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.
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.
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.
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Chester Whitley, MD
Phone: 612-625-1594
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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