ClinicalTrials.gg
RecruitingNCT07662096Updated Sep 15, 2026

A First-In-Human Study of ARO-033 in Adult Participants

A Phase 1 interventional study of ARO-033 and Placebo in Age-related Macular Degeneration, sponsored by Arrowhead Pharmaceuticals. Recruiting at 1 site in New Zealand. Open to participants aged 18 Years to 65 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-09-15.

Sponsored by Arrowhead Pharmaceuticals · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started Jun 2026; still recruiting 4 months later.
Phase
Phase 1
Study type
Interventional
Enrollment
84
Allocation
Randomized
Ages
18 Years to 65 Years
Sex
All
01

Study summary

This study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-033 compared to placebo in adult normal healthy volunteers (NHVs) and the safety, tolerability, PK, PD, and efficacy of multiple doses of ARO-033 in participants with age-related macular degeneration (AMD).

02

Conditions studied

  • Age-related Macular Degeneration
03

In context

Macular Degeneration

1,459 studies on the registry are indexed under Macular Degeneration; 238 are open to participants now.

This study's planned enrollment of 84 is above the median of 56 across 985 interventional studies indexed under Macular Degeneration.

Browse Macular Degeneration studies →

Lead sponsor

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.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

Eligibility criteria

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.
  • (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:

  • (HIV) infection, as shown by the presence of anti-HIV antibody (seropositive).
  • Seropositive for virus (HBV) (hepatitis B surface antigen positive at screening) or 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 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 agents.
  • History of major surgery within 90 days of Screening.
  • Use of an 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.

Terms in these criteria, in plain language(7)

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.

BMI (body mass index)

Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.

What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

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.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

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.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
84 participants (estimated)

Study arms

  • Experimental
    NHV Cohorts: ARO-033

    Participants will receive either a single dose of ARO-033 on Day 1 (single-ascending dose \[SAD\]) or 2 doses (multiple-ascending dose \[MAD\]) of ARO-033 administered on Day 1 and Day 29.

    Drug: ARO-033

  • Placebo comparator
    NHV Cohorts: Placebo

    Participants will receive either a single dose of placebo on Day 1 (SAD) or 2 doses (MAD) of placebo administered on Day 1 and Day 29.

    Drug: Placebo

  • Experimental
    AMD Cohorts: ARO-033

    Participants will receive up to 5 doses of ARO-033.

    Drug: ARO-033

  • Placebo comparator
    AMD Cohorts: Placebo

    Participants will receive up to 5 doses of placebo.

    Drug: Placebo

Interventions

  • DrugARO-033

    ARO-033 will be administered as a subcutaneous (SC) injection per schedule specified in the arm description.

  • DrugPlacebo

    Placebo matching to ARO-033 will be administered as SC injection per schedule specified in the arm description.

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Up to Day 337

Secondary outcomes

  1. Maximum Observed Plasma Concentration (Cmax) of ARO-033

    Time frame: SAD: Predose (0 hour) up to 48 hours postdose (Day 1 up to Day 3); MAD: Predose up to 18 hours postdose (Days 1 and 29), 24 hours postdose (Days 2 and 30), and 48 hours postdose (Days 3 and 31); AMD cohorts: Up to 4 hours postdose (Days 1 and 29)

  2. Area Under the Plasma Concentration-time Curve From Time 0 to the Last Quantifiable Plasma Concentration (AUC0-t) of ARO-033

    Time frame: SAD: Predose (0 hour) up to 48 hours postdose (Day 1 up to Day 3); MAD: Predose up to 18 hours postdose (Days 1 and 29), 24 hours postdose (Days 2 and 30), and 48 hours postdose (Days 3 and 31); AMD cohorts: Up to 4 hours postdose (Days 1 and 29)

  3. NHV Cohorts Only: Amount of ARO-033 Excreted in the Urine From Time 0 to 24 Hours After Dosing (Ae)

    Time frame: SAD: Predose (0 hour) up to 8 hours postdose (Day 1), and up to 24 hours postdose (Day 2); MAD: Predose (0 hour) up to 8 hours postdose (Days 1 and 29)

07

Study locations

1 of 1 sites recruiting
  • Research Site 1
    Auckland, 1010, New Zealand
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Jun 2026 (actual). Primary completion is estimated for Jul 2028, 24 months after the start.

Of 60 industry-sponsored phase 1 interventional studies under Macular Degeneration, half reached primary completion within 15 months of starting (the median), counting those still running; a quarter within 9 and three quarters within 27. This study's planned 24 months is above that.

Counting every sponsor, half of 75 studies reached it within 18 months; a quarter within 10 and three quarters within 30.

How these figures are worked out, and which studies are left out of them.

Registered
Jun 18, 2026
First posted
Jun 23, 2026
StartActual
Jun 25, 2026
Primary completionEst.
Jul 1, 2028
CompletionEst.
Jul 1, 2028
Last update
Sep 15, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Sep 15, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07662096
Responsible party
Sponsor
First posted
Jun 23, 2026
Start date
Jun 25, 2026
Primary completion
Jul 1, 2028 (estimated)
Completion
Jul 1, 2028 (estimated)
Last update
Sep 15, 2026

Study contacts

Medical Monitor
Contact
ARO033-1001@arrowheadpharma.com
626-304-3400

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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