Alpha-1 Research Registry

Alpha-1 Foundation

Status: Enrolling

Conditions: Alpha 1-Antitrypsin Deficiency

Study type: Observational

Funder types: Other

Identifiers

NCT04157049

Alpha-1 Registry Protocol

Details and patient eligibility

About

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.

Full description

The purpose and goal of the Alpha-1 Research Registry (Registry, or group of patients) will be to obtain uniform, longitudinal (over the course of time), complete and accurate data that can be organized, and made available for the public to query. The collective number of Registry members enables investigators to enroll sufficient subjects to carry out their studies. The community benefits from having more research and potential therapies performed in their disease. Regular updates from patients will give objective data-points to measure the progression of disease.

Enrollment: 4,000 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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

Trial design

4,000 participants in 2 patient groups

Alpha-1 Diagnosed Individuals

Description:

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

Description:

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.