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Clinical Trial Report

Gene Therapy Trial Report

Summary

Phase I/II/III Gene Transfer Clinical Trial of ScAAV9.U1a.hSGSH

NCTID NCT02716246 (View at clinicaltrials.gov)
Description
Indication MPS IIIA, Sanfilippo Syndrome, Sanfilippo a, Mucopolysaccharidosis III
Compound Name Rebisufligene etisparvovec (scAAV9.U1a.hSGSH)
Sponsor Ultragenyx Pharmaceutical Inc
Funder Type Industry
Status
Recruiting
Enrollment Count 36

Therapy Information

Target Gene/Variant SGSH
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 0.5E13 vg/kg
Dose 2 1.0E13 vg/kg
Dose 3 3.0E13 vg/kg
Dose 4
Dose 5

Study Record Dates

Current Stage Phase2, Phase3
Submit Date 2016-03-17
Completion Date 2027-07
Last Update 2025-02-19

Participation Criteria

Eligible Age
Standard Ages Child, Adult, Older adult
Eligible Sex ALL

Locations

No.of Trial Sites 5
Locations United States,Australia,Spain

Regulatory Information

Has US IND True
Recent Updates Accelerated Approval BLA filing planned for 2024/early 2025

Resources/Links

News and Press Releases
Preclinical Publications