A Phase 1 interventional study of ARO-AAT Injection and Sterile Normal Saline (0.9% NaCl) in Alpha 1-Antitrypsin Deficiency, sponsored by Arrowhead Pharmaceuticals. Completed at 1 site in New Zealand. Open to participants aged 18 Years to 55 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-12-24.
Sponsored by Arrowhead Pharmaceuticals · Phase 1, Interventional, and Treatment
Among 16 studies in alpha 1-Antitrypsin Deficiency with a site in New Zealand — 4 open to participants now, 5 under way, 1 of them phase 3. alpha 1-Antitrypsin Deficiency · New Zealand.
Among the 16 studies in alpha 1-Antitrypsin Deficiency with a site in New Zealand, this one is the 8th largest by enrollment, is the only one that started in 2018, is one of 5 in phase 1, and was the 5th registered.
142 studies on the registry are indexed under alpha 1-Antitrypsin Deficiency; 32 are open to participants now.
Browse alpha 1-Antitrypsin Deficiency studies →4 of the 16 studies in alpha 1-Antitrypsin Deficiency with a site in New Zealand are open now. Recruiting first, the newest first:
AIRNA Corporation · Sites in New Zealand, Australia, Georgia and 6 more countries
Ages 18–74
Wave Life Sciences USA, Inc. · Sites in New Zealand, Australia, Canada and 3 more countries
Ages 18–70
Beam Therapeutics Inc. · Sites in New Zealand, United States, Australia and 3 more countries
Ages 18–75
Grifols Therapeutics LLC · Sites in New Zealand, United States, Australia and 9 more countries
Ages 20–72
A study can be open while its sites in New Zealand have not opened yet or have stopped. Only those recruiting at a site in New Zealand →
Of the 23 alpha 1-Antitrypsin Deficiency studies recruiting now, 11 (48%) set no upper age limit and 9 (39%) stop at 75 or younger; 4 take children under 18; 5 take healthy volunteers.
This study: 18 Years to 55 Years · all sexes · healthy volunteers too.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
16 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 9 · Phase 2 8 · Phase 3 2 · Phase 4 1
First in 2005; this study’s year is marked. 1 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 45 is above the median of 33 across 86 interventional studies indexed under alpha 1-Antitrypsin Deficiency.
Median enrollment 44 across the 16 interventional studies. Of the 9 that completed or were terminated, 3 (33%) have results posted.
This tab as a report to print or save as PDF → With an account.
16 of 16 have an industry lead sponsor. The 10 most frequent:
Arrowhead Pharmaceuticals is the lead sponsor of 50 studies on the registry; 9 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 12 (50%) have results posted.
Arrowhead Pharmaceuticals has 5 studies filed under alpha 1-Antitrypsin Deficiency — fewer than ten, so there is no comparison to draw.
1 of the studies in alpha 1-Antitrypsin Deficiency with a site in New Zealand that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06405633Wave Life Sciences USA, Inc.A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD | 2027-09 |
Add it to your calendar (.ics) → With an account.
Among the 16 studies in alpha 1-Antitrypsin Deficiency with a site in New Zealand, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
NOTE: additional inclusion/exclusion criteria may apply, per protocol
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: ARO-AAT Injection
Other: Sterile Normal Saline (0.9% NaCl)
Single or multiple doses of ARO-AAT by subcutaneous (sc) injections
Calculated volume to match active comparator
Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment
Time frame: Part A (single-ascending dose [SAD] phase): up to 29 (+/- 2) days post-dose; Part B (multiple-ascending dose [MAD] phase): up to 113 (+/- 2) days post-dose
Pharmacokinetics (PK) of ARO-AAT: Maximum Observed Plasma Concentration (Cmax)
Time frame: Part A (single-ascending dose [SAD] phase): up to 48 hours post-dose; Part B (multiple-ascending dose [MAD] phase): up to 48 hours post-dose
PK of ARO-AAT: Time to Maximum Plasma Concentration (Tmax)
Time frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose
PK of ARO-AAT: Terminal Elimination Half-Life (t½)
Time frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose
PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)
Time frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose
PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf)
Time frame: Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose
Percent Change in Serum Alpha-1 Antitrypsin (AAT) Levels From Day 1 Pre-Dose Baseline to Nadir
Time frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days
Duration of Response of Serum AAT levels From Nadir Back to Above 20% of Baseline or Above 90 mg/dL
Time frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days
Plan to share: No
No publications or documents are linked to this record.
Ran from Mar 2018 to primary completion in Oct 2018, 7 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Fazirsiran
alpha 1-Antitrypsin Deficiency →