An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA for the Treatment of Mucopolysaccharidosis Type IIIA

ysogene SA0 sites20 target enrollmentSeptember 19, 2018

Overview

No summary available.

Registry

who.int

Start Date

September 19, 2018

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

ysogene SA

•1\. Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
•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 performed
•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.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 20
•F.1\.2 Adults (18\-64 years) no
•F.1\.2\.1 Number of subjects for this age range

•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 opinion of the investigator, place them at risk of bleeding following surgery
•6\. Presence of concomitant medical condition precluding lumbar puncture
•7\. Presence of any item (e.g., metal braces) precluding undergoing MRI
•8\. Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids
•9\. History of significant non\-MPS IIIA related CNS impairment or behavioral disturbances that would confound scientific rigor or interpretation of results