An open-label, single-arm, multicenter study of intracerebral administration of adeno-associated viral vectors serotype rh.10 carrying the human N-sulfoglycosamine sulfohydrolase (SGSH) cDNA for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA) - P4-SAF-302

ysogene SA0 个研究点目标入组 10 人待定

概览

暂无简介。

注册库

who.int

开始日期

待定

结束日期

待定

最后更新

2年前

研究类型

Interventional

发起方

ysogene SA

•1\. Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene
•2\. Age \>\= 30 months at screening (main cohort) or \>\= 6 months and \< 30 months
•(ancillary cohort)
•3\. Cognitive DQ score on BSID\-III \>\= 50%
•4\. Signed written informed consent before any study related procedure is
•5\. Medical status sufficiently stable, in the opinion of the investigator, to
•adhere to the study visit schedule and other protocol procedures
•6\. Confirmation by the study neurosurgeon and anesthesiologist of the
•feasibility of the neurosurgical procedure.

•1\. Homozygous for the S298P mutation or non\-severe form of MPS IIIA, based on
•investigator\*s judgement
•2\. Past participation in another gene or cell therapy clinical trial
•3\. Past use of SGSH enzyme replacement therapy for a cumulative period
•exceeding 3 months. In addition, a washout period of at least 2 months is
•required prior to screening
•4\. Current participation in a clinical trial of another investigational
•medicinal product. NOTE: Nutritional supplements, including Genistein are
•permitted if they are taken outside the context of an investigational trial
•5\. History of bleeding disorder or current use of medications that, in the

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