Condition (MeSH)
alpha 1-Antitrypsin Deficiency
ClinicalTrials.gov lists 138 trials for alpha 1-Antitrypsin Deficiency or a narrower condition beneath it. Grifols Therapeutics LLC has the most among industry and government lead sponsors, with 12.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase (log scale)
1 more section with rows
138 of the 138 trials are tagged with alpha 1-Antitrypsin Deficiency itself; the rest are tagged with a narrower condition beneath it.
When did alpha 1-Antitrypsin Deficiency trials start? (30)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 9
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 9 |
| 2025 | 8 |
| 2024 | 9 |
| 2023 | 7 |
| 2022 | 5 |
| 2021 | 4 |
| 2020 | 4 |
| 2019 | 9 |
| 2018 | 8 |
| 2017 | 7 |
Showing 10 of 30 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 9 trials starting in 2026 and about 8 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 13 trials still running at the 2026-10-09 snapshot; registered median of 30 months across 81 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are alpha 1-Antitrypsin Deficiency trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 19 |
| Phase 1/2 | 14 |
| Phase 2 | 25 |
| Phase 2/3 | 2 |
| Phase 3 | 11 |
| Phase 4 | 9 |
| Not applicable | 12 |
| Observational | 45 |
| Expanded access | 1 |
How many alpha 1-Antitrypsin Deficiency trials are recruiting? (10)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 6 |
| Recruiting | 18 |
| Enrolling by invitation | 3 |
| Active, not recruiting | 7 |
| Completed | 66 |
Showing 5 of 10 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most alpha 1-Antitrypsin Deficiency trials? (10)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Grifols Therapeutics LLC | 12 |
| Baxalta now part of Shire | 8 |
| Arrowhead Pharmaceuticals | 5 |
| Takeda | 5 |
| Vertex Pharmaceuticals Incorporated | 5 |
Showing 5 of 10 sponsors.
Which drugs are being tested for alpha 1-Antitrypsin Deficiency? (2)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Belcesiran | 3 |
| Fazirsiran | 3 |
Interventions as named in the registry (13)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alpha1-antitrypsin | 18 |
| Alpha-1 MP | 5 |
| Liver biopsy | 2 |
| rAAV1-CB-hAAT | 2 |
| SAR447537 | 2 |
Showing 5 of 13 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do alpha 1-Antitrypsin Deficiency trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Chronic obstructive pulmonary disease | 44 | 7 |
| Pneumonia | 31 | 9 |
| Abdominal Pain | 14 | 1 |
| Lower respiratory tract infection | 9 | 2 |
| Pneumothorax | 9 | 5 |
Showing 5 of 10 adverse events.
Which publications cover alpha 1-Antitrypsin Deficiency? (161)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Alpha-1 Antitrypsin Inclusions Sequester GRP78 in a Bile Acid-Inducible Manner. | Liver international : official journal of the International Association for the Study of the Liver | 2025 | Yes |
| Characterisation of patients with Alpha-1 antitrypsin deficiency using unsupervised machine learning tools. | Respiratory medicine | 2025 | Yes |
| Efficacy and safety of the Spiration Valve System™ for the treatment of severe emphysema in patients with Alpha-1 antitrypsin deficiency (EMPROVE). | Respiratory medicine | 2024 | Yes |
| Risk of lung disease in the PI*SS genotype of alpha-1 antitrypsin: an EARCO research project. | Respiratory research | 2024 | Yes |
| Fazirsiran for Adults With Alpha-1 Antitrypsin Deficiency Liver Disease: A Phase 2 Placebo Controlled Trial (SEQUOIA). | Gastroenterology | 2024 | Yes |
Showing 5 of 161 publications.
Prevalence (18)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Alpha-1-antitrypsin deficiency | Prevalence at birth | 1-5 / 10 000 | United States | US | Unspecified |
| Alpha-1-antitrypsin deficiency | Prevalence at birth | 1-5 / 10 000 | United States | US | Exact |
| Alpha-1-antitrypsin deficiency | Prevalence at birth | 1-5 / 10 000 | United States | US | Exact |
| Alpha-1-antitrypsin deficiency | Point prevalence | 1-5 / 10 000 | Specific population | Not stated | Exact |
| Alpha-1-antitrypsin deficiency | Point prevalence | 1-5 / 10 000 | Germany | Not stated | Exact |
Showing 5 of 18 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Alpha-1-antitrypsin deficiency | 60 | Exact | Orphadata Science knowledge files, Product 1 |
| Alpha-1-antitrypsin deficiency | 60 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.