Grant: Project Grant

Anti-neutrophil cytoplasm antibody associated vasculitis, or AAV, is an uncommon disease affecting approximately 10,000 Canadians. AAV can affect any organ in the body by inflaming blood vessels. Before effective treatments were available, 90% of patients with AAV died within the first year of diagnosis. Treatments to suppress inflammation and the immune system can control AAV but it still commonly causes kidney failure. In addition to failing to prevent kidney failure, treatments for AAV cause serious side effects. Prednisone, the most common, and one of the most effective treatments, causes infections, diabetes, mental health problems, bone thinning, impaired healing, weight gain and many other issues. A recent, Canadian led, study demonstrated lower doses of prednisone reduce some side effects without higher risk of kidney failure. However, even at these lower doses many patients with AAV still develop many side-effects and kidney failure. A newer drug, avacopan, appears as effective as prednisone without most of the side effects. Avacopan was only studied in patients with AAV that did not threaten kidney failure. However, the small number of patients with kidney problems that were studied appeared to do even better with avacopan than prednisone. It is uncertain whether avacopan is really as good or better than prednisone in patients with more severe kidney disease from AAV. We will compare avacopan to the standard regimen of prednisone in patients with AAV with serious kidney involvement to see if it controls the AAV and maintains kidney function. We call this study the Glucocorticoids or Avacopan in peopLe with high-risk renal Anti-Neutrophil cytoplasm antibody associated vasculitis Trial (GALANT). Because rare diseases can be challanging to conduct, we will first conduct a small study to make sure GALANT is feasible (GALANT-Pilot). If GALANT-Pilot is successful, we will launch an international study to definitively answer this important question.