Age-related Macular DegenerationPhase 1NCT07662096
A First-In-Human Study of ARO-033 in Adult Participants
A Phase 1 study testing ARO-033 in people with Age-related Macular Degeneration, run by Arrowhead Pharmaceuticals at 1 site in New Zealand.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Age-related Macular Degeneration
- What is being tested?
- ARO-033
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 84 participants
- Where
- 1 siteNew Zealand
- Study dates
- 25 Jun 2026to 1 Jul 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 12Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A First-In-Human Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of ARO-033 in Adult Participants
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).
- Conditions
- Age-related Macular Degeneration
- Being tested
- ARO-033, Placebo
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARO-033Drug
ARO-033 will be administered as a subcutaneous (SC) injection per schedule specified in the arm description.
PlaceboDrug
Placebo matching to ARO-033 will be administered as SC injection per schedule specified in the arm description.
| Study group | Role | What they receive |
|---|---|---|
| NHV Cohorts: ARO-033 | Experimental | 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. Receives: Drug: ARO-033 |
| NHV Cohorts: Placebo | Placebo comparator | 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. Receives: Drug: Placebo |
| AMD Cohorts: ARO-033 | Experimental | Participants will receive up to 5 doses of ARO-033. Receives: Drug: ARO-033 |
| AMD Cohorts: Placebo | Placebo comparator | Participants will receive up to 5 doses of placebo. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Measured: Up to Day 337
Secondary outcomes (3)
Maximum Observed Plasma Concentration (Cmax) of ARO-033
Measured: 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
Measured: 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)
Measured: 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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2026
- Study start25 Jun 2026
- Primary completion1 Jul 2028 (estimated)
- Study completion1 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Age-related Macular DegenerationStudies of the same condition, open and under way.
- More trials run by Arrowhead PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 84 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arrowhead Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07662096
- Sponsor's protocol ID
- ARO033-1001
- Status last verified
- Sep 2026
- First submitted
- 18 Jun 2026
- Last updated
- 15 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No