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
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).
Key Inclusion Criteria:
Key Exclusion Criteria:
All Cohorts:
AMD Cohorts Only:
Note: Other protocol-defined inclusion and exclusion criteria may apply.
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
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
Participants will receive up to 5 doses of ARO-033.
Drug: ARO-033
Participants will receive up to 5 doses of placebo.
Drug: Placebo
ARO-033 will be administered as a subcutaneous (SC) injection per schedule specified in the arm description.
Placebo matching to ARO-033 will be administered as SC injection per schedule specified in the arm description.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to Day 337
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)
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)
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)
Plan to share: No
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet 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.
Arrowhead Pharmaceuticals