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

• Register: ClinicalTrials.gov
• Last refreshed on: 18 October 2021
• Main ID: NCT01379469
• Date of registration: 15/06/2011
• Prospective Registration: Yes
• Primary sponsor: Washington University School of Medicine
• Public title: Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency CBZ
• Scientific title: A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
• Date of first enrolment: January 2012
• Target sample size: 20
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01379469
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: David H. Perlmutter, M.D.
• Affiliation: Washington University School of Medicine

Key inclusion & exclusion 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.



Age minimum: 14 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Liver Cirrhosis

Alpha-1-antitrypsin Deficiency

Intervention(s)

Drug: Carbamazepine (Tegretol XR) Placebo

Drug: Drug-Carbamazepine (Tegretol XR)

Primary Outcome(s)

The Primary Outcome Will be to Determine the Effect of Carbamazepine on Hepatic ATZ Load. [Time Frame: 52 weeks]

Secondary Outcome(s)

For the Secondary Outcomes we Will Determine the Effect of Carbamazepine Treatment on Hepatic Fibrosis. [Time Frame: 52 weeks]

Secondary ID(s)

201510060-PRO09070279

1R21DK092567-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

University of Pittsburgh

National Institutes of Health (NIH)

Novartis

Results

• Results available: Yes
• Date Posted: 12/10/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01379469