NCT07715084尚未招募2 期
96-week, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
Azafaros B.V.7 个研究点 分布在 3 个国家目标入组 20 人开始时间: 2026年9月28日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 20
- 试验地点
- 7
- 主要终点
- Change in hemoglobin levels from Baseline
研究概览
简要总结
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a confirmed diagnosis of GD3 disease
- •Have at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening
- •Be aged 4-30 years old at the time of informed consent
- •Have the following symptoms of GD3 identified at Screening: GD-related anemia, thrombocytopenia, splenomegaly
- •Female participants of childbearing potential who are sexually active and willing to follow contraceptive guidance
- •Male participants with a female partner of childbearing potential who are willing to follow contraceptive guidance
排除标准
- •Any condition at Screening or Baseline that, in the opinion of the Principal Investigator, could interfere with study assessments
- •History of medical conditions other than GD3 that, in the opinion of the Principal Investigator, could confound scientific rigor or interpretation of results
- •Body weight <10 kg at Screening
- •Presence of a neurologic disease other than GD3
- •Transfusion dependence
- •Prior splenectomy
- •The presence of severe renal impairment at Screening
- •Prior use of an investigational drug within 3 months before Screening
- •Prior participation in a clinical study involving gene therapy or stem cell transplantation
- •Current treatment, or treatment in the last 12 months before Screening, with enzyme replacement therapy (ERT) and/or substrate reduction therapy (SRT)
- •A positive serum pregnancy test (for women of childbearing potential)
- •Electrocardiogram (ECG) with an average Fridericia-corrected QT interval (QTcF) of >450 msec for males and >470 msec for females at Screening
- •Known allergies to azasugars or any study drug excipient
结局指标
主要结局
Change in hemoglobin levels from Baseline
时间窗: 24 weeks
Change in platelet count from Baseline
时间窗: 24 weeks
Change in total spleen volume from Baseline
时间窗: 24 weeks
Assessed using magnetic resonance imaging (MRI)
次要结局
- Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylsphingosine (glcSph)(24 weeks)
- Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylceramide (GlcCer) C16:0 and C18:0(24 weeks)
- Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline(24 weeks)
- Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline(24 weeks)
- Clinical Global Impression of Change (CGI-C) score(24 weeks)
- Participant/Caregiver Global Impression of Change (PGI-C) score(24 weeks)
- Peak plasma concentration (Cmax) of Nizubaglustat(24 weeks)
- Area under the plasma concentration versus time curve (AUC0-24) for Nizubaglustat(24 weeks)
研究者
研究点 (7)
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