RECRUITING

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

Conditions

Alpha1-Antitrypsin Deficiency Drug Therapy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

Prospective Observational Study on the Natural History of Alpha-1 Antitrypsin Deficiency and Associated Liver Disease

Quick Facts

Study Start:2024-09-25

Study Completion:2032-04-06

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06512454

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Willing to provide written informed consent or currently enrolled in an ongoing participating AATD patient registry that does not require reconsenting to participate in the study. 2. \>=18 years of age at enrollment in this study. 3. Participants with documented diagnosis of AATD, meeting the following criteria: 1. Cohort 1 (AATD-Pi\ZZ genotype/phenotype). 2. Cohort 2 (AATD-Pi\SZ genotype/phenotype with liver disease manifestation). * Pi\SZ genotype as documented from rapid genetic assay, sequencing, or PCR, or Pi\SZ phenotype as documented from IEF electrophoresis, and * Moderate-advanced or severe liver disease manifestation as defined by either liver biopsy or surrogate laboratory measures, as determined through: * Lab and imaging measures to define liver disease manifestation	1. Documented AATD genotype/phenotype other than Pi\ZZ or Pi\SZ. 2. History of liver transplant. 3. No results for either biopsies, magnetic resonance elastography (MRE), fibro scan (vibration controlled transient elastography \[VCTE\]), or Aspartate aminotransferase to platelet ratio index (APRI) in the 24 months prior to the study baseline date, and has none of these tests ordered in the current visit that will be available in the next 90 days. 4. Participants who had previously been treated or in an active participation in an interventional trial studying liver or lung disease. 5. Treatment with liver directed AATD investigational therapy as part of a compassionate use request.

Inclusion Criteria

Exclusion Criteria

1. Willing to provide written informed consent or currently enrolled in an ongoing participating AATD patient registry that does not require reconsenting to participate in the study.
2. \>=18 years of age at enrollment in this study.
3. Participants with documented diagnosis of AATD, meeting the following criteria:
1. Cohort 1 (AATD-Pi\*ZZ genotype/phenotype).
2. Cohort 2 (AATD-Pi\*SZ genotype/phenotype with liver disease manifestation).
* Pi\*SZ genotype as documented from rapid genetic assay, sequencing, or PCR, or Pi\*SZ phenotype as documented from IEF electrophoresis, and
* Moderate-advanced or severe liver disease manifestation as defined by either liver biopsy or surrogate laboratory measures, as determined through:
* Lab and imaging measures to define liver disease manifestation

1. Documented AATD genotype/phenotype other than Pi\*ZZ or Pi\*SZ.
2. History of liver transplant.
3. No results for either biopsies, magnetic resonance elastography (MRE), fibro scan (vibration controlled transient elastography \[VCTE\]), or Aspartate aminotransferase to platelet ratio index (APRI) in the 24 months prior to the study baseline date, and has none of these tests ordered in the current visit that will be available in the next 90 days.
4. Participants who had previously been treated or in an active participation in an interventional trial studying liver or lung disease.
5. Treatment with liver directed AATD investigational therapy as part of a compassionate use request.

Contacts and Locations

Study Contact

Takeda Contact

CONTACT

+1-877-825-3327

medinfoUS@takeda.com

Principal Investigator

Study Director

STUDY_DIRECTOR

Takeda

Study Locations (Sites)

University of Florida

Gainesville, Florida, 32611

United States

Collaborators and Investigators

Sponsor: Takeda

Study Director, STUDY_DIRECTOR, Takeda

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-09-25

Study Completion Date2032-04-06

Study Record Updates

Study Start Date2024-09-25

Study Completion Date2032-04-06

Terms related to this study

Keywords Provided by Researchers

Drug Therapy

Additional Relevant MeSH Terms

Alpha1-Antitrypsin Deficiency