Study of ARO-CFB in Adult Healthy Volunteers

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ARO-CFB in Adult Healthy Volunteers and Adult Patients With Complement-Mediated Kidney Disease

The purpose of AROCFB-1001 is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ARO-CFB Injection in adult healthy volunteers (HVs). HVs will receive either one or two doses of ARO-CFB or placebo.

Study Overview

• Status: Completed
• Conditions: IgA Nephropathy
• Intervention / Treatment: Drug: ARO-CFB; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 49
• Phase: Phase 1

Contacts and Locations

Study Locations

• New Zealand
  • Auckland, New Zealand, 1010
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

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. 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 type B
• Body Mass Index (BMI) between 18.0 and 35.0 kg/m2
• Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or the last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days 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 adversely impact study results.

Exclusion Criteria:

• History of recurrent or chronic infections including infections caused by encapsulated bacterial organisms or viruses
• History of active bacterial, viral, or fungal infection within 14 days prior to treatment administrations
• Seropositive for Human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV)
• History of meningococcal infection
• History of asplenia
• History of severe aplastic anemia or concurrent severe aplastic anemia
• Known or suspected hereditary complement deficiency or other primary immunodeficiency syndrome
• History of diabetes mellitus (Type 1 or Type 2)
• Uncontrolled hypertension

Note: Additional Inclusion/Exclusion criteria may apply per protocol

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ARO-CFB (Healthy Volunteers); 1 or 2 doses of ARO-CFB by subcutaneous (sc) injection	Drug: ARO-CFB; ARO-CFB for sc injection
Experimental: Placebo (Healthy Volunteers); placebo calculated volume to match active treatment by sc injection	Drug: Placebo; sterile normal saline (0.9% NaCl for sc injection)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)	up to Day 169 (End of Study [EOS])

Secondary Outcome Measures

Outcome Measure	Time Frame
Pharmacokinetics (PK) of ARO-CFB: Maximum Observed Plasma Concentration (Cmax)	up to 48 hours postdose
PK of ARO-CFB: Time to Maximum Observed Plasma Concentration (Tmax)	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)	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)	up to 48 hours post-dose
PK of ARO-CFB: Area Under the Plasma Concentration Versus Time Curve from Zero Extrapolated to Infinity (AUCinf)	up to 48 hours post-dose
PK of ARO-CFB: Terminal Elimination Half-Life (t1/2)	up to 48 hours post-dose
PK of ARO-CFB: Apparent Clearance (CL/F)	up to 48 hours post-dose
PK of ARO-CFB: Volume of Distribution (Vz/F)	up to 48 hours post-dose
PK of ARO-CFB: Amount of Drug Recovered in Urine Over Zero - 24 Hours Post-dose (Ae)	up to 24 hours post-dose
PK of ARO-CFB: Fraction of Drug Excreted Unchanged (fe)	up to 24 hours post-dose
PK of ARO-CFB: Renal Clearance (CLr)	up to 24 hours post-dose

Collaborators and Investigators

• Sponsor: Arrowhead Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): April 5, 2024
• Primary Completion (Actual): March 31, 2025
• Study Completion (Actual): March 6, 2026

Study Registration Dates

• First Submitted: January 5, 2024
• First Submitted That Met QC Criteria: January 5, 2024
• First Posted (Actual): January 17, 2024

Study Record Updates

• Last Update Posted (Actual): May 20, 2026
• Last Update Submitted That Met QC Criteria: May 19, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Autoimmune Diseases; Immune System Diseases; Glomerulonephritis; Nephritis; Glomerulonephritis, IGA
• Other Study ID Numbers: AROCFB-1001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes