EUCTR2021-005200-35-BE进行中（未招募）1 期
A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 54
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Participants aged =2 to <6 years (Cohort A) or =6 to <26 years (Cohort B)
- •2. Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
- •3. For non-run-in Cohort A and Cohort B only: be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months (ie, 16 weeks) of idursulfase therapy during the period immediately prior to screening.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 54
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 6
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Have a documented pathogenic or likely pathogenic variants that are known to cause developmental delay or decline, cognitive dysfunction, seizures, or other significant CNS disorders.
- •2. Previously received an IDS gene therapy or stem cell therapy
- •3. Received any CNS-targeted MPS ERT within 6 months prior to screening
- •4. Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRIs)
- •5. Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study
研究者
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