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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
NCTID
NCT03315182
(View at clinicaltrials.gov)
Description
Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein
(Show More)
Development Status
Inactive
Indication
Mucopolysaccharidosis Type 3 B
Disease Ontology Term
DOID:0111394
Compound Name
ABO-101
Compound Description
rAAV9.CMV.hNAGLU
Sponsor
Abeona Therapeutics, Inc
Funder Type
Industry
Recruitment Status
Terminated
Enrollment Count
11
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
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV9
Editor Type
Dose 1
2E13 vg/kg
Dose 2
5E13 vg/kg
Dose 3
1E14 vg/kg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2017-10-10
Completion Date
2022-04-07
Last Update
2022-05-05
Participation Criteria
Eligible Age
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
4
Locations
United States,France,Germany,Spain
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Abeona decided to discontinue this program due to lack of drug supply
Resources/Links
News and Press Releases
Abeona Therapeutics Announces Strategy Update and 2021 Financial Results
ABO-101 Gene Therapy | MPS IIIB | Phase I-II | Abeona
(Abstract 675) A Phase 1/2 Clinical Trial of Systemic Gene Transfer of rAAV9.CMV.hNAGLU for MPS IIIB: Safety, Tolerability, and Preliminary Evidence of Biopotency - ASGCT 2018
Preclinical Publications
Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery
Related NCTID
Long Term Follow-Up: NCT04655911