Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease

NCT ID: NCT05083364

Last Updated: 2025-12-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 62 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-02-01
• Study Completion Date: 2025-10-31

Brief Summary

The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). In Part 1 of the study, HVs will receive either one or two doses of ARO-C3 or placebo. In Part 2 of the study, adult patients with C3G/IgAN will receive 3 open-label doses of ARO-C3. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

Conditions

C3 Glomerulopathy; IgA Nephropathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

ARO-C3 (Healthy Volunteers)

1 or 2 doses of ARO-C3 by subcutaneous (sc) injection

Group Type: EXPERIMENTAL

ARO-C3

Intervention Type: DRUG

ARO-C3 for sc injection

Placebo (Healthy Volunteers)

placebo calculated volume to match active treatment by sc injection

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

sterile normal saline (0.9% NaCl) for sc injection

ARO-C3 (Adult Patients with C3G or IgAN)

3 doses of ARO-C3 by sc injection

Group Type: EXPERIMENTAL

ARO-C3

Intervention Type: DRUG

ARO-C3 for sc injection

Interventions

ARO-C3

ARO-C3 for sc injection

Intervention Type: DRUG

Placebo

sterile normal saline (0.9% NaCl) for sc injection

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Willing to provide written informed consent and to comply with study requirements
• Female participants must be non-pregnant/non-lactating
• Healthy volunteers must be willing to be vaccinated with a meningococcal and pneumococcal vaccine. C3G and IgAN participants must have been vaccinated or willing to undergo vaccination
• All participants must be willing to be vaccinated or have a history of vaccination for Haemophilus influenzae
• Body Mass Index (BMI) between 18.0 and 35.0 kg/m2
• 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety at discretion of investigator
• Participants of childbearing potential must use highly effective contraception during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Males must not donate sperm during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later.
• No abnormal finding of clinical relevance at the Screening evaluation that, in the opinion of the investigator, could adversely impact participant safety or study results

• Diagnosis of C3G or IgAN
• Clinical evidence of ongoing disease based on significant proteinuria
• Estimated glomerular filtration rate ≥30 mL/Min/1.73 m2 at Screening and currently not on dialysis
• Must be on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB)

Exclusion Criteria

• Seropositive for human immunodeficiency virus (HIV) infection,hepatitis B virus, or hepatitis C virus
• History of recurrent or chronic infections
• Uncontrolled hypertension
• Regular use of alcohol within 30 days prior to Screening
• Use of illicit drugs within 1 year prior to Screening or positive urine drug screen at Screening
• History of meningococcal infection
• History of asplenia or splenectomy
• Known contraindication or history of anaphylactic reaction to any vaccine or vaccine component or prophylactic antibiotics planned for use in the study
• Any medical or surgical condition that, in the opinion of the investigator, would expose the participant to a significant safety risk or compromise the results of the study

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Arrowhead Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Research Site 1

Camperdown, New South Wales, Australia

Research Site 3

Concord, New South Wales, Australia

Research Site 2

Clayton, Victoria, Australia

Research Site 2

Tbilisi, , Georgia

Research Site 2

Cologne, , Germany

Research Site 4

Erlangen, , Germany

Research Site

Auckland, , New Zealand

Research Site 1

Gamcheon, Busan, South Korea

Research Site 2

Haeundae, Busan, South Korea

Research Site 3

Daegu, Daegu, South Korea

Research Site 4

Goyang-si, Gyeonggi-do, South Korea

Research Site 6

Soeul, , South Korea

Research Site 8

Soeul, , South Korea

Research Site 2

Bangkok, , Thailand

Research Site 3

Chiang Mai, , Thailand

Research Site 4

Oxford, , United Kingdom

Countries

Australia; Georgia; Germany; New Zealand; South Korea; Thailand; United Kingdom

References

Tunnicliffe DJ, Reid S, Craig JC, Samuels JA, Molony DA, Strippoli GF. Non-immunosuppressive treatment for IgA nephropathy. Cochrane Database Syst Rev. 2024 Feb 1;2(2):CD003962. doi: 10.1002/14651858.CD003962.pub3.

Reference Type: DERIVED

PMID: 38299639 (View on PubMed)

Other Identifiers

AROC3-1001

Identifier Type: -

Identifier Source: org_study_id