跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NCT07176546
NCT07176546招募中2 期

TAVNEOS for Otolaryngologic Manifestations of Granulomatosis With Polyangiitis

Robert Spiera, MD6 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2026年5月22日最近更新:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
适应症
干预措施
相关药物

试验速览

阶段
2 期
状态
招募中
发起方
入组人数
30
试验地点
6
主要终点
Proportion of patients in ENT remission without relapse
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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.

研究设计

研究类型
Interventional
分配方式
Randomized
干预模型
Parallel
主要目的
Treatment
盲法
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

入排标准

年龄范围
18 Years 至 —（Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •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

排除标准

  • •Creatinine >4.0mg/dl or GFR <15 at baseline or dependence on dialysis
  • •Respiratory failure requiring mechanical ventilatory support or had experienced alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study
  • •Previous treatment with TAVNEOS within 6 months of screening
  • •Inability to comply with study and/or follow-up procedures at investigator discretion.
  • •Intravenous glucocorticoids in the 4 weeks prior to screening except as premedication prior to infusion of rituximab
  • •Pregnant or breast-feeding
  • •Any other known multi-system autoimmune disease including eosinophilic granulomatosis with polyangiitis (Churg-Strauss), systemic lupus erythematosus, IgA vasculitis (Henoch-Schonlein), rheumatoid vasculitis, Sjogren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis
  • •Required dialysis or plasma exchange within 12 weeks prior to screening
  • •Have had a kidney transplant
  • •Any of the following within 12 weeks prior to screening: symptomatic congestive heart failure requiring prescription medication, unstable angina (unless successfully treated with stent or bypass surgery), clinically significant cardiac arrhythmia, myocardial infarction or stroke
  • •History or presence of any form of cancer within the 5 years prior to screening, with the exception of excised basal cell or squamous cell carcinoma of the skin, or carcinoma in situ such as cervical or breast carcinoma in situ that has been excised or resected completely and is without evidence of local recurrence or metastasis
  • •Evidence of tuberculosis based on interferon gamma release assay (IGRA), tuberculin purified protein derivative (PPD) skin test, or chest radiography (X rays or CT scan) done at screening or within 6 weeks prior to screening
  • •HBV, HCV, or HIV viral screening test showing evidence of active or chronic viral infection done at screening or within 6 weeks prior to screening
  • •Received a live vaccine within 4 weeks prior to screening
  • •WBC count less than 3500/uL, or neutrophil count less than 1500/uL, or lymphocyte count less than 500/uL before start of dosing
  • •Evidence of hepatic disease: AST, ALT, alkaline phosphatase, or bilirubin >3 times the upper limit of normal before start of dosing
  • •Known hypersensitivity to avacopan or inactive ingredients of the TAVNEOS capsules
  • •Participated in any clinical study of an investigational product within 30 days prior to screening or within 5 half-lives after taking the last dose
  • •Participated previously in an TAVNEOS study
  • •Concurrent use of strong inducers of the CYP450 ie. carbamazepine, phenobarbital, phenytoin, rifampin, or St. John's wort
  • •Known hypersensitivity to avacopan or inactive ingredients of the avacopan capsules (including gelatin, polyethylene glycol, or Cremophor)
  • •History or presence of any medical condition or disease which, in the opinion of the Investigator, may place the patient at unacceptable risk for study participation

研究组 & 干预措施

TAVNEOS

Active Comparator

BID dose of 30 mg TAVNEOS

干预措施: Avacopan (Drug)

Placebo

Placebo Comparator

BID dose of 30 mg TAVNEOS-matching placebo

干预措施: Placebo (Drug)

结局指标

主要结局

Proportion of patients in ENT remission without relapse

时间窗: Week 52

The proportion of patients in ENT remission without relapse in each treatment group at week 52 with no glucocorticoid (GC) exposure 4 weeks prior to week 52, where ENT remission is defined as a score of 0 on GPA ENT disease activity score

次要结局

  • Mean ENT disease activity score(Week 52)
  • Change in ENT disease activity score(Between week 26 and week 52)
  • Duration of steroid-free remission(Through study completion, an average of 60 weeks)
  • ANCA-Associated Vasculitis Patient-Reported Outcome (AAV-PRO)(Through study completion, an average of 60 weeks)
  • Number of surgical procedures in the ENT domain required(Through study completion, an average of 60 weeks)
  • Number of ENT flares(Through study completion, an average of 60 weeks)
  • Proportion of patients in ENT remission with BVAS 0 at week 52 and sustained remission(Week 52)
  • Cumulative steroid dose(Through study completion, an average of 60 weeks)
  • Change in Vasculitis Damage Index (VDI)(Week 52)
  • Sino-nasal Outcome Test (SNOT-22)(Through study completion, an average of 60 weeks)
  • Time to ENT relapse(Through study completion, an average of 60 weeks)
  • Number of GPA flares(Through study completion, an average of 60 weeks)

研究者

发起方
Robert Spiera, MD
申办方类型
Other
责任方
Sponsor Investigator
主要研究者

Robert Spiera, MD

Principal Investigator

Hospital for Special Surgery, New York

研究点 (6)

Loading locations...

相似试验

已完成
不适用
.A.-N30N30
PER-002-96ABORATORIOS BAGO DEL PERU S.A.,
进行中（未招募）
不适用
A double-blind, placebo-controlled multi-centre trial of memantine in patients with Parkinsons’ disease dementia or dementia with Lewy bodies - MEMDLBPDDParkinsons disease (PD)and dementia with Lewy bodies (DLB) are both common disease of the elderly. The majority of patients who survive for more than 10 years with PD eventually develope dementia (PDD). The brain changes and clinical presentations of PDD and DLB are similar. The clinical characteristics are cognitive impairment with pronounced visuospatial and executive impairment, visual hallucinations, parkinsonism, fluctuations in cognition and REM sleep disorder.
EUCTR2005-004109-27-SEeuropsychiatric clinic60
已完成
不适用
A Phase I Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AZD3152 in Healthy Japanese Adults
jRCT2071230040Astrazeneca K.K24
已完成
不适用
A Phase I Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AZD7442 in Healthy Japanese Participants
jRCT2071200111Astrazeneca K.K32
进行中（未招募）
不适用
A Phase I Double-blind, Placebo-controlled Study to Evaluate theSafety, Tolerability and Pharmacokinetics of AZD5148 in HealthyJapanese AdultsClostridioides difficile Infection
jRCT2071240071Astrazeneca K.K16