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

Status:	Recruiting
Conditions:	Other Indications, Metabolic
Therapuetic Areas:	Pharmacology / Toxicology, Other
Healthy:	No
Age Range:	Any
Updated:	3/13/2019
Start Date:	December 17, 2018
End Date:	January 2022
Contact:	Lysogene trial inquiries
Email:	patientadvocacy@lysogene.com
Phone:	+33 (0) 1 41 43 03 90

Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

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.

The study is interventional, single arm and multi-center. Evolution under treatment will be
compared to expected natural evolution based on natural history studies.

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.

We found this trial at

sites

Baylor College of Medicine/Texas Children's Hospital

Houston, Texas

Phone: 832-825-1717

from

Houston, TX