Alpha 1-Antitrypsin DeficiencyNCT04157049
Alpha-1 Research Registry
An observational study of Alpha 1-Antitrypsin Deficiency, run by Alpha-1 Foundation at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- How many people
- 5,000 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Jun 2019to 20 Jun 2029
- How long will I take part?
- 3 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The current protocol seeks to enroll 4,000 individuals of all age, race, and sex.
Who may be able to join
- Patients diagnosed with Alpha-1 Antitrypsin Deficiency (PiZZ, PiZNull, PiSZ etc.)
- Alpha-1 carriers (PiMZ, PiMS etc.)
Who cannot join
- Failure to provide informed consent
- Normal healthy individuals (MM)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Alpha-1 Foundation
Coral Gables, Florida, 33134
Alison Keaveny, MBBS, 1-877-228-7321 ext. 252, akeaveny@alpha1.org
Nadine Nuchovich, MPH, 1-877-228-7321 ext. 245, nnuchovich@alpha1.org
About this study
Official title: Alpha-1 Research Registry Protocol
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.
Read the detailed 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.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Keywords
- AATD, ALPHA 1, COPD, AAT, ZZ, MZ, CARRIER, MM, MS, PiZZ
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Pulmonary Disease, Chronic Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Establish the Alpha-1 Research Registry using REDCap
To gather accurate patient data for longitudinal prospective follow up/analysis of Alpha-1 progression.
Measured: 8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2019
- Study start20 Jun 2019
- Primary completion20 Jun 2029 (estimated)
- Study completion20 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 5,000 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Alpha-1 Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeanine D'Armiento, MD, PhD, Principal investigator, Alpha-1 Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04157049
- Sponsor's protocol ID
- Alpha-1 Registry Protocol
- Status last verified
- Aug 2026
- First submitted
- 6 Nov 2019
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Alpha-1 Research Registry Protocol
- ClinicalTrials.gov ID
- NCT04157049
- Acronym / sponsor ID
- Alpha-1 Registry Protocol
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Keywords
- AATD, ALPHA 1, COPD, AAT, ZZ, MZ, CARRIER, MM
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- Alpha-1 FoundationUniversity, hospital or other
- Study start
- 20 Jun 2019 (actual)
- Primary completion
- 20 Jun 2029 (estimated)
- Study completion
- 20 Jun 2029 (estimated)
- First posted
- 8 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The current protocol seeks to enroll 4,000 individuals of all age, race, and sex.
Key inclusion criteria (2 listed)
- Patients diagnosed with Alpha-1 Antitrypsin Deficiency (PiZZ, PiZNull, PiSZ etc.)
- Alpha-1 carriers (PiMZ, PiMS etc.)
Key exclusion criteria (2 listed)
- Failure to provide informed consent
- Normal healthy individuals (MM)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Establish the Alpha-1 Research Registry using REDCap
Time frame: 8 years
To gather accurate patient data for longitudinal prospective follow up/analysis of Alpha-1 progression.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jeanine D'Armiento, MD, PhD principal investigator
Alpha-1 Foundation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alpha 1-Antitrypsin Deficiency, run by Alpha-1 Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alison Keaveny, MBBS (1-877-228-7321 ext. 252, akeaveny@alpha1.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Establish the Alpha-1 Research Registry using REDCap (time frame: 8 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2026.