Clinical trial NCT06794827: Early-access Avacopan in Real-world ANCA-associated Vasculitis
Official title: Evaluating Clinical Practice of Early Access Avacopan Treatment for ANCA-associated Vasculitis in European Countries
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: ANCA Associated Vasculitis
Lead sponsor: Leiden University Medical Center
Sponsor class: OTHER
Collaborators: name: University of Udine
name: University of Modena and Reggio Emilia
name: Hospital Universitari de Bellvitge
name: ZOL Hospital Genk Belgium
First posted: 2025-01-27
Enrollment: 100
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

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.

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

Eligibility criteria: 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

Primary outcomes:
measure: Assess physician's treatment indications for starting avacopan as reported in the database questionnaire
timeFrame: 1 year
measure: Assess AAV patients' clinical characteristics at baseline as reported in the database questionnaire
timeFrame: 1 year
measure: Assess concomitant immunosuppressive treatment strategies employed with avacopan as registered in the database questionnaire
timeFrame: 1 year
measure: Assess the relevant clinical benefit of avacopan treatment at the individual patient level based on the BVAS score [0-63]
timeFrame: 2 years
Secondary outcomes:
measure: Investigate determinants of non-response and/or relapse upon avacopan treatment based on baseline characteristics registered in the database questionnaire
timeFrame: 2 years
Other outcomes:
Locations:
city: Leiden
country: Netherlands
facility: Leiden University Medical Center
Study officials:
affiliation: Leiden University Medical Center
name: Y.K. Onno Teng
role: PRINCIPAL_INVESTIGATOR
References: