NCT05682144招募中1 期
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
Immusoft of CA, Inc.2 个研究点 分布在 1 个国家目标入组 11 人开始时间: 2023年4月12日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 11
- 试验地点
- 2
- 主要终点
- Number of participants with treatment-related adverse events and serious adverse events
研究概览
简要总结
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
详细描述
This is a Phase 1, first-in-human, open-label, single-arm study in which patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system (ISP-001). This study will evaluate the safety and tolerability of ISP-001.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
- •Age ≥ 10 years at time of study registration.
- •Creatinine clearance, calculated or measured directly, that is >60ml/min/1.73m
- •Ejection fraction ≥ 40% by echocardiogram.
- •Must commit to traveling to the study site for the necessary follow-up evaluations.
- •Must agree to stay <45-minute drive from the study site for a minimum of 5 days after cell infusion.
排除标准
- •Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations.
- •History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.
- •Evidence of active graft-vs-host disease.
- •Underwent a previous hematopoietic stem cell transplant (HSCT).
- •Requirement for systemic immune suppression.
- •Requirement for continuous supplemental oxygen.
- •Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment.
- •In the investigator's judgement, the subject is unlikely to complete all protocol-required study visits or procedures, including follow up visits, or comply with the study requirements for participation.
- •Other protocol defined inclusion/exclusion criteria may apply.
结局指标
主要结局
Number of participants with treatment-related adverse events and serious adverse events
时间窗: 24 Weeks
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
次要结局
- Determination of Absolute Numbers of B and T cell populations(1Year)
- Assessment of Storage Material (glycosaminoglycan, or GAG)(1 Year)
- Number of participants with treatment-related adverse events and serious adverse events(48 Weeks)
- Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)(1 Year)
- Concentration of IDUA(1 Year)
- Analysis of PBMCs(1 Year)
研究者
研究点 (2)
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