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

NCT05083364 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: AROC3-1001
• Study Type: interventional
• Target: 62
• Locations: 7 countries
• Sites: 16

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

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) at Day 169

  up to day 169 (End of Study [EOS])

Secondary Outcomes (7)

• Pharmacokinetics (PK) of ARO-C3: Maximum Observed Plasma Concentration (Cmax)

  up to 48 hours post-dose

• PK of ARO-C3: Area under the Plasma Concentration Versus Time Curve from Zero to 24Hours (AUC0-24)

  up to 48 hours post-dose

• PK of ARO-C3: Area Under the Plasma Versus Time Concentration Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

  up to 48 hours post-dose

• PK of ARO-C3: Area Under the Plasma Concentration Versus Time Curve from Zero Extrapolated to Infinity (AUCinf) PK of ARO-C3:

  up to 48 hours post-dose

• PK of ARO-C3: Terminal Elimination Half-Life (t1/2)

  up to 48 hours post-dose

Study Arms (3)

ARO-C3 (Healthy Volunteers)

EXPERIMENTAL

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

Drug: ARO-C3

Placebo (Healthy Volunteers)

PLACEBO COMPARATOR

placebo calculated volume to match active treatment by sc injection

Drug: Placebo

ARO-C3 (Adult Patients with C3G or IgAN)

EXPERIMENTAL

3 doses of ARO-C3 by sc injection

Drug: ARO-C3

Interventions

ARO-C3 DRUG

ARO-C3 for sc injection

ARO-C3 (Adult Patients with C3G or IgAN); ARO-C3 (Healthy Volunteers)

Placebo DRUG

sterile normal saline (0.9% NaCl) for sc injection

Placebo (Healthy Volunteers)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (16)

Research Site 1

Camperdown, New South Wales, 2050, Australia

Location

Research Site 3

Concord, New South Wales, 2139, Australia

Location

Research Site 2

Clayton, Victoria, 3168, Australia

Location

Research Site 2

Tbilisi, 0112, Georgia

Location

Research Site 2

Cologne, 50937, Germany

Location

Research Site 4

Erlangen, 91054, Germany

Location

Research Site

Auckland, 1010, New Zealand

Location

Research Site 1

Gamcheon, Busan, 49267, South Korea

Location

Research Site 2

Haeundae, Busan, 48108, South Korea

Location

Research Site 4

Goyang-si, Gyeonggi-do, 10444, South Korea

Location

Research Site 3

Daegu, 42601, South Korea

Location

Research Site 6

Soeul, 03722, South Korea

Location

Research Site 8

Soeul, 05030, South Korea

Location

Research Site 2

Bangkok, 10400, Thailand

Location

Research Site 3

Chiang Mai, 50200, Thailand

Location

Research Site 4

Oxford, OX3 7LE, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 38299639 DERIVED

MeSH Terms

Conditions

Glomerulonephritis, IGA

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Part 1, participants are randomized to receive either ARO-C3 or placebo. Participants,care providers, investigator and outcomes assessors are all blinded to treatment assignment. Part 2 in patients with C3G or IgAN is open-label and there is no masking.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2021
• First Posted: October 19, 2021
• Study Start: February 1, 2022
• Primary Completion: May 5, 2025
• Study Completion: October 31, 2025
• Last Updated: April 29, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

DE (2); NZ (1); TH (2); GE (1); AU (3); KR (6); GB (1)