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Completed

NCT Number: NCT05083364

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

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.

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Key information

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site 1, Camperdown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

(All Participants):

  • 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

Inclusion criteria

(C3G and IgAN Participants):

  • 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

(All Participants):

  • 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

Note: Additional Inclusion/Exclusion criteria may apply per protocol

Treatment and study plan

ARO-C3

Drug

ARO-C3 for sc injection

Placebo

Drug

sterile normal saline (0.9% NaCl) for sc injection

Primary outcomes

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

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

Secondary outcomes

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

    Time frame: up to 48 hours post-dose

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

    Time frame: up to 48 hours post-dose

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

    Time frame: up to 48 hours post-dose

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

    Time frame: up to 48 hours post-dose

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

    Time frame: up to 48 hours post-dose

  6. PK of ARO-C3: Apparent Total Body Clearance of ARO-C3 from Plasma (CL)

    Time frame: up to 48 hours post-dose

  7. PK of ARO-C3: Volume of Distribution (Vz/F)

    Time frame: up to 48 hours post-dose

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study title

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Complement-Mediated Renal Disease

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Oct 19, 2021
Registry last updated
May 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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