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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 (ACTUAL)
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
Delivery System Viral transduction
Vector Type AAVrh.10
Dose 1 1E12 vg
Dose 2 4E12 vg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2013-01-28
Completion Date 2022-12-20
Last Update 2026-03-02

Participation Criteria


Eligible Age 6 Months - 5 Years
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Boys or girls with an early onset form of MLD. * Age between 6 months and 5 years, inclusive * Diagnostic of MLD based on the measurement of ARSA activity in leukocytes and the accumulation of sulfatides in urine, along with normal activity of at least one other sulfatase * Informed consent signed up and willingness for monitoring 2 years after treatment. * Normal values for standard laboratory tests Exclusion Criteria: * Absence of ARSA protein by immunocytochemistry and/or ELISA * Gestational age \<32 weeks of amenorrhoea and age \< 1 year * Brain atrophy with a subdural space \> 10 mm in the frontal region * Performance IQ\<50 at WPPSI-III or cognitive function \< 3rd percentile at the Bayley's test of infant development * If age \> 16 months at inclusion, inability to walk few steps alone OR inability to walk few steps with support on one side along with inability to stand up alone * Impossibility for anesthesia * Malignancy, cardiac malformation, liver dysfunction, or renal dysfunction * Neurological disorder, except benign, not related to MLD. * Any other clinically significant untreated co-morbid medical condition as determined by the clinical investigator, including cardiac, pulmonary or kidney disease. * MRI impossibility * Evoked potential impossibility * Participation to another therapeutic clinical trial for MLD. * Unaffiliated to any French or any other National Health Insurance.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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