NCT07847411尚未招募3 期
A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy in Children With Gaucher Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Primary Endpoints
研究概览
简要总结
This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.
研究设计
- 研究类型
- 干预性
- 分配方式
- 不适用
- 干预模型
- 单组
- 主要目的
- 治疗
- 盲法
- 开放（无盲法）
入排标准
- 年龄范围
- 2 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- Subject has a documented diagnosis of GD1 or GD3 according to the diagnostic criteria of the Expert Consensus on the Diagnosis and Treatment of Gaucher Disease in Chinese Children 2021;
- Written informed consent provided by the subject's parent/legally authorized representative;
- Subject is less than 12 years of age;
- Subject has GD-related splenomegaly, defined as at least 2 to 3 cm below the left costal margin by palpation, and one or more of the following:
- GD-related anemia, with a decrease of ≥1 g/dL in hemoglobin concentration below the lower limit of normal for age of the central laboratory; or
- GD-related hepatomegaly by palpation; or
- GD-related thrombocytopenia, with a platelet count < 100 × 10^9/L;
- Subject has not received ERT or SRT for at least 3 months prior to screening; Female subjects of childbearing age have a negative serum pregnancy test during screening.
排除标准
- Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening;
- Subjects have received erythropoietin, whole blood transfusion or transfusion of red blood cells, or long-term (continuous treatment for more than 3 months) systemic corticosteroids 3 months prior to screening; or received a platelet transfusion within 1 month prior to screening;
- Subject has non-Gaucher disease-related anemia (such as due to iron, folic acid, and/or vitamin B12 deficiency or infection/immune-mediated reasons);
- Subject has had a prior hepatectomy and/or splenectomy (including partial liver and/or splenectomy) or plans to have a hepatectomy and/or splenectomy (including partial liver and/or splenectomy) during the study;
- Subject has received organ transplantation, including hematopoietic stem cell transplantation;
- Subject has had a history of CTCAE Grade 3 or above infusion-related reaction or hypersensitivity reaction to imiglucerase or other ERTs (approved or experimental).
- Injection with a live vaccine within 30 days of the first study dose.
研究组 & 干预措施
velaglucerase beta 60 U/kg
Experimental
This is a single-arm study. All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks.
干预措施: Velaglucerase beta (Drug)
结局指标
主要结局
Primary Endpoints
时间窗: From Baseline to Week 39
Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase
Mean percent change in MN spleen volume
时间窗: From Baseline to Week 39
Normal spleen volume = 0.2% body weight. Multiples of normal (MN) spleen volume= actual MRI testing volume / normal spleen volume
次要结局
- Mean change in weight Z-score(From Baseline to Week 39)
- Mean change in hemoglobin concentration (g/dL)(From baseline to Week 39)
- Mean percent change in MN liver volume(From Baseline to Week 39)
- Mean percent change in platelet count ( 10^9/L)(From Baseline to Week 39)
- Mean change in height Z-score(From Baseline to Week 39)
研究者
研究点 (1)
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标识符
- NCT 编号
- NCT07847411
- 其他研究编号
- CAN103-GD-301
日期
- 首次提交
- (上个月)
- 首次发布
- (昨天)
- 主要完成日期
- (明年)
- 研究完成日期
- (明年)
- 最近核实
- (29天前)
- 最近更新
- (昨天)
监管与共享
- FDA 监管药物
- 否
- FDA 监管器械
- 否
- 个体参与者数据共享计划
- 是
- 是否有结果
- 否
only IPD used in the results publication
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