Intracerebral Gene Therapy for MLD

Active, not recruiting

Conditions: Early onset forms of MLD
MedDRA version: 14.1Level: SOCClassification code 10029205Term: Nervous system disordersSystem Organ Class: 10029205 - Nervous system disorders
MedDRA version: 14.1Level: PTClassification code 10067609Term: Metachromatic leukodystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1Level: SOCClassification code 10027433Term: Metabolism and nutrition disordersSystem Organ Class: 10027433 - Metabolism and nutrition disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2011-004410-42-FR

Lead Sponsor: Inserm

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

- Boys or girls with an ealry onset form of MLD
- Age between 6 months ans 4 years, inclusive
- Interval between age of first symptoms and age of inclusion muyst be 12 or less months
- 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 > 10mm in the frontal region.
-MLD MRI severity score >14
- Performance IQ<70 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 dysfuncion
- 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 impossibilty
- Evoked potential impossibility
- Participation to another therapeutic clinical trial for MLD
- Unaffiliated to any French health insurance or any other European National Health Insurance