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Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)

N/A

Waitlist Available

Led By Mark Brantly, MD

Research Sponsored by University of Florida

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at each study visit including screening, first liver biopsy, year 1, year 2, and year 3 visits.

Awards & highlights

Approved for 10 Other Conditions

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

We hypothesize that individuals with Alpha-1 Antitrypsin (AAT) deficiency have ongoing liver injury which is not detected by the usual blood tests used to look at liver function. This ongoing liver injury leads to cirrhosis in a significant number of adults with AAT deficiency.

Eligible Conditions

Alpha-1 Antitrypsin Deficiency
Liver Fibrosis

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at each study visit including screening, first liver biopsy, year 1, year 2, and year 3 visits.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at each study visit including screening, first liver biopsy, year 1, year 2, and year 3 visits.

This trial's timeline: 3 weeks for screening, Varies for treatment, and at each study visit including screening, first liver biopsy, year 1, year 2, and year 3 visits. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

To estimate the prevalence and histologic spectrum of liver injury in an adult with Alpha-1 Antitrypsin deficiency having a ZZ genotype or other rare allele.

Secondary study objectives

To define the diagnostic accuracy of non-invasive markers of fibrosis in AAT liver disease.

Fibrosis

To identify environmental and host risk factors for clinically significant liver fibrosis.

+1 more

Awards & Highlights

Approved for 10 Other Conditions

This treatment demonstrated efficacy for 10 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: AATD ZZ and Rare Alleles GroupExperimental Treatment13 Interventions

Participants will get a history and physical (H\&P) and have an intravenous catheter (IV) placed, for blood draws, at the screening and years 1-3 visits. An IV will also be placed at the liver biopsy visit(s) for the administration of medication. An abdominal ultrasound will be done at the screening and year 3 visits along with the completion of a liver questionnaire. Finally, participants will have a liver biopsy done, with the use of lidocaine, lorazepam, or midazolam and fentanyl, after the screening visit and potentially at the year 3 study visit, depending on the results of the first liver biopsy. Participants who experience pain after the liver biopsy may receive acetaminophen or oxycodone/acetaminophen. Any subject experiencing nausea may receive ondansetron.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Abdominal ultrasound

2020

Completed Phase 4

~1240

Intravenous catheter

2013

N/A

~110