NCT06149403进行中（未招募）3 期
A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects With Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care With Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
Orchard Therapeutics10 个研究点 分布在 4 个国家目标入组 41 人开始时间: 2023年12月11日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 41
- 试验地点
- 10
- 主要终点
- Event-free survival
研究概览
简要总结
A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).
详细描述
The study is a multi-center, randomized, active controlled clinical trial designed to evaluate the efficacy and safety of OTL-203 in patients with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT). A total of 40 patients with a confirmed diagnosis of MPS-IH who meet the study inclusion criteria will be randomized to receive either OTL-203 or allo-HSCT. The trial will comprise of a screening, baseline, and treatment period, with a follow-up period of 5 years post-treatment, and primary analysis performed at 2 years follow-up of the last treated subject.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 28 Days 至 30 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Norm-referenced cognitive standard score of ≥70 measured by age-appropriate cognitive domains of either Bayley Scale of Infant Development (BSID)-III or Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-IV.
- •Confirmed laboratory diagnosis of MPS-IH as demonstrated by biallelic mutation(s) in the gene coding for IDUA enzyme
- •Final confirmation of MPS-IH diagnosis by a Diagnostic Review Committee (DRC).
排除标准
- •Previous allo-HSCT or gene therapy
- •Current enrollment or past treatment in any other interventional study/trial using a novel investigational agent and/or treated with prohibited medications listed in the protocol
- •Positivity to serological testing for Human Immunodeficiency Virus (HIV)-1 or HIV-2, Human T Lymphotropic Virus (HTLV)-1 or HTLV-2, Hepatitis B Virus (HBV) core, Hepatitis C Virus (HCV), mycoplasma, active tuberculosis (TB) and not meeting the microbiology biological screening requirements.
- •Malignant neoplasia (except local skin cancer).
- •Myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)
- •History of uncontrolled seizures
- •Subjects with an active infection not responsive to treatment, end-organ damage, or any other disease that contraindicates performance of any of the procedures detailed in the protocol, or medical conditions or extenuating circumstances that, in the opinion of the Investigator, might compromise the subject's well-being or safety, or the interpretability of the subject's clinical data.
- •Subjects, who in the opinion of the Investigator, may not be able to comply with protocol requirements or cooperate fully with the study procedures and necessary long-term follow up
结局指标
主要结局
Event-free survival
时间窗: 2 years
Defined by events of death, rescue transplant, treatment failure, immunological complications, severe cognitive and/or growth impairment.
次要结局
- Change from baseline to Year 2 in α-L-iduronidase (IDUA) activity in leukocytes(Day 30 and multiple visits up to 5 years post-treatment)
- Change from baseline to Year 2 in the ratio to the upper limit of normal (ULN) of urinary heparan sulfate levels(Day 30 and multiple visits up to 5 years post-treatment)
- Safety of OTL-203 compared to allo-HSCT procedure(Up to 5 years post-treatment)
- Malignancy or abnormal clonal proliferation (ACP) using different tests and procedures (e.g., general clinical evaluation, blood counts, and specialized assessments such as integration site analysis).(Up to 5 years post-treatment)
- Replication Competent Lentivirus (RCL)(Up to 5 years post-treatment)
- Immune response against IDUA enzyme(Up to 5 years post-treatment)
研究者
研究点 (10)
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