Registry for individuals with Alpha-1 Antitrypsin Deficiency
Alpha-1 Research Registry Protocol
This study is creating a database to gather information from people with Alpha-1 Antitrypsin Deficiency and carriers to help researchers find better treatments and understand the disease better.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 4000 (estimated) |
| Sex | All |
| Sponsor | Alpha-1 Foundation Academic / other |
| Locations | 1 site (Coral Gables, Florida) |
| Trial ID | NCT04157049 on ClinicalTrials.gov |
What this trial studies
The Alpha-1 Research Registry is a confidential database that collects data from individuals diagnosed with Alpha-1 Antitrypsin Deficiency and those identified as carriers. This registry aims to gather uniform and longitudinal data to support research initiatives and enhance the development of treatments and potential cures for Alpha-1. By enrolling a significant number of participants, the registry facilitates the ability of investigators to conduct studies and measure disease progression over time.
Who should consider this trial
Good fit: Ideal candidates for this registry include individuals diagnosed with Alpha-1 Antitrypsin Deficiency and Alpha-1 carriers.
Not a fit: Patients who are normal healthy individuals without Alpha-1 Antitrypsin Deficiency or carriers will not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could lead to improved treatments and a potential cure for Alpha-1 Antitrypsin Deficiency.
How similar studies have performed: Other studies utilizing patient registries for rare diseases have shown success in advancing research and treatment options.
Eligibility criteria
Show full inclusion / exclusion criteria
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)
Where this trial is running
Coral Gables, Florida
- Alpha-1 Foundation — Coral Gables, Florida, United States (Recruiting)
Study contacts
- Study coordinator: Alison Keaveny, MBBS
- Email: akeaveny@alpha1.org
- Phone: 1-877-228-7321
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.