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Clinical Trial Report

Gene Therapy Trial Report

Summary

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome


NCTID NCT03300453 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Sanfilippo Syndrome B
Disease Ontology Term DOID:0111394
Compound Name AMT-110
Compound Description rAAV2.5-hNAGLU
Sponsor UniQure Biopharma B.V.
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 4
Results Posted Not Available

Therapy Information


Target Gene/Variant NAGLU
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2/5
Editor Type none
Dose 1 Total: 4E12 vg (960uL) ; 16 sites: 2.4E11 vg/site
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2016-06-15
Completion Date 2019-11-27
Last Update 2019-12-02

Participation Criteria


Eligible Age 18 Months - 60 Months
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND
FDA Designations
Recent Updates UniQure announced discontinuation in November 2016

Resources/Links