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Clinical Trial Report

Gene Therapy Trial Report

Summary

Intracerebral Gene Therapy for Children with Early Onset Forms of Metachromatic Leukodystrophy


NCTID NCT01801709 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Metachromatic Leukodystrophy
Disease Ontology Term DOID:10581
Compound Name AAVrh.10-CAG/cuARSA
Sponsor Institut National de la Santé Et de la Recherche Médicale, France
Funder Type Other gov
Recruitment Status
Completed
Enrollment Count 5
Results Posted Not Available

Therapy Information


Target Gene/Variant ARSA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracerebral
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAVrh.10
Editor Type
Dose 1 1E12 vg
Dose 2 4E12 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2013-01-28
Completion Date 2022-12-20
Last Update 2025-02-04

Participation Criteria


Eligible Age 6 Months - 5 Years
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 INSERM is developing a new therapy with a different AAV (PMID: 34093126)

Resources/Links