Can a patient registry unlock a cure for Alpha-1?
NCT ID NCT04157049
First seen Aug 19, 2026 · Last updated Aug 20, 2026 · Updated 1 time
Summary
This study creates a confidential registry of people diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and those who are carriers. The goal is to gather detailed patient data over time to help researchers understand how the condition progresses and to support the development of better treatments and a cure. Participants include individuals with specific genetic types (like PiZZ or PiMZ) who provide informed consent.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this registry could provide the data needed to develop improved treatments and, ultimately, a cure for Alpha-1 Antitrypsin Deficiency.
- What could go wrong
- As a registry, it does not test a treatment directly, so any benefits depend on future research using its data. Participation involves sharing personal health information, though confidentiality is maintained.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
About 5,000 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Jun 2019
- Expected to finish
-
Jun 2029
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
The current protocol seeks to enroll 4,000 individuals of all age, race, and sex.
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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)
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Alpha 1 antitrypsin deficiency are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Alpha-1 Foundation
RECRUITINGCoral Gables, Florida, 33134, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- AI model could predict who needs a lung transplant for rare emphysema
- Could a simple shot replace IV drips for Alpha-1 patients?
- Hidden liver harm: study tracks silent damage in genetic disorder
- Promising liver drug trial halted early: what it means for patients
- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition