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

Status unconfirmed · Phase 2/Phase 3

Conditions studied: Mucopolysaccharidosis Type IIIA

In brief

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.

Key facts

Study ID

NCT03612869

Run by

LYSOGENE

People needed

20

Starts

2018-12-17

Expected to finish

2022-03-01

Last updated by the study team

2021-08-31

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
• Cognitive DQ score on BSID-III: 50% and above

You may not qualify if…

• 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.

Where it is running

• CHOC Children's — Orange, California, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Weill Cornell Medical College — New York, New York, United States
• Baylor college of medicine / Texas children's hospital — Houston, Texas, United States
• Armand Trousseau Public Hospital — Paris, France
• University Medical Center Hamburg-Eppendorf — Hamburg, Germany
• Amsterdam UMC — Amsterdam, Netherlands
• Great Ormond Street Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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