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.
- Registration Number
- NCT06321601
- Lead Sponsor
- Amgen
- Brief Summary
The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 20
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Avacopan Avacopan Participants will receive avacopan twice-daily (BID) administered as oral tablets or liquid formula for 52 weeks.
- Primary Outcome Measures
Name Time Method Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS) Week 26 Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS Week 52
- Secondary Outcome Measures
Name Time Method Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI) Baseline up to Week 52 Taste and Acceptability Score of Avacopan per TASTY Faces Scale Day 1 and Week 2 The TASTY faces scale will be utilized to assess the taste of the oral pediatric formulation. A 7-point version of the scale will be used, where higher scores correspond to faces showing favorable tastes. Upon drug administration, the child will be shown the TASTY scale and asked to rate his/her perception of tastiness by pointing to the appropriate face on the scale.
Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS) Week 26 Proportion of Participants With BVAS of 0 Over Time Through Week 52 Up to Week 52 Plasma Concentrations of Avacopan Day 1 up to Week 52 Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE) Day 1 up to approximately Week 60 TEAEs are any event that occurred after the participant received study treatment. Any clinically significant changes in vital signs, electrocardiograms, and clinical laboratory tests that occurred after study treatment administration were recorded as TEAEs. A serious TEAE is any untoward medical occurrence in a clinical study participant after first dose irrespective of a causal relationship with the study treatment(s) that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, or another medically important serious event.
Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS Week 52 Proportion of Participants With PVAS of 0 Over Time Through Week 52 Up to Week 52 Number of Glucocorticoid Dosages Administered Screening up to Week 52 Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR) Baseline up to Week 52 Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR) Baseline up to Week 52 Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity Baseline up to Week 52 Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale Day 1 and Week 2 The TASTY faces scale will be utilized to assess the taste of the oral pediatric formulation. A 7-point version of the scale will be used, where higher scores correspond to faces showing favorable tastes. Upon drug administration, the child will be shown the TASTY scale and asked to rate his/her perception of tastiness by pointing to the appropriate face on the scale.
Trial Locations
- Locations (24)
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
University of Minnesota Masonic Childrens Hospital Discovery Clinic
🇺🇸Minneapolis, Minnesota, United States
Cohen Children Medical Center
🇺🇸Lake Success, New York, United States
Wake Forest University Health Sciences
🇺🇸Charlotte, North Carolina, United States
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
Akron Childrens Hospital
🇺🇸Akron, Ohio, United States
Upmc Childrens Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Texas Childrens Hospital
🇺🇸Houston, Texas, United States
Universitair Ziekenhuis Leuven - Gasthuisberg
🇧🇪Leuven, Belgium
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
CHU Sainte Justine
🇨🇦Montreal, Quebec, Canada
Vseobecna fakultni nemocnice v Praze
🇨🇿Praha 2, Czechia
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
Hospices Civils de Lyon Hopital Femme Mere Enfant
🇫🇷Bron cedex, France
Hopital Necker Enfants Malades
🇫🇷Paris, France
Semmelweis Egyetem
🇭🇺Budapest, Hungary
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdansk, Poland
Uniwersytecki Szpital Dzieciecy w Krakowie
🇵🇱Krakow, Poland
Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
🇵🇱Warszawa, Poland
Narodny ustav detskych chorob
🇸🇰Bratislava, Slovakia
Hospital Universitari Vall d Hebron
🇪🇸Barcelona, Cataluña, Spain
Hospital Sant Joan de Deu
🇪🇸Esplugues De Llorbregat, Cataluña, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain