Early-access Avacopan in Real-world ANCA-associated Vasculitis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: ANCA Associated Vasculitis. Basic parameters: from 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Netherlands
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Evaluating Clinical Practice of Early Access Avacopan Treatment for ANCA-associated Vasculitis in European Countries
Overview
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
Primary outcome measures
- Assess physician's treatment indications for starting avacopan as reported in the database questionnaire [Time frame: 1 year]
- Assess AAV patients' clinical characteristics at baseline as reported in the database questionnaire [Time frame: 1 year]
- Assess concomitant immunosuppressive treatment strategies employed with avacopan as registered in the database questionnaire [Time frame: 1 year]
- Assess the relevant clinical benefit of avacopan treatment at the individual patient level based on the BVAS score [0-63] [Time frame: 2 years]
Secondary outcome measures (1)
- Investigate determinants of non-response and/or relapse upon avacopan treatment based on baseline characteristics registered in the database questionnaire [Time frame: 2 years]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Netherlands · 1 center
- Leiden University Medical Center — Leiden
Identifiers
NCT: NCT06794827 · IIS-AVA-2023-0005