The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Oral administration
Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
Time frame: Week 26
Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS
Time frame: Week 52
Plasma Concentrations of Avacopan
Time frame: Day 1 up to Week 52
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)
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.
Time frame: Day 1 up to approximately Week 60
Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS)
Time frame: Week 26
Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS
Time frame: Week 52
Proportion of Participants With PVAS of 0 Over Time Through Week 52
Time frame: Up to Week 52
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Emory University
Atlanta, Georgia, United States
RECRUITINGRiley Hospital for Children
Indianapolis, Indiana, United States
RECRUITINGUniversity of Minnesota Masonic Childrens Hospital Discovery Clinic
Minneapolis, Minnesota, United States
RECRUITINGCohen Children Medical Center
Lake Success, New York, United States
RECRUITINGUniversity of North Carolina
Chapel Hill, North Carolina, United States
RECRUITINGWake Forest University Health Sciences
Charlotte, North Carolina, United States
RECRUITINGAkron Childrens Hospital
Akron, Ohio, United States
RECRUITINGCleveland Clinic Foundation
Cleveland, Ohio, United States
RECRUITINGUpmc Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
RECRUITINGTexas Childrens Hospital
Houston, Texas, United States
RECRUITING...and 20 more locations
Proportion of Participants With BVAS of 0 Over Time Through Week 52
Time frame: Up to Week 52
Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)
Time frame: Baseline up to Week 52
Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline up to Week 52
Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity
Time frame: Baseline up to Week 52
Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)
Time frame: Baseline up to Week 52
Number of Glucocorticoid Dosages Administered
Time frame: Screening up to Week 52
Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale
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.
Time frame: Day 1 and Week 2
Taste and Acceptability Score of Avacopan per TASTY Faces Scale
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.
Time frame: Day 1 and Week 2