A First-In-Human Study of ARO-033 in Adult Participants
ConditionAge-related Macular Degeneration
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorArrowhead Pharmaceuticals
About this trial
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).
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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 (m²), 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.
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
ARO-033
DrugARO-033 will be administered as a subcutaneous (SC) injection per schedule specified in the arm description.
Placebo
DrugPlacebo matching to ARO-033 will be administered as SC injection per schedule specified in the arm description.
Treatment groups
84 Participants
are divided into 4 treatment groupsGroup A: NHV Cohorts: ARO-033Experimental treatment 1 intervention
Group B: NHV Cohorts: PlaceboPlacebo comparator 1 intervention
Group C: AMD Cohorts: ARO-033Experimental treatment 1 intervention
Group D: AMD Cohorts: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame
Up to Day 337Secondary 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)Sponsors and contacts
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Source ClinicalTrials.gov