Cure My Disease

Don't miss your cure!

 Log in / Sign up

Healthy volunteers help test new Alpha-1 drug safety

NCT ID NCT03362242

Knowledge-focused Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tested the safety and how the body processes a new drug called ARO-AAT in 45 healthy adults. The goal was to see if the drug is safe and to measure its levels in the blood. This research is a first step toward a possible treatment for Alpha-1 Antitrypsin Deficiency, a genetic condition that can harm the lungs and liver.

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.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

45 people

The number who actually took part.

Started

Mar 2018

Finished

Mar 2020

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 55 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show 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: * Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception * Willing to provide written informed consent and to comply with study requirements * Non-smoker for at least one year * Normal lung function * No abnormal finding of clinical relevance at Screening * Normal AAT level at Screening visit Exclusion Criteria: * Clinically significant health concerns * Regular use of alcohol within one month prior to Screening * Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study * Recent use of illicit drugs * Use of any drugs or dietary/herbal supplements know to interfere with liver metabolism NOTE: additional inclusion/exclusion criteria may apply, per protocol

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.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alpha 1 antitrypsin deficiency

Contacts and locations

Locations

  • Research Site 1

    Grafton, Auckland, 1010, New Zealand

More trials for these conditions

Other studies related to the condition(s) this trial covers.