Early-access Avacopan in Real-world ANCA-associated Vasculitis

NCT06794827 | Recruiting

• 1 other identifier: IIS-AVA-2023-0005
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

ANCA Associated Vasculitis

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (1)

• Investigate determinants of non-response and/or relapse upon avacopan treatment based on baseline characteristics registered in the database questionnaire

  2 years

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Leiden University Medical Center: lead
• University of Udine: collaborator
• University of Modena and Reggio Emilia: collaborator
• Hospital Universitari de Bellvitge: collaborator
• ZOL Hospital Genk Belgium: collaborator

Study Sites (1)

Leiden University Medical Center

Leiden, Netherlands

RECRUITING

MeSH Terms

Conditions

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Y.K. Onno Teng

  Leiden University Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Y.K.O. Teng, MD, PhD

CONTACT

+31715262148; y.k.o.teng@Lumc.nl

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: March 1, 2024
• First Posted: January 27, 2025
• Study Start: November 1, 2023
• Primary Completion: January 1, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: January 27, 2025

Record last verified: 2025-01

Locations

NL (1)