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

NCT ID: NCT06209177

Last Updated: 2025-12-17

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 66 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-04-05
• Study Completion Date: 2026-03-31

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.

Conditions

IgA Nephropathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

ARO-CFB (Healthy Volunteers)

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

Group Type: EXPERIMENTAL

ARO-CFB

Intervention Type: DRUG

ARO-CFB for sc injection

Placebo (Healthy Volunteers)

placebo calculated volume to match active treatment by sc injection

Group Type: EXPERIMENTAL

Placebo

Intervention Type: DRUG

sterile normal saline (0.9% NaCl for sc injection)

ARO-CFB (Adult Patients with IgAN)

3 doses of ARO-CFB by sc injection

Group Type: EXPERIMENTAL

ARO-CFB

Intervention Type: DRUG

ARO-CFB for sc injection

Interventions

ARO-CFB

ARO-CFB for sc injection

Intervention Type: DRUG

Placebo

sterile normal saline (0.9% NaCl for sc injection)

Intervention Type: DRUG

Eligibility Criteria

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.

• Diagnosis of IgA Nephropathy based on renal biopsy within 5 years
• Clinical evidence of ongoing disease based on significant proteinuria
• Estimated glomerular filtration rate ≥30 mL/min/1.73m2 at Screening and currently not on dialysis
• Must have stable or worsening renal disease, on stable and optimized treatment for at least 30 days prior to Screening and willing to stay on a stable standard of care regimen for the duration of the study
• Must be on a maximally recommended or tolerated dose of an angiotensin-converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB)

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

• Nephrotic syndrome or rapidly progressive glomerulonephritis
• Suspicion for secondary etiologies of IgAN
• Evidence of non-IgAN kidney disease on renal biopsy
• Renal biopsy showing interstitial fibrosis/tubular atrophy of more than 50%
• Use of complement inhibitors or monoclonal antibody therapies for treatment of IgAN

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Arrowhead Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Research Site

Auckland, , New Zealand

Countries

New Zealand

Other Identifiers

AROCFB-1001

Identifier Type: -

Identifier Source: org_study_id