CTIS2024-511970-66-00进行中（未招募）1 期
A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA, for the treatment of early onset Metachromatic Leukodystrophy (MLD) - 205756
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Documented biochemical and molecular diagnosis of MLD, based on ARSA activity below the normal range and identification of two disease-causing ARSA alleles, either known or novel mutations. Novel mutations will be analyzed with in silico prediction tools and excluded from being known common polymorphisms. In the case of a novel mutation(s), a 24-hour urine collection must show elevated sulfatide levels., Eligible participants must have EITHER: a) an older sibling affected by MLD (index case), whose age of symptom onset was =6 years of age (i.e. had not celebrated 7th birthday). Participants will be classified as Late Infantile, Early Juvenile or Intermediate LI/EJ based on age of symptom onset in the index case and their ARSA genotype: i. LI: symptom onset in index case =30 months of age; genotype typically 0/0 ii. EJ: symptom onset in index case >30 months and =6 years of age; genotype typically 0/R iii. Intermediate LI/EJ: symptom onset in index case =6 years of age but unable to unambiguously characterize index case as LI or EJ OR b) If MLD is diagnosed in a pre-symptomatic child without an older affected sibling, (e.g. incidentally or via newborn screening) and the totality of the data available to the investigator strongly suggest that the patient has an early onset variant of MLD likely to benefit from gene therapy, and the patient is =6 years of age (i.e. has not celebrated 7th birthday), the patient may be considered eligible after discussion and approval by the Orchard Therapeutics (Europe) Ltd. Medical Monitor (Orchard-MM)
排除标准
- •If LI MLD variant, clinical manifestations of the disease defined as EITHER of the following: i. Delay in expected achievement of independent standing or independent walking, together with abnormal signs at neurological evaluation OR ii. Documented neurological signs and symptoms of MLD associated with cognitive, motor, or behavioral functional impairment or regression (substantiated by neurological examination and/or neuropsychological tests appropriate for age), If EJ MLD variant, symptoms of MLD resulting in the loss of capacity of walking independently as defined by a GMFC level =2 or symptoms consistent with cognitive impairment as defined by an IQ<85 using age-appropriate neurocognitive instruments
研究者
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