Early-access Avacopan in Real-world ANCA-associated Vasculitis (AVAC-EUR)

Evaluating Clinical Practice of Early Access Avacopan Treatment for ANCA-associated Vasculitis in European Countries

The current study proposal aims to investigate the clinical practice variation of the initial use of avacopan for AAV patients in Europe. The study will describe patient characteristics and organ-specific clinical benefit of avacopan treatment in a unique, first-experience cohort of AAV patients. By studying the clinical practice variation of first-experience avacopan treatment, this study will be uniquely positioned to identify pivotal issues on the real-life implementation of avacopan treatment. These observational data can ultimately serve to address current knowledge gaps on avacopan treatment in AAV patients, improve the care and drug-access for AAV patients with avacopan treatment and generate new areas of research.

Study Overview

• Status: Recruiting
• Conditions: ANCA Associated Vasculitis

Detailed Description

Objective: Analyse the clinical practice variation of the early access use of avacopan in real-world treated patients with ANCA-associated vasculitis.

Study design: Observational cohort study Study population: AAV patients treated with avacopan.

Main study parameters/endpoints:

• Describe physician's treatment indications for starting avacopan
• Describe AAV patients clinical characteristics
• Describe the concomitant treatment strategies employed with avacopan
• Describe clinical outcomes of avacopan treatment
• Investigate determinants of clinical benefit as well as non-response and/or relapse within the study cohort

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Y.K.O. Teng, MD, PhD; Phone Number: +31715262148; Email: y.k.o.teng@Lumc.nl

Study Locations

• Netherlands
  • Leiden, Netherlands
    • Recruiting
    • Leiden University Medical Center
    • Contact: YKO Teng, MD, PhD; Email: y.k.o.teng@Lumc.nl

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

A multicenter, non-interventional, longitudinal cohort study which will collect incident as well as prevalent AAV patients treated with an avacopan-based regimen. The cohort will include retrospective and prospective data capture on real-life management and outcomes.

Description

Inclusion Criteria:

• Established diagnosis of GPA or MPA
• Treatment (initiation) with avacopan
• Informed consent for use of medical data

Exclusion Criteria:

• If there is no AAV disease activity present that can be supported by an objectifiable clinical measure or assessment
• If a subject is below 16 years old

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess physician's treatment indications for starting avacopan as reported in the database questionnaire	Assess physician's treatment indications for starting avacopan as reported in the database questionnaire. Options to choose are: Refractory disease, Relapsing disease, Steroid dependent, Steroid toxicity or Other	1 year
Assess AAV patients' clinical characteristics at baseline as reported in the database questionnaire	Assess AAV patients' clinical characteristics at baseline as reported in the database questionnaire including: Gender (Male/Female) Age Date of AAV diagnosis Type AAV (GPA or MPA) Autoantibodies (PR3 or MPO) Organs affected	1 year
Assess concomitant immunosuppressive treatment strategies employed with avacopan as registered in the database questionnaire	Assess concomitant treatment strategies employed with avacopan at baseline, 1 month, 3 months, 6 months, 9 months, 12 months, 18 months and 24 monhts. Possible options include steroids, cyclophosphamide, rituximab, azathioprine or other	1 year
Assess the relevant clinical benefit of avacopan treatment at the individual patient level based on the BVAS score [0-63]	Assess the relevant clinical benefit of avacopan treatment at the individual patient level based on the BVAS score [0-63] as reported at baseline, 1 month, 3 months, 6 months, 9 months, 12 months, 18 months and 24 monhts.	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Investigate determinants of non-response and/or relapse upon avacopan treatment based on baseline characteristics registered in the database questionnaire	Investigate determinants of non-response and/or relapse upon avacopan treatment based on baseline characteristics registered in the database questionnaire including: Gender (Male/Female) Age Date of AAV diagnosis Type AAV (GPA or MPA) Autoantibodies (PR3 or MPO) Organs affected	2 years

Collaborators and Investigators

• Sponsor: Leiden University Medical Center
• Collaborators: University of Udine; Hospital Universitari de Bellvitge; University of Modena and Reggio Emilia; ZOL Hospital Genk Belgium
• Investigators: Principal Investigator: Y.K. Onno Teng, Leiden University Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2023
• Primary Completion (Estimated): January 1, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: March 1, 2024
• First Submitted That Met QC Criteria: January 22, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 22, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Skin Diseases, Vascular; Systemic Vasculitis; Vasculitis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
• Other Study ID Numbers: IIS-AVA-2023-0005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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