-antitrypsin Deficiency

Treatments: Drug: Drug-Carbamazepine (Tegretol XR); Drug: Carbamazepine (Tegretol XR) Placebo

Study type: Interventional

Funder types: Other; Industry; NIH

Identifiers

NCT01379469

1R21DK092567-01 (U.S. NIH Grant/Contract)

201510060-PRO09070279

Details and patient eligibility

About

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 .

Full 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.

Enrollment: 20 patients

Sex: All

Ages: 14 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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20 participants in 2 patient groups, including a placebo group

Drug-Carbamazepine (Tegretol XR)

Active Comparator group

Description:

One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.

Treatment:

Drug: Drug-Carbamazepine (Tegretol XR)

Drug-Carbamazepine (Tegretol XR) Placebo

Placebo Comparator group

Description:

One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.

Treatment:

Drug: Carbamazepine (Tegretol XR) Placebo