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Gene Therapy Trial Report

Summary

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


NCTID NCT03612869 (View at clinicaltrials.gov)
Description
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 (ESTIMATED)
Results Posted Not Available

Therapy Information


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
Delivery System Viral transduction
Vector Type AAV2rh.10
Dose 1 Phase 1/2: 7.2E11 vg (n=4)
Dose 2 Phase 2/3: 7.2E12 vg

Study Record Dates


Current Stage Phase2, Phase3
Submit Date 2018-07-04
Completion Date 2022-03
Last Update 2021-08-31

Participation Criteria


Eligible Age >=6 Months
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


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

Regulatory Information


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

Resources/Links