EUCTR2011-004410-42-FR进行中（未招募）不适用
A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA cDNA to children with metachromatic leukodystrophy - Intracerebral Gene Therapy for MLD
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- Inserm
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •- 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
排除标准
- •- 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
研究者
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