Clinical trial NCT05897684: Avacostar - (PASS)
Official title: Avacostar - A Post Authorization Safety Study (PASS) to Evaluate the Incidence of Safety Events of Interest in Patients Treated With Avacopan for ANCA-associated Vasculitis (AAV)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: ANCA-associated Vasculitis
Keywords: anti-neutrophil cytoplasmic antibody (ANCA)-AAV
Lead sponsor: Vifor Fresenius Medical Care Renal Pharma
Sponsor class: INDUSTRY
First posted: 2023-06-09
Enrollment: 500
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The Avacostar PASS is a non-interventional, multi-national, prospective cohort study that will collect data from 2 cohorts of patients: those treated with avacopan for active severe AAV, and a second cohort treated with a cyclophosphamide or rituximab-based induction regimen without avacopan for active severe AAV.

Brief summary: The Avacostar PASS is a non-interventional, multi-national, prospective cohort study that will collect data from 2 cohorts of patients: those treated with avacopan for active severe AAV, and a second cohort treated with a cyclophosphamide or rituximab-based induction regimen without avacopan for active severe AAV. The overall study duration is anticipated to be up to 7 years, including a recruitment period of approximately 3 years.

Detailed description: The Avacostar PASS is a non-interventional, multi-national, prospective cohort study that will collect data from 2 cohorts of patients: those treated with avacopan for active severe AAV, and a second cohort treated with a cyclophosphamide or rituximab-based induction regimen without avacopan for active severe AAV. The overall study duration is anticipated to be up to 7 years, including a recruitment period of approximately 3 years. Enrolled patients will be followed until the last patient last visit (LPLV) milestone, which will be 4 years after the last participant is enrolled.

Germany and the United Kingdom (UK) have been selected for the study. Additional countries may be considered according to availability of avacopan and suitability for the study. Patients will be enrolled prospectively, but up to 6 months of data may be collected retrospectively if necessary. Baseline visit is defined as the day that induction treatment (avacopan or non-avacopan standard of care (SoC) cyclophosphamide or rituximab) is started for active severe AAV. Patients who started avacopan/SoC induction therapy for active severe AAV within 6 months of the enrolment visit and fulfil eligibility criteria may be enrolled in the PASS. Individual participant follow-up data will be collected periodically at routine clinic visits until the LPLV, which will be 4 years after the last participant is enrolled.

The primary objective of the study is:

To evaluate the incidence of defined Medical Events of Special Interest (MESIs) in patients with AAV commencing avacopan.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of AAV (MPA or GPA), as determined by the Investigator according to their usual practice.
* Active, severe AAV at the time of commencing avacopan or non-avacopan SoC induction therapy, in the opinion of the Investigator.
* Age ≥18 years of either sex.
* Has provided written informed consent.
* Has commenced within the previous 6 months, or is planned to commence avacopan, cyclophosphamide or rituximab for the treatment of severe, active AAV outside of an interventional clinical study.

Exclusion Criteria:

• Concurrent participation in an interventional study, unless prospectively discussed and agreed with the Medical Monitor.

Primary outcomes:
measure: To evaluate the incidence of defined MESIs
timeFrame: up to 7 years
Secondary outcomes:
measure: Incidence rates of AE in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: AEs leading to discontinuation of therapy in the avacopan group
timeFrame: up to 7 years
measure: Incidence of SAEs in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Incidence of ADRs in the avacopan group
timeFrame: up to 7 years
measure: Incidence of SADRs in the avacopan group
timeFrame: up to 7 years
measure: Change in eGFR over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in IgG over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in CPK over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in ALT over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in AST over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in billirubin over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in WBC over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change Albumin over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Time to first flare over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Change in VDI scores over time in the avacopan and non-avacopan groups
timeFrame: up to 7 years
measure: Incidence of MESIs in the non-avacopan group
timeFrame: up to 7 years
measure: Incidence rates of SAEs and MESIs in the avacopan group compared to the non-avacopan group,
timeFrame: up to 7 years
measure: Incidence rates of MESIs in the avacopan group compared to the non-avacopan group,
timeFrame: up to 7 years
measure: Use of concomitant immunosuppression over time and cumulative by treatment group,
timeFrame: up to 7 years
measure: Duration of GC-free periods
timeFrame: up to 7 years
measure: Proportion of GC-free patients over time by treatment group.
timeFrame: up to 7 years
measure: Duration of treatment with avacopan by reason for treatment discontinuation.
timeFrame: up to 7 years
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Charité University Medicine
city: Cologne
country: Germany
facility: University Hospital of Cologne
city: Dresden
country: Germany
facility: Municipal Hospital Dresden
city: Essen
country: Germany
facility: University Hospital Essen
city: Freiburg
country: Germany
facility: University Hospital Freiburg
city: Göttingen
country: Germany
facility: University Medical Center Göttingen
city: Hamburg
country: Germany
facility: University Hospital Eppendorf
city: Hannover
country: Germany
facility: KRH Klinikum Siloah
city: Herne
country: Germany
facility: Rheumazentrum Ruhrgebiet
city: Munich
country: Germany
facility: LMU
city: Plochingen
country: Germany
facility: Medius Kliniken
city: Sendenhorst
country: Germany
facility: St. Josef-Stift Sendenhorst
city: Birmingham
country: United Kingdom
facility: UHB NHS Foundation Trust
city: Bradford
country: United Kingdom
facility: Bradford Teaching Hospitals NHS Foundation Trust
city: Bristol
country: United Kingdom
facility: North Bristol NHS Trust
city: Cambridge
country: United Kingdom
facility: Addenbrookes Hospital
city: Canterbury
country: United Kingdom
facility: East Kent Hospitals University NHS FT
city: Cardiff
country: United Kingdom
facility: Cardiff and Vale UHB
city: Carshalton
country: United Kingdom
facility: Epsom & St. Helier NHS Trust
city: Coventry
country: United Kingdom
facility: University Hospitals Coventry and Warwickshire
city: Exeter
country: United Kingdom
facility: Royal Devon University Healthcare NHS Foundation Trust
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow & Clyde
city: Leicester
country: United Kingdom
facility: University Hospitals of Leicester NHS Trust
city: London
country: United Kingdom
facility: Barts Health
city: London
country: United Kingdom
facility: Hammersmith Hospital, Imperial College Healthcare NHS Trust
city: London
country: United Kingdom
facility: Kings College Hospital
city: London
country: United Kingdom
facility: Royal Free
city: London
country: United Kingdom
facility: St Thomas' Hospital
city: Manchester
country: United Kingdom
facility: Manchester University NHS Foundation Trust
city: Nottingham
country: United Kingdom
facility: Nottingham university hospitals NHS trust
city: Oxford
country: United Kingdom
facility: Rheumatology Department, Oxford University Hospitals NHS Foundation Trust
city: Reading
country: United Kingdom
facility: Royal Berkshire NHS foundation trust
city: Salford
country: United Kingdom
facility: Northern Care Alliance
city: Swansea
country: United Kingdom
facility: Swansea Bay University LHB
city: York
country: United Kingdom
facility: York & Scarborough Teaching Hospitals NHS FT
Study officials:
References: