NCT06226662尚未招募2 期
A Phase II, Randomized, Double-Blind, Placebo-Controlled Study of NM8074 in Patients with Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV)
NovelMed Therapeutics0 个研究点目标入组 12 人开始时间: 2026年6月最近更新:
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 12
- 主要终点
- Proportion of subjects achieving disease response at Day 85 defined as BVAS percent decrease of at least 50% from baseline.
研究概览
简要总结
This is a Randomized, Double-Blind, Placebo-Controlled Study designed to assess safety, tolerability, and efficacy of NM8074 in AAV patients when used in combination with Standard of Care (SOC) cyclophosphamide/azathioprine or rituximab plus corticosteroids.
详细描述
The proposed study, NM8074-AAV-501, will initially assign six (6) patients per cohort in a 2-cohort trial. In the first cohort, we will evaluate a biweekly dosing regimen with NM8074 and SOC whereas in the second cohort, we will evaluate placebo with SOC. These studies will assess safety, tolerability, and efficacy of NM8074 in AAV patients when used in combination with SOC cyclophosphamide/azathioprine or rituximab plus corticosteroids.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
This is a double-blind study (subjects and on-site medical/nursing staff at the study site are blinded to study drug/dose assignment). The pharmacy staff preparing the investigational products will not be blinded to NM8074 study drug assignment, but all other site staff, including the Investigator, will be blinded. Unblinding should only be considered for the safety of the subject.
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
- •Clinical diagnosis of granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), or renal limited vasculitis.
- •Male and female subjects aged at least 18 years, with newly diagnosed or relapsed Associated Vasculitis (AAV) where treatment with cyclophosphamide or rituximab is needed.
- •At least 1 major item, or at least 3 non-major items, or at least the 2 renal items of proteinuria and hematuria on Birmingham Vasculitis Activity Score (BVAS).
- •Estimated glomerular filtration rate (eGFR) ≥ 20 mL/ minute.
- •Positive ANCA Test: indirect immunofluorescence (IIF) test for P-ANCA or C-ANCA, or positive ELISA test for anti-proteinase-3 (PR3) or anti-myeloperoxidase (MPO) at Screening.
- •All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-
- •Meningitis B (MenB) meningococcal serogroup B vaccine (Bexsero®) will be administered per local guidelines. If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics. Patients will also be required to have confirmation or administration of vaccination against S. pneumoniae and H. influenzae.
- •Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and complying with the study visit schedule.
- •Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for at least 8 weeks after stopping the investigational drug, and for at least 6 months after the last cyclophosphamide dose (if receiving cyclophosphamide) and at least 12 months after the last rituximab dose (if receiving rituximab).
- •Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to refrain from donating sperm for the duration of the study.
排除标准
- •Severe disease as determined by alveolar hemorrhage, hemoptysis, rapid onset mononeuritis multiplex or central nervous system involvement.
- •Patients with rapidly progressive glomerulonephritis
- •Any other known multi-system autoimmune disease including eosinophilic granulomatosis with polyangiitis (EGPA, Churg Strauss), systemic lupus erythematosus, Immunoglobulin A (lgA) vasculitis (HenochSchönlein purpura), rheumatoid vasculitis, Sjogren's disease, anti
- •glomerular basement membrane disease, or cryoglobulinemia.
- •Required dialysis or plasma exchange within 12 weeks prior to screening.
- •Have a kidney transplant or disease.
- •Received cyclophosphamide within 12 weeks prior to screening; if on azathioprine, mycophenolate mofetil or methotrexate at the time of screening, these drugs must be withdrawn prior to receiving the cyclophosphamide or rituximab dose on Day
- •Received intravenous glucocorticoids, >3000 mg methylprednisolone equivalent, within 4 weeks prior to screening.
- •Have been taking an oral daily dose of a glucocorticoid of more than 10 mg prednisone-equivalent for more than 6 weeks continuously prior to screening.
