Clinical trial NCT01379469: Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
Official title: A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Alpha-1-antitrypsin Deficiency
Liver Cirrhosis
Keywords: Carbamazepine (Tegretol) use
severe liver disease
alpha-1-antitrypsin deficiency
Lead sponsor: Washington University School of Medicine
Sponsor class: OTHER
Collaborators: name: Novartis
name: National Institutes of Health (NIH)
name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
name: University of Pittsburgh
First posted: 2011-06-23
Enrollment: 20
Sex: ALL
Minimum age (years): 14
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: 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.

Primary outcomes:
measure: The Primary Outcome Will be to Determine the Effect of Carbamazepine on Hepatic ATZ Load.
timeFrame: 52 weeks
Secondary outcomes:
measure: For the Secondary Outcomes we Will Determine the Effect of Carbamazepine Treatment on Hepatic Fibrosis.
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Saint Louis
country: United States
facility: Washington University in St. Louis School of Medicine
state: Missouri
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh, UPMC
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center, Presbyterian Hospital
state: Pennsylvania
Study officials:
affiliation: Washington University School of Medicine
name: David H. Perlmutter, M.D.
role: PRINCIPAL_INVESTIGATOR
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