Clinical trial NCT06321601: Study to Evaluate Avacopan in Combination With a Rituximab or Cyclophosphamide-containing Regimen, in Children From 6 Years to < 18 Years of Age With AAV.
Official title: A Phase 3, Open-label, Uncontrolled Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Avacopan in Combination With a Rituximab or a Cyclophosphamide-containing Regimen in Children From 6 Years to < 18 Years of Age With Active ANCA-associated Vasculitis (AAV)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Vasculitis
Keywords: Pediatric
Avacopan
Tavneos®
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2024-03-20
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.


Eligibility criteria: Inclusion Criteria:

* Male and female children and adolescents from 6 to \< 18 years of age
* Clinical diagnosis of granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013)
* Newly diagnosed or relapsed AAV with positive test for anti-PR3 or anti-MPO antibodies
* At least 1 PVAS major item, at least 3 PVAS nonmajor items, or at least the 2 renal items of proteinuria and hematuria.
* eGFR \> 15 mL/min/1.73 m2 (using modified Schwartz equation per central lab guidelines)
* Participants must have a bodyweight of ≥ 15 kg at day 1.

Exclusion Criteria:

* Any other known multisystem autoimmune disease including eosinophilic granulomatosis with polyangiitis (Churg-Strauss), systemic lupus erythematosus , IgA vasculitis (Henoch-Schönlein), rheumatoid vasculitis, Sjogren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis
* Alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study
* Any medical condition requiring or expected to require continued use of immunosuppressive treatments, including corticosteroids that may cause confoundment with study assessments and study conclusions.

Primary outcomes:
measure: Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
timeFrame: Week 26
measure: Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS
timeFrame: Week 52
Secondary outcomes:
measure: Plasma Concentrations of Avacopan
timeFrame: Day 1 up to Week 52
measure: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)
timeFrame: Day 1 up to approximately Week 60
measure: Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS)
timeFrame: Week 26
measure: Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS
timeFrame: Week 52
measure: Proportion of Participants With PVAS of 0 Over Time Through Week 52
timeFrame: Up to Week 52
measure: Proportion of Participants With BVAS of 0 Over Time Through Week 52
timeFrame: Up to Week 52
measure: Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)
timeFrame: Baseline up to Week 52
measure: Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Baseline up to Week 52
measure: Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity
timeFrame: Baseline up to Week 52
measure: Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)
timeFrame: Baseline up to Week 52
measure: Number of Glucocorticoid Dosages Administered
timeFrame: Screening up to Week 52
measure: Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale
timeFrame: Day 1 and Week 2
measure: Taste and Acceptability Score of Avacopan per TASTY Faces Scale
timeFrame: Day 1 and Week 2
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Childrens Hospital Discovery Clinic
state: Minnesota
city: Lake Success
country: United States
facility: Cohen Children Medical Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Charlotte
country: United States
facility: Wake Forest University Health Sciences
state: North Carolina
city: Akron
country: United States
facility: Akron Childrens Hospital
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Pittsburgh
country: United States
facility: Upmc Childrens Hospital of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Texas Childrens Hospital
state: Texas
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Leuven
country: Belgium
facility: Universitair Ziekenhuis Leuven - Gasthuisberg
city: Edmonton
country: Canada
facility: Stollery Childrens Hospital
state: Alberta
city: Montreal
country: Canada
facility: CHU Sainte Justine
state: Quebec
city: Praha 2
country: Czechia
facility: Vseobecna fakultni nemocnice v Praze
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole
city: Bron cedex
country: France
facility: Hospices Civils de Lyon Hopital Femme Mere Enfant
city: Paris
country: France
facility: Hopital Necker Enfants Malades
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Szeged
country: Hungary
facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Krakow
country: Poland
facility: Uniwersytecki Szpital Dzieciecy w Krakowie
city: Warszawa
country: Poland
facility: Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
city: Bratislava
country: Slovakia
facility: Narodny ustav detskych chorob
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
state: Cataluña
city: Esplugues De Llorbregat
country: Spain
facility: Hospital Sant Joan de Deu
state: Cataluña
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: