Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
NCT ID: NCT01379469
Last Updated: 2021-10-12
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
20 participants
INTERVENTIONAL
2012-01-31
2017-02-28
Brief Summary
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Detailed Description
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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
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Drug-Carbamazepine (Tegretol XR)
One arm receives Drug-Carbamazepine (Tegretol XR).All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.
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
One arm receives Carbamazepine (Tegretol-XR) placebo.All subjects have severe liver disease due to alpha-1-antitrypsin deficiency.
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.
Interventions
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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..
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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
14 Years
80 Years
ALL
No
Sponsors
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Novartis
INDUSTRY
National Institutes of Health (NIH)
NIH
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
University of Pittsburgh
OTHER
Washington University School of Medicine
OTHER
Responsible Party
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Principal Investigators
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David H. Perlmutter, M.D.
Role: PRINCIPAL_INVESTIGATOR
Washington University School of Medicine
Locations
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Washington University in St. Louis School of Medicine
St Louis, Missouri, United States
Children's Hospital of Pittsburgh, UPMC
Pittsburgh, Pennsylvania, United States
University of Pittsburgh Medical Center, Presbyterian Hospital
Pittsburgh, Pennsylvania, United States
Countries
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References
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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201510060-PRO09070279
Identifier Type: -
Identifier Source: org_study_id
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