Contact SCGE




Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB


NCTID NCT03315182 (View at clinicaltrials.gov)
Description
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