A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems

Part of paid clinical trials in Gainesville, Florida.

Sponsor
Takeda
Study ID
NCT06512454
Status
Recruiting
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Conditions

  • Alpha1-Antitrypsin Deficiency

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • No Intervention — OTHER
    This is a non-interventional study.

Study Details

The main aim of this study is to learn about liver problems caused by the lack of alpha-1 antitrypsin (called Alpha-1 Antitrypsin Deficiency or AATD) in adults when not treated (this is called the natural history of a condition) over 5 years. Other aims are to learn what can predict the AATD-liver condition starting and getting better or worse, describe how this condition is currently being diagnosed and watched in normal hospital care, and describe how the AATD also affects and adult's lung function. Data in this study will be collected to include medical history of a participant, including the date AATD was first identified and/or the date on which the first AATD-related liver or lung problems were diagnosed. At study start and then every year until study end, participants will be asked to completed questionnaires (called patient-reported outcomes or PRO).

Key Dates

Start date
Sep 25, 2024
Status verified
Oct 2025
Primary completion
Apr 6, 2032
Completion
Apr 6, 2032

Study Design

Enrollment
500 participants (estimated)

Arms

  • Arm: Cohort 1: AATD-Pi*ZZ Genotype/Phenotype
    Participants who have been diagnosed with Alpha-1 Antitrypsin Deficiency homozygote ZZ (AATD-Pi\*ZZ) genotype/phenotype with mild or without liver disease manifestations will be enrolled and data will be prospectively collected per routine care throughout the follow-up period.
  • Arm: Cohort 2: AATD-Pi*SZ Genotype/Phenotype
    Participants who have been diagnosed with alpha-1 antitrypsin deficiency heterozygous SZ (AATD-Pi\*SZ) genotype/phenotype with moderate-advanced or severe liver disease manifestations will be enrolled and data will be prospectively collected per routine care throughout the follow-up period.

Primary Outcome Measure

Number of Participants With Liver Disease Progression [ Time Frame: Baseline up to 5 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of FloridaGainesvilleFlorida32611
Site Contact
[email protected]
Virginia Clark, Dr (PRINCIPAL_INVESTIGATOR)

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By research site
University of Florida· Gainesville, FL

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