Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

• Status: Active, not recruiting
• Trial end date: 2022-03-01
• Target enrollment: 0
• Participant gender: All

Summary

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: LYSOGENE

Criteria

Inclusion Criteria:

• Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations

• Cognitive DQ score on BSID-III: 50% and above

Exclusion Criteria:

• Homozygous for the S298P mutation or non-classical severe form of MPS IIIA, based on investigator's judgement.

• Participation in another gene or cell therapy clinical trial.

• Past use of SGSH enzyme replacement therapy for a period exceeding 3 months. A washout period of at least 2 months is required prior to screening.

• Current participation in a clinical trial of another investigational medicinal product.

• History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following surgery.

• Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids.