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

Phase 1

Active, not recruiting

• Conditions: C3 Glomerulopathy; IgA Nephropathy
• Interventions: Drug: ARO-C3; Drug: Placebo

• Registration Number: NCT05083364
• Lead Sponsor: Arrowhead Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-10-08
• Study First Submitted QC: 2021-10-08
• Study First Posted: 2021-10-19
• Study Start: 2022-02-02
• Status Verified: 2025-01
• Last Update Submitted: 2025-03-23
• Last Update Posted: 2025-03-26
• Primary Completion: 2025-05
• Study Completion: 2025-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 62

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
ARO-C3 (Adult Patients with C3G or IgAN)	ARO-C3	3 doses of ARO-C3 by sc injection
ARO-C3 (Healthy Volunteers)	ARO-C3	1 or 2 doses of ARO-C3 by subcutaneous (sc) injection
Placebo (Healthy Volunteers)	Placebo	placebo calculated volume to match active treatment by sc injection

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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: Apparent Total Body Clearance of ARO-C3 from Plasma (CL)	up to 48 hours post-dose
Pharmacodynamics (PD): Change From Baseline in Complement 3 (C3) up to Day 169	Baseline, through Day 169 (EOS)
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 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
PK of ARO-C3: Volume of Distribution (Vz/F)	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
PD: Percent Change From Baseline in C3 up to Day 169	Baseline, through Day 169 (EOS)

Trial Locations

Locations (8)

Research Site 3; 🇹🇭 Chiang Mai, Thailand

Research Site; 🇳🇿 Auckland, New Zealand

Research Site 2; 🇹🇭 Bangkok, Thailand

Research Site 4; 🇬🇧 Oxford, United Kingdom

Research Site 6; 🇬🇧 Newcastle, United Kingdom

Research Site 7; 🇰🇷 Soeul, Korea, Republic of

Research Site 1; 🇬🇧 Leicester, United Kingdom

Research Site 8; 🇰🇷 Soeul, Korea, Republic of