Alpha-1 Research Registry

NCT04157049 | Recruiting

• 1 other identifier: Alpha-1 Registry Protocol
• Study Type: observational
• Target: 4,000
• Locations: 1 country
• Sites: 1

Brief Summary

The Alpha-1 Research Registry is a confidential database made up of individuals diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and individuals identified as Alpha-1 carriers. The Registry was established to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1.

Conditions

Alpha 1-Antitrypsin Deficiency

Keywords

AATD; ALPHA 1; COPD; AAT; ZZ; MZ; CARRIER; MM; MS; PiZZ

Outcome Measures

Primary Outcomes (1)

• Establish the Alpha-1 Research Registry using REDCap

  To gather accurate patient data for longitudinal prospective follow up/analysis of Alpha-1 progression.

  2 years

Study Arms (2)

Alpha-1 Diagnosed Individuals

Larger patient cohorts are needed to support the clinical trials coming in the next 3-5 years. Despite widespread invitations to the Alpha-1 community from the Alpha-1 Foundation Research Registry, it is estimated that the Alpha-1 Foundation Research Registry now contains \<40% of the identified PiZZ individuals in the US.

Carriers of Alpha-1

Larger patient cohorts are needed to support the clinical trials coming in the next 3-5 years. Despite widespread invitations to the Alpha-1 community from the Alpha-1 Foundation Research Registry, it is estimated that the Alpha-1 Foundation Research Registry now contains \<40% of the identified PiZZ individuals in the US.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The current protocol seeks to enroll 4,000 individuals of all age, race, and sex.

You may qualify if:

• Patients diagnosed with Alpha-1 Antitrypsin Deficiency (PiZZ, PiZNull, PiSZ etc.)
• Alpha-1 carriers (PiMZ, PiMS etc.)

You may not qualify if:

• Failure to provide informed consent
• Normal healthy individuals (MM)

Sponsors & Collaborators

• Alpha-1 Foundation: lead

Study Sites (1)

Alpha-1 Foundation

Coral Gables, Florida, 33134, United States

RECRUITING

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency; Pulmonary Disease, Chronic Obstructive

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Lung Diseases, Obstructive; Chronic Disease; Disease Attributes

Central Study Contacts

Alison Keaveny, MBBS

CONTACT

1-877-228-7321; akeaveny@alpha1.org

Randel Plant

CONTACT

1-877-228-7321; rplant@alpha1.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2019
• First Posted: November 8, 2019
• Study Start: June 20, 2019
• Primary Completion (Estimated): June 20, 2029
• Study Completion (Estimated): June 20, 2029
• Last Updated: January 30, 2024

Record last verified: 2024-01

Locations

US (1)