NCT01379469

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

A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Washington University School of Medicine · Academic / Other
• Sex: All
• Age: 14 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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 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.

Conditions

• Alpha-1-antitrypsin Deficiency
• Liver Cirrhosis

Interventions

Type	Name	Description
DRUG	Drug-Carbamazepine (Tegretol XR)	To reduce the likelihood of hypersensitivity reactions the subjects will be started on 400 mg/day in 2 doses and the dose will be increased weekly by 200mg/day until reaching a stable therapeutic concentration with a dose not exceeding 1200mg/day(or 1000mg/day in subjects less than 15 years of age). The CBZ tablets will be encapsulated..
DRUG	Carbamazepine (Tegretol XR) Placebo	Carbamazepine (Tegretol XR)Placebo-the subjects will be started on 400mg/day in 2 doses and the dose will be increased weekly by 200 mg/day until reaching a dose not exceeding 1200 mg/day (or 1000 mg/day in subjects less than 15 years of age). The placebo group will receive encapsulated tables without Carbamazepine.

Timeline

• Start date: 2012-01-01
• Primary completion: 2017-02-01
• Completion: 2017-02-01
• First posted: 2011-06-23
• Last updated: 2021-10-12
• Results posted: 2021-10-12

Locations

3 sites across 1 country: United States