NL-OMON45765撤回2 期
An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients - SOBI003_001
Swedish Orphan International0 个研究点目标入组 1 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 入组人数
- 1
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
- 年龄范围
- 2 至 11（—）
入选标准
- •1. Informed consent obtained from the patient*s legally authorized representative(s)
- •2. Patients with MPS IIIA, as confirmed by both:
- •- A documented deficiency in sulfamidase enzyme activity in concordance with a diagnosis of MPS IIIA*, and
- •-Normal enzyme activity level of at least one other sulfatase measured in leukocytes
- •3. Chronological age of >=12 and <=72 months (i.e., 1 to 6 years) at the time of the first SOBI003 infusion and has a developmental age >=12 months at screening as assessed by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
- •4. Medically stable patient who is expected to be able to comply with study procedures;* as determined by the reference ranges applied by the Greenwood Genetic Center
排除标准
- •1. At least one S298P mutation in the SGSH gene
- •2. Contraindications for anesthetic procedures, surgical procedure (venous access port) MRI
- •scans and/or lumbar punctures
- •3. History of poorly controlled seizures
- •4. Patients is currently receiving psychotropic or other medications which in the investigator's opinion, would be likely to substantially confound test results
- •5. Significant non-MPS IIIA-related CNS impairment or behavioral disturbances, which in the investigator's opinion, would confound the scientific integrity or interpretation of study assessments
- •6. Prior administration of stem cell or gene therapy, or ERT for MPS IIIA
- •7. Concurrent or prior (within 30 days of enrolment into this study) participation in a study
- •involving invasive procedures
研究者
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