Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
Information source: University of Pittsburgh
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Alpha-1-antitrypsin Deficiency; Liver Cirrhosis
Intervention: Drug-Carbamazepine (Tegretol XR) (Drug); Carbamazepine (Tegretol XR) Placebo (Drug)
Phase: Phase 2
Status: Recruiting
Sponsored by: University of Pittsburgh Official(s) and/or principal investigator(s):
David H. Perlmutter, M.D., Principal Investigator, Affiliation: Children's Hospital of Pittsburgh,UPMC
Overall contact:
Adam D Kufen, RN, BS, CCRC, Phone: 412-692-6558, Email: adam.kufen@chp.edu
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 .
Clinical Details
Official title: A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: The primary outcome will be to determine the effect of Carbamazepine on hepatic ATZ load.
Secondary outcome: For the secondary outcomes we will determine the effect of Carbamazepine treatment on hepatic fibrosis.For the secondary outcome we will determine if Carbamazepine treatment reduces the MELD score.
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
Minimum age: 14 Years.
Maximum age: 80 Years.
Gender(s): Both.
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.
Locations and Contacts
Adam D Kufen, RN, BS, CCRC, Phone: 412-692-6558, Email: adam.kufen@chp.edu
Children's Hospital of Pittsburgh, UPMC, Pittsburgh, Pennsylvania 15201, United States; Recruiting
David H Perlmutter, M.D., Phone: 412-692-8071, Email: David.perlmutter@chp.edu
David H. Perlmutter, M.D., Principal Investigator
Robert H. Squires, JR., M.D., Sub-Investigator
Benjamin Shneider, M.D., Sub-Investigator
Alejandro Hoberman, M.D., Sub-Investigator
Ira Bergman, M.D., PhD., Sub-Investigator
Andrew Chu, MD, Sub-Investigator
University of Pittsburgh Medical Center, Presbyterian Hospital, Pittsburgh, Pennsylvania 15213, United States; Recruiting
David H. Perlmutter, M.D., Phone: 412-692-8071, Email: David.perlmutter@chp.edu
David H. Perlmutter, M.D., Principal Investigator
George Michalopoulos, M.D., PhD., Sub-Investigator
Albert Zajko, M.D., Sub-Investigator
Kapil Chopra, M.D., Sub-Investigator
Additional Information
Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
Starting date: January 2012
Last updated: December 2, 2014
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