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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

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

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.
  • 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.

04

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.

05

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)

06

Study locations

1 of 1 sites recruiting
  • Research Site 1
    Auckland, 1010, New Zealand
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07662096
Lead sponsor
Arrowhead Pharmaceuticals
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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