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

NCTID	NCT03612869 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Inactive
Indication	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Disease Ontology Term	DOID:0111395
Compound Name	LYS-SAF301
Compound Alias	Olenasufligene relduparvovec
Compound Description	AAVrh.10-CAG-SGSH
Sponsor	LYSOGENE
Funder Type	Industry
Recruitment Status	Unknown
Enrollment Count	20
Results Posted	Not Available

Target Gene/Variant	SGSH
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracerebral
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2rh.10
Editor Type	none
Dose 1	7.2E11 vg (used in Phase 1/2)
Dose 2	7.2E12 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	8
Locations	Netherlands,United States,United Kingdom,France,Germany

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Phase 2/3 study failed primary efficacy endpoint