Vasculitis
Conditions
Keywords
Pediatric, Avacopan, Tavneos®
Brief summary
The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.
Interventions
DRUGAvacopan
Oral administration
Sponsors
Amgen
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Male and female children and adolescents from 6 to \< 18 years old of age. * Clinical diagnosis of Granulomatosis with Polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013). * Positive anti-PR3(Anti-Proteinase 3) or anti-MPO(Anti-Myeloperoxidase) antibody documented at Screening or historically. Historical positivity is acceptable (even if Screening is negative) if supported by verifiable lab source documentation obtained during AAV diagnosis or disease course; use the most recent positive result. * At least 1 PVAS major item, at least 3 PVAS nonmajor items, or atleast the 2 renal items of proteinuria and hematuria. * Estimated glomerular filtration rate (eGFR) of ≥ 15 mL/minute/1.73 m\^2 at screening and day 1. * Participants must have a bodyweight of ≥ 15 kg at day 1.
Exclusion criteria
* Any other known multisystem autoimmune disease including and not limited to eosinophilic granulomatosis with polyangiitis (EGPA, previously, Churg-Strauss disease), systemic lupus erythematosus, IgA vasculitis / Henoch-Schönlein, Purpura, rheumatoid vasculitis, Sjögren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis. * Renal replacement therapy / plasmapheresis: subjects will be excluded who received, require, or initiate CRRT (continuous renal replacement therapy), hemodialysis, any renal dialysis, or plasmapheresis within 14 days prior to Screening or between Screening and Day 1. * History of kidney transplantation or is anticipated to require renal transplantation during the study. * 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, ongoing treatment with immunosuppressive, therapy (including systemic glucocorticoids) for a non-AAV indication that in the judgment of the investigator, could confound study assessments or interpretation of study results. * Female subjects of childbearing potential must have a negative highly sensitive serum pregnancy test at Screening and a negative sensitive urine pregnancy test, on day 1, with results confirmed prior to the first administration of investigational product. * Known hypersensitivity or contraindication to avacopan, its excipients, or to any investigational product or required concomitant medication used in this study. * Subject likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures (eg, Clinical Outcome Assessments) to the best of the subject and investigator's knowledge. * History or evidence of any other clinically significant disorder, condition, or disease (other than those specified above) that, in the investigator's judgment, would pose an unacceptable risk to subject safety, or interfere with study assessments or completion. The investigator may consult the Amgen medical monitor as needed. The rationale for exclusion and any consultation must be documented in the subject's source record.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 26 According to the Birmingham Vasculitis Activity Score (BVAS) | Week 26 | — |
| 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 | — |
| Proportion of Participants With BVAS of 0 Over Time Through Week 52 | 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 | — |
| Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI) | Baseline up to Week 52 | — |
| Number of Glucocorticoid Dosages Administered | Screening 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. |
| 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. |
Countries
Belgium, Canada, Czechia, France, Hungary, Poland, Slovakia, Spain, Turkey (Türkiye), United States
Contacts
STUDY_DIRECTORMD
Amgen
Outcome results
None listed