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: 2024年4月15日

研究概览

暂无简介。

•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

•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

发起方

Swedish Orphan International