Trial · NCT01379469
Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
NCT01379469, Phase 2, Terminated, Washington University School of Medicine; results first posted 2021-10-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency. Trials, record NCT01379469. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01379469
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
- NCT number
- NCT01379469
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Washington University School of Medicine
- Conditions
- Alpha-1-antitrypsin Deficiency, Liver Cirrhosis
- Interventions
- Drug-Carbamazepine (Tegretol XR), Carbamazepine (Tegretol XR) Placebo
- First posted
- 2011-06-23
- Last updated
- 2021-10-12
- Results first posted
- 2021-10-12
- Limitations and caveats
The primary \& secondary outcomes could not be assessed because the number of subjects with available pre \& post biopsies was insufficient in subject number and sample quality to conduct the analyses. As described in more detail in Outcome Measures.~.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The Primary Outcome Will be to Determine the Effect of Carbamazepine on Hepatic ATZ Load. | 52 weeks | The effect of Carbamazepine on hepatic ATZ load will be measured by the number of hepatocytes with PAS+/diastase-resistant globules and/or steady state levels of ATZ by immunoblot analysis. |
| Secondary | For the Secondary Outcomes we Will Determine the Effect of Carbamazepine Treatment on Hepatic Fibrosis. | 52 weeks | For the secondary outcomes we will determine the effect of Carbamazepine treatment on hepatic fibrosis on the basis of sirius red staining and hydroxyproline concentration and whether Carbamazepine treatment changes portal pressure as determined by Hepatic Venous Pressure Gradient. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Primary Outcome Will be to Determine the Effect of Carbamazepine on Hepatic ATZ Load. | 52 weeks | The primary outcome was not assessable because the number of subjects with available pre \& post PAS+/diastase stained liver biopsies \& tissue for immunoblot was insufficient in subject number and sample quality. To elaborate only 3 subjects had both pre \& post biopsies stained for PAS, 2 from the placebo \& 1 from the Carbamazepine arms \& the available histology quality was insufficient for histomorphormetry. Frozen liver samples were also of insufficient number \& mass for immunoblot. | ||
| SECONDARY | For the Secondary Outcomes we Will Determine the Effect of Carbamazepine Treatment on Hepatic Fibrosis. | 52 weeks | The secondary outcome was also not assessable because the number of subjects with available pre \& post trichrome stained liver biopsies \& tissue for hydroxyproline was also insufficient in subject number and sample quality. Again only 3 subjects had both pre \& post biopsies stained for trichrome, 2 placebo- \& 1 Carbamazepine treated. Available histology quality was also insufficient here for histomorphormetry \&.frozen liver samples insufficient for hydroxyproline determination. |
Source ClinicalTrials.gov
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Drug-Carbamazepine (Tegretol XR) | 13 | |
| Overall Study | STARTED | Drug-Carbamazepine (Tegretol XR) Placebo | 7 | |
| Overall Study | COMPLETED | Drug-Carbamazepine (Tegretol XR) | 1 | |
| Overall Study | COMPLETED | Drug-Carbamazepine (Tegretol XR) Placebo | 3 | |
| Overall Study | NOT COMPLETED | Drug-Carbamazepine (Tegretol XR) | 12 | |
| Overall Study | NOT COMPLETED | Drug-Carbamazepine (Tegretol XR) Placebo | 4 |
Source ClinicalTrials.gov
Adverse events50
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Cardiac | Cardiac disorders | Drug-Carbamazepine (Tegretol XR) | 1 | 13 | 2 |
| other | Cardiac | Cardiac disorders | Drug-Carbamazepine (Tegretol XR) Placebo | 3 | 7 | 4 |
| other | Child-Pugh Score Increase | Hepatobiliary disorders | Drug-Carbamazepine (Tegretol XR) | 1 | 13 | 1 |
| other | Child-Pugh Score Increase | Hepatobiliary disorders | Drug-Carbamazepine (Tegretol XR) Placebo | 1 | 7 | 2 |
| other | Constitutional | General disorders | Drug-Carbamazepine (Tegretol XR) | 12 | 13 | 63 |
| other | Constitutional | General disorders | Drug-Carbamazepine (Tegretol XR) Placebo | 7 | 7 | 17 |
| other | Dermatologic | Skin and subcutaneous tissue disorders | Drug-Carbamazepine (Tegretol XR) | 2 | 13 | 2 |
| other | Dermatologic | Skin and subcutaneous tissue disorders | Drug-Carbamazepine (Tegretol XR) Placebo | 2 | 7 | 4 |
| other | Endocrine | Endocrine disorders | Drug-Carbamazepine (Tegretol XR) | 3 | 13 | 3 |
| other | Endocrine | Endocrine disorders | Drug-Carbamazepine (Tegretol XR) Placebo | 1 | 7 | 1 |
1 to 10 of 50Source ClinicalTrials.gov
See all 50 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Drug-Carbamazepine (Tegretol XR) | 0 | 13 |
| Total, all-cause mortality | Drug-Carbamazepine (Tegretol XR) Placebo | 2 | 7 |
| Total, other adverse events | Drug-Carbamazepine (Tegretol XR) | 13 | 13 |
| Total, other adverse events | Drug-Carbamazepine (Tegretol XR) Placebo | 7 | 7 |
| Total, serious adverse events | Drug-Carbamazepine (Tegretol XR) | 6 | 13 |
| Total, serious adverse events | Drug-Carbamazepine (Tegretol XR) Placebo | 4 | 7 |
Source ClinicalTrials.gov
Papers44
The papers the trial lists on ClinicalTrials.gov.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R21DK092567 | Carbamazepine for severe liver disease due to antitrypsin deficiency | PERLMUTTER, DAVID H | UNIVERSITY OF PITTSBURGH AT PITTSBURGH | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2011 to 2012 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Drug-Carbamazepine (Tegretol XR) vs Drug-Carbamazepine (Tegretol XR) Placebo: deaths from any cause | Trial comparisons |
| Drug-Carbamazepine (Tegretol XR) vs Drug-Carbamazepine (Tegretol XR) Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01379469
GET https://conductscience.org/api/trials/v1/NCT01379469/events?page=2