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.
The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.
Eligibility
Sex
ALL
Min age
6 Years
Max age
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.
Primary outcome measure(s)
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
Trial sites (34)
Facility
City
Region
Status
Emory University
Atlanta
Georgia
Riley Hospital for Children
Indianapolis
Indiana
University of Minnesota Masonic Childrens Hospital Discovery Clinic
Minneapolis
Minnesota
Cohen Children Medical Center
Lake Success
New York
University of North Carolina
Chapel Hill
North Carolina
Wake Forest University Health Sciences
Charlotte
North Carolina
Akron Childrens Hospital
Akron
Ohio
Cleveland Clinic Foundation
Cleveland
Ohio
University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh
Pittsburgh
Pennsylvania
Texas Childrens Hospital
Houston
Texas
Universitair Ziekenhuis Gent
Ghent
Belgium
Universitair Ziekenhuis Leuven - Gasthuisberg
Leuven
Belgium
Alberta Childrens Hospital
Calgary
Alberta
Stollery Childrens Hospital
Edmonton
Alberta
British Columbia Childrens Hospital
Vancouver
British Columbia
CHU Sainte Justine
Montreal
Quebec
Vseobecna fakultni nemocnice v Praze
Prague
Czechia
Fakultni nemocnice Motol a Homolka
Prague
Czechia
Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin
Bordeaux
France
Hospices Civils de Lyon Hopital Femme Mere Enfant
Bron
France
Hopital Necker Enfants Malades
Paris
France
Hopital Necker
Paris
France
Semmelweis Egyetem
Budapest
Hungary
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar
Szeged
Hungary
Uniwersyteckie Centrum Kliniczne
Gdansk
Poland
Uniwersytecki Szpital Dzieciecy w Krakowie
Krakow
Poland
Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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