Early safety study of ARO-033 in healthy adults and people with macular degeneration
Tests ARO-033 against placebo in healthy adults and adults with age-related macular degeneration.
Plain-language title generated from the registry record
Registry title: A First-In-Human Study of ARO-033 in Adult Participants
Key facts
- Where
- Auckland, New ZealandSee the site
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT07662096, who can take part
Where in New Zealand
- AucklandAuckland, New ZealandTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Arrowhead Pharmaceuticals
- Trial code
- NCT07662096
- Record updated
- 15 September 2026
- Original description
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).Read full original descriptionShow less
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).
Inclusion: 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.
Exclusion: 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; 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; 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); History of major surgery within 90 days of Screening; 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; 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:; All Cohorts:.