Study of ARO-CFB in Adult Healthy Volunteers and Patients With Complement-Mediated Kidney Disease

Phase 1

Recruiting

• Conditions: IgA Nephropathy
• Interventions: Drug: ARO-CFB; Drug: Placebo

• Registration Number: NCT06209177
• Lead Sponsor: Arrowhead Pharmaceuticals

Brief Summary

The purpose of AROCFB-1001 is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ARO-CFB Injection in adult healthy volunteers (HVs) and in adult patients with complement-mediated kidney disease (IgA Nephropathy \[IgAN\]). In Part 1 of the study, HVs will receive either one or two doses of ARO-CFB or placebo. In Part 2 of the study, adult patients with IgAN will receive 3 open-label doses of ARO-CFB. 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: 2024-01-05
• Study First Submitted QC: 2024-01-05
• Study First Posted: 2024-01-17
• Status Verified: 2024-04
• Study Start: 2024-04-22
• Last Update Submitted: 2024-04-23
• Last Update Posted: 2024-04-24
• Primary Completion: 2026-11
• Study Completion: 2027-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 66

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)	Part 1: up to Day 169 (End of Study [EOS]); Part 2: up to Day 225 (EOS)

Secondary Outcome Measures

Name	Time	Method
PK of ARO-CFB: Volume of Distribution (Vz/F)	Part 1 only: up to 48 hours post-dose
Change from Baseline in Serum Complement Factor B (CFB) and Its Breakdown Products Ba and Bb	Part 1: up to Day 169 (End of Study [EOS]); Part 2: up to Day 225 (EOS)
Pharmacokinetics (PK) of ARO-CFB: Maximum Observed Plasma Concentration (Cmax)	Part 1 only: up to 48 hours postdose
PK of ARO-CFB: Time to Maximum Observed Plasma Concentration (Tmax)	Part 1 only: up to 48 hours post-dose
PK of ARO-CFB: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)	Part 1 only: up to 48 hours post-dose
PK of ARO-CFB: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)	Part 1 only: up to 48 hours post-dose
PK of ARO-CFB: Area Under the Plasma Concentration Versus Time Curve from Zero Extrapolated to Infinity (AUCinf)	Part 1 only: up to 48 hours post-dose
PK of ARO-CFB: Terminal Elimination Half-Life (t1/2)	Part 1 only: up to 48 hours post-dose
PK of ARO-CFB: Fraction of Drug Excreted Unchanged (fe)	Part 1 only: up to 24 hours post-dose
PK of ARO-CFB: Renal Clearance (CLr)	Part 1 only: up to 24 hours post-dose
Percent Change from Baseline in Serum Complement Factor B (CFB) and Its Breakdown Products Ba and Bb	Part 1: up to Day 169 (End of Study [EOS]); Part 2: up to Day 225 (EOS)
PK of ARO-CFB: Apparent Clearance (CL/F)	Part 1 only: up to 48 hours post-dose
PK of ARO-CFB: Amount of Drug Recovered in Urine Over Zero - 24 Hours Post-dose (Ae)	Part 1 only: up to 24 hours post-dose

Trial Locations

Locations (1)

New Zealand Clinical Research OPCO Ltd; 🇳🇿 Auckland, New Zealand