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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II


NCTID NCT05665166 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Mucopolysaccharidosis II
Disease Ontology Term DOID:12799
Compound Name AVR-RD-05
Compound Description Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII
Sponsor University of Manchester
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 5
Results Posted Not Available

Therapy Information


Target Gene/Variant IDS
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-12-07
Completion Date 2027-09
Last Update 2024-10-02

Participation Criteria


Eligible Age 3 Months - 22 Months
Standard Ages Child
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations United Kingdom

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates AVROBIO supported this IIT, Program development was halted in July 2023

Resources/Links