2024-519412-13-00尚未招募1/2 期
Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of rAAVOlig001-ASPA Gene Therapy for Treatment of Children with Typical Canavan Disease
适应症
试验速览
- 阶段
- 1/2 期
- 状态
- 尚未招募
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- Change from baseline to Month 6 in CSF NAA concentrations, as measured by biochemical assay.
研究概览
简要总结
Assess the effect of rAAV-OLIG001-ASPA on cerebrospinal fluid (CSF) NAA concentrations in children with Canavan disease as measured by biochemical assay
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Definitive diagnosis of typical CD by a neurologist (see Section 3.2 of protocol for detailed description).
- •Age between 3 and 60 months at the time of neurosurgical administration of rAAV- Olig001-ASPA or over the age of 60 months for untreated patients in Cohort
- •Written informed consent from parent(s)/guardian(s). Consent to enroll into the study will include a written agreement to comply with all the conditions of the study, including attendance at follow-up visits.
- •MRI or CT within the past 3 months, for preliminary neurosurgical planning; if no MRI or CT within 3 months is available, MRI can be done during Screening assuming that the results of all other Inclusion/Exclusion Criteria are acceptable.
排除标准
- •At the discretion of the PI, any significant chronic medical condition, including, but not limited to neurological, cardiac, hepatic, renal, hematological, gastrointestinal, endocrine, pulmonary, or infectious disease, which would put the subject at increased risk during surgery or which would interfere with participation in the study, interpretation of safety monitoring, or the integrity of the study data.
- •History of severe allergic reaction or anaphylaxis.
- •Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment.
- •Any absolute contraindication to immunosuppression.
- •Any absolute contraindication to MRI.
- •Any vaccination less than 1 month prior to gene therapy.
- •Anticipated life expectancy of less than 12 months for any reason.
- •GMFM-88 total raw score >35%.
- •Clinically significant out-of-range lab values as indicated in Appendix G, at the discretion of clinical PI.
结局指标
主要结局
Change from baseline to Month 6 in CSF NAA concentrations, as measured by biochemical assay.
Change from baseline to Month 6 in CSF NAA concentrations, as measured by biochemical assay.
次要结局
- Rate of change from baseline in Gross Motor Function Measure-88 specific domains, comparing FIH gene therapy–treated cohort to an external natural history cohort: - Lying/rolling domain - Sitting domain - Crawling domain - Standing domain - Walking domain
- Rate of change from baseline in multivariate MSEL domains age-equivalent scores compared with an external natural history cohort. MSEL domains include: Visual Reception, Fine Motor, Receptive Language, Expressive Language, Gross Motor
- Rate of change from baseline in univariate MSEL domains age-equivalent scores compared with an external natural history cohort: - MSEL Receptive Language domain - MSEL Expressive Language domain - MSEL Visual Reception domain - MSEL Fine Motor domain - MSEL - Gross Motor domain
- Change from baseline to Month 6 in urine NAA concentrations, as measured by biochemical assays
- Rate of change in myelin volume in the brain as measured by SyMRI through Month 12.
- Rate of change from baseline in Gross Motor Function Measure-88 total score, comparing FIH gene therapy–treated cohort to an external natural history cohort.
研究者
Olga Flamini
Scientific
Myrtelle Inc.
研究点 (1)
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