Evaluating ARO-CFB in Healthy Adults and Patients with Kidney Disease
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
This study is testing a new injection called ARO-CFB to see how safe it is and how well it works in healthy adults and patients with a kidney disease called IgA Nephropathy.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 66 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Arrowhead Pharmaceuticals Industry-sponsored |
| Locations | 1 site (Auckland) |
| Trial ID | NCT06209177 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-CFB Injection in both healthy adult volunteers and patients suffering from IgA Nephropathy, a type of complement-mediated kidney disease. The trial is divided into two parts: the first part involves healthy volunteers receiving either ARO-CFB or a placebo, while the second part focuses on adult patients with IgAN receiving three open-label doses of ARO-CFB. The dosing for patients will be informed by the safety and pharmacodynamic data collected from the healthy volunteer phase.
Who should consider this trial
Good fit: Ideal candidates for this study include adult patients diagnosed with IgA Nephropathy and healthy adults aged 18-65 who meet the study's eligibility criteria.
Not a fit: Patients with severe kidney impairment or those who do not meet the inclusion criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to a new treatment option for patients with IgA Nephropathy, potentially improving their kidney function and overall health.
How similar studies have performed: While this approach is being evaluated in this specific context, similar studies targeting complement-mediated diseases have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
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. 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. Inclusion Criteria (IgAN Participants): * 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 (All Cohorts): * 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 Exclusion Criteria (IgAN Participants): * 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 Note: Additional Inclusion/Exclusion criteria may apply per protocol
Where this trial is running
Auckland
- New Zealand Clinical Research OPCO Ltd — Auckland, New Zealand (Recruiting)
Study contacts
- Study coordinator: Medical Monitor
- Email: gshekhtman@arrowheadpharma.com
- Phone: 626-696-4702
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.