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First human doses of experimental drug ARO-033 begin safety testing

NCT ID NCT07662096

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 16, 2026 · Updated 4 times

Summary

This study is the first time the experimental drug ARO-033 is being given to humans. Its main goal is to check the safety and tolerability of single and multiple doses in healthy adult volunteers. Researchers will also measure how the drug moves through the body. This is an early step to see if ARO-033 is safe enough for future studies in people with specific diseases.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ARO-033
What this could lead to
If safe and well-tolerated, this could pave the way for future studies of ARO-033 in specific diseases.
What could go wrong
This is a very early, small study in healthy people, not patients. It only tests safety and drug levels, not whether the drug works for any condition. Side effects are possible.

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

About 84 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Jul 2028

An estimate. End dates often move.

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 65 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.

Key Inclusion Criteria: * NHV cohorts only: Adults 18 to 65 years of age who are not pregnant, not breastfeeding, and do not plan to become pregnant (or impregnate their partners) during the study and for at least 90 days following the end of the study. * AMD cohorts only: Adults ≥50 years of age who are not pregnant, not breastfeeding, and do not plan to become pregnant (or impregnate their partners) during the study and for at least 90 days following the end of the study. * Body mass index (BMI) between 18.0 and 35.0 kilograms (kg)/square meter (m\^2), inclusive. * No abnormal finding of clinical relevance at the Screening evaluation that in the opinion of the Investigator could adversely impact participant's safety during the study or adversely impact study results. * AMD cohorts only: A clinical diagnosis of AMD in both eyes as determined by the Investigator and confirmed by the central reading center. Key Exclusion Criteria: All Cohorts: * Human immunodeficiency virus (HIV) infection, as shown by the presence of anti-HIV antibody (seropositive). * Seropositive for hepatitis B virus (HBV) (hepatitis B surface antigen positive at screening) or hepatitis C virus (HCV) (HCV antibody positive with reflex confirmation using HCV RNA amplification at Screening). Cured HCV (positive antibody test without detectable HCV RNA) is permitted if HCV RNA has been negative for at least 2 years. * Uncontrolled hypertension (resting systolic blood pressure ≥160 millimeters of mercury \[mmHg\] or diastolic blood pressure ≥95 mmHg, confirmed by repeat measurement, at Screening). * Evidence of clinically significant immunocompromising condition (for example, primary immunodeficiency syndrome, aplastic anemia, known or suspected complement factor deficiency, or any other condition resulting in significantly impaired immune response as evidenced by recurrent infections), or recent/ongoing treatment with immunosuppressive agents. * History of major surgery within 90 days of Screening. * Use of an investigational agent or device within 30 days or 5 half-lives (whichever is longer) prior to dosing or current participation in an investigational study. Participants recently participating in studies involving investigational agents with prolonged therapeutic effect (such as ribonucleic acid interference \[RNAi\] therapeutics, cell or gene therapies) and whose last dose was less than 12 months prior to the first dose of study intervention, should be discussed with the Medical Monitor. * Any medical condition or clinically significant laboratory abnormality at Screening that in the opinion of the Investigator should exclude the participant from participation, preclude safe and successful completion of the study, or confound study results. AMD Cohorts Only: * Any ocular condition in the study eye that may be expected to progress and that may affect central vision or otherwise be a confounding factor during the study, as determined by the Investigator. * Atrophic retinal disease due to causes other than AMD (for example, drug-induced, Stargardt disease, cone rod dystrophy) in either eye. Note: Other protocol-defined inclusion and exclusion criteria may apply.

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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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Research Site 1

    RECRUITING

    Auckland, 1010, New Zealand

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