TAVNEOS for Otolaryngologic Manifestations of Granulomatosis With Polyangiitis
This is a single center double-blind placebo-controlled study. Patients with GPA and active ears, nose, and throat (ENT) disease in at least two ENT domains, as defined after endoscopic visualization of the upper airway and audiometric evaluation, if applicable, by a single otolaryngologist using a validated GPA ENT disease activity score, will be eligible for inclusion. Patients will be treated with standard of care (SOC) treatment as determined by their treating rheumatologist. In addition to SOC, patients will be randomized to receive TAVNEOS 30mg BID or placebo. Patients will be followed for 52 weeks with standardized ENT assessment along with rheumatologic evaluation of overall disease activity with BVAS.
• GPA diagnosis defined by score of ≥5 on 2022 ACR/EULAR Classification Criteria for GPA
• Active GPA (both newly diagnosed and relapsing disease) in the ENT domain within 1 month prior to screening, where the active disease is defined as a score of ≥2 on a GPA ENT disease activity score (7 items scored as 1= present 0= absent) performed by direct endoscopic visualization of the upper airway and audiometric evaluation, if applicable, by a single expert otolaryngologist. Items included in the GPA ENT disease activity score are:
‣ Bloody rhinorrhea (Daily blood stained nasal discharge)
⁃ Objective stridor (Stridor assessed by doctor)
⁃ Inflammation on nasal examination (Ulcers, granulation, friable mucosa on rigid nasal endoscopy. Excluding crusting)
⁃ Inflammation on flexible laryngoscopy (Ulcers, granulation, friable mucosa in the larynx)
⁃ Inflamed TM\*/middle ear (Persistent inflammation or granulation tissue in tympanic membrane/middle ear)
⁃ Sudden sensorineural hearing loss (30db drop in 3 frequencies within 72 hours)
• Other ENT/upper airway manifestations of active GPA observed during structured ENT exam including but not limited to lacrimal gland dacryocystitis and endobronchial disease
• Age 18 and older
• Willing and able to comply with treatment and follow-up procedures
• Men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for twelve months after completion of treatment.
• Willing and able to provide written informed consent
• Adequate liver function as defined by AST or ALT \<2x Upper Limit of Normal