Status:
COMPLETED
Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Lead Sponsor:
Institut National de la Santé Et de la Recherche Médicale, France
Collaborating Sponsors:
European Leukodystrophy Association
Assistance Publique - Hôpitaux de Paris
Conditions:
Metachromatic Leukodystrophy
Eligibility:
All Genders
6-5 years
Phase:
PHASE1
PHASE2
Brief 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 ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 20 2022
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT01801709
Start Date
June 1 2014
End Date
December 20 2022
Last Update
November 18 2025
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Bicêtre Hospital - Paris Sud
Le Kremlin-Bicêtre, France