- •Received rituximab or other B-cell antibody within 52 weeks of screening or 26 weeks provided B cell reconstitution has occurred (i.e., Cluster of Differentiation 19 (CD19) count > 0.01x10^9/L); received anti-tumor necrosis factor (TNF) treatment or other complement inhibitor treatment within 12 weeks prior to screening.
- •Currently or previously under other complement inhibitor treatments less than 3 months prior to study Day
- •Patients who need the initiation of renal replacement therapy within 7 days
- •Have any other clinically significant abnormal laboratory value in the opinion of the investigator.
- •History of bone marrow, hematopoietic stem cell, or solid organ transplantation.
- •History of currently active primary or secondary immunodeficiency.
- •Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections.
- •Pregnant, planning to become pregnant, or nursing female subjects.
- •Females who have a positive pregnancy test result at Screening or on Day 1.
研究组 & 干预措施
NM8074
Experimental
6 subjects will receive a biweekly dose of 20 mg/kg of NM8074 plus SOC (cyclophosphamide/azathioprine or rituximab plus corticosteroids)
干预措施: NM8074 (Drug)
Placebo
Placebo Comparator
6 subjects will receive a biweekly dose of placebo plus SOC (cyclophosphamide/azathioprine or rituximab plus corticosteroids)
干预措施: Placebo (Drug)
结局指标
主要结局
Proportion of subjects achieving disease response at Day 85 defined as BVAS percent decrease of at least 50% from baseline.
时间窗: Up to Study Day 85
次要结局
- Number of patients with ANCA positivity (anti-PR3 and anti-MPO)(Up to Study Day 133)
- Change from Baseline or Percent Change from Baseline in BVAS (Birmingham Vasculitis Activity Score)(Up to Study Day 133)
- Change from Baseline or Percent Change from Baseline in Vasculitis Damage Index (VDI)(Up to Study Day 133)
- Change from Baseline or Percent Change from Baseline in C-reactive protein concentration(Up to Study Day 133)
- Proportion of patients requiring rescue glucocorticoid treatment(Up to Study Day 133)
- Proportion of patients achieving disease remission on Day 85 defined as BVAS of 0.(Up to Study Day 85)
- Percent Change from Baseline in urinary Monocyte Chemoattractant Protein-1 (MCP-1):creatinine ratio(Up to Study Day 133)
- proportion of subject achieving renal response at Day 85(Up to Study Day 85)
- Change from Baseline or Percent Change from Baseline in quality of life (QoL) Assessed via the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale, Version 4.(Up to Study Day 133)
- Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) Assessed via the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale (QLQ- C30), Version 3.0(Up to Study Day 133)
- Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) assessed via EQ-5D-5L(Up to Study Day 133)
- Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) assessed via SF-36v2(Up to Study Day 133)
研究者
相似试验
已完成
2 期
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Twice Daily Topical Applications of AP611074 5% Gel for up to 16 Weeks in Condyloma PatientsCondylomaNCT02724254Vaxart218
已完成
2 期
Study to Assess the Safety, Tolerability and Preliminary Efficacy of NLX-112 Versus Placebo in L-dopa-induced DyskinesiaMedication-Induced DyskinesiaNCT05148884Neurolixis SAS27
已完成
2 期
A Phase 2, Double-blind, Placebo-controlled Study of the Efficacy and Safety of Belumosudil in Subjects With Moderate/Severe Chronic Plaque PsoriasisChronic Plaque PsoriasisNCT02852967Kadmon Corporation, LLC110
已完成
2 期
Safety, Tolerance and Activity of HE3286 in Patients With Type 2 Diabetes MellitusType 2 Diabetes MellitusNCT00694057Harbor Therapeutics164
已完成
1 期
Safety, Tolerability and Potential Efficacy of AVT001 in Patients With Type 1 DiabetesType 1 Diabetes MellitusNCT03895996Avotres Inc.25