NCT05492799Enrolling By Invitation4 期
Open-Label Multicenter Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients
Allievex Corporation6 个研究点 分布在 5 个国家目标入组 15 人开始时间: 2022年12月2日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 15
- 试验地点
- 6
- 主要终点
- Primary - neurocognition
研究概览
简要总结
This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144 weeks) of treatment with AX 250 administered by ICV infusion every other week. Subjects will be evaluated for neurocognitive function, communication, adaptive behavior, quality of life, imaging characteristics and biochemical markers of disease burden. Safety will be assessed by adverse events, clinical labs, and physical exams.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have completed 240 weeks of Study 250-202 and enter 250-401 within 8 weeks of dosing completion.
- •Provides written informed consent from parent or legal guardian and assent from subject, if required
- •Has the ability to comply with protocol requirements in the opinion of the investigator
- •If female with childbearing potential, must have a negative pregnancy test at the Screening visit and be willing to have additional pregnancy tests during the study
排除标准
- •Has (1) a cognitive age equivalent (AEq ) score ≤ 18 months, (2) a development quotient (DQ) score ≤ 20, and (3) no evidence of improvement during the 250-202 study in secondary or exploratory efficacy endpoints
- •Would not benefit from enrolling in the study in the opinion of the investigator
- •Has received stem cell, gene therapy or enzyme replacement therapy (other than AX 250) for MPS IIIB
- •Has a history of poorly controlled seizure disorder
- •Is prone to complications from ICV drug administration including patients with hydrocephalus or ventricular shunts
- •Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study
- •Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's well-being or safety, or the interpretability of the subject's clinical data
研究组 & 干预措施
single arm
Experimental
AX 250 300 mg - open label
干预措施: AX 250 (Drug)
结局指标
主要结局
Primary - neurocognition
时间窗: baseline to 144 weeks of treatment
the rate of change in score from neurocognitive assessment using validated scale
次要结局
未报告次要终点
研究者
研究点 (6)
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