Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
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
This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.
(Show More)
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
Clinical Publications
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome
Intracerebral Gene Therapy in Four Children with Sanfilippo B Syndrome: 5.5-Year Follow-Up Results
News and Press Releases
uniQure Completes Strategic Review to Refocus its Pipeline, Reduce Operating Costs and Deliver Long-Term Shareholder Value
Preclinical Publications
Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes