MedTrace Logo

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

NCT01801709 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-03-02

No results posted yet for this study

Summary

The objective of this open-label, single arm, monocentric, phase I/II clinical study is to assess safety and efficacy of ARSA gene transfer in the brain of children affected with early onset forms of Metachromatic Leukodystrophy (MLD). For this purpose, an adeno-associated virus serotype rh.10 (AAVrh.10) vector will be used to transfer the ARSA cDNA coding for Arylsulfatase A (ARSA) enzyme into the brain of children. Five patients with early onset form of MLD, age ranging from 6 months to 4 years, will be included in this protocol and will be followed during 24 months.

Patients will be selected at presymptomatic or early stage of their disease, following clinical, neuropsychological and brain imaging criteria.

Twelve simultaneous injections of the investigational medicinal product will be performed in the white matter of both brain hemispheres, through 6 image-guided tracks, with 2 deposits per track.

A low dose (1x10EXP12 vg total) will be administered to the first 2 patients, while the last 3 will receive a higher dose (4x10EXP12 vg total).

Safety and efficiency will be evaluated based on clinical, neuropsychological, radiological, electrophysiological and biological parameters.

Conditions

  • Metachromatic Leukodystrophy

Interventions

GENETIC

intracerebral administration of AAVrh.10cuARSA

Sponsors & Collaborators

  • European Leukodystrophy Association

    collaborator OTHER

  • Assistance Publique - Hôpitaux de Paris

    collaborator OTHER

  • Institut National de la Santé Et de la Recherche Médicale, France

    lead OTHER_GOV

Principal Investigators

  • Patrick Aubourg, MD-PhD · Assistance Publique - Hôpitaux de Paris and Institut National de la Santé et de la Recherche Médicale

  • Caroline Sevin, MD-PhD · Assistance Publique - Hôpitaux de Paris

  • Michel Zerah, MD, PhD · Assistance Publique - Hôpitaux de Paris

  • Thomas Roujeau, MD, PhD · Assistance Publique - Hôpitaux de Paris

  • Nathalie Cartier, MD, PhD · Institut National de la Santé et de la Recherche Biomédicale

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-06-30
Primary Completion
2016-06-30
Completion
2022-12-20

Countries

  • France

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01801709 on ClinicalTrials.gov