A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
- 试验地点
- 3
- 主要终点
- Safety and tolerability
研究概览
简要总结
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
详细描述
Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.
This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.
Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.
All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Female and male ambulant patients
- •2. Patients ≥ 16 years old
- •3. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- •4. Moderate diaphragmatic muscle impairment
排除标准
- •1. Detectable serum neutralizing antibodies against AAV9
- •2. Cardiomyopathy
研究组 & 干预措施
Cohort 1
Cohort 1: single intravenous injection 9.0E+12 vg/Kg
干预措施: ATA-100 (AAV9 encoding FKRP gene) (Biological)
Cohort 2
Cohort 2: single intravenous injection 2.7E+13 vg/Kg
干预措施: ATA-100 (AAV9 encoding FKRP gene) (Biological)
结局指标
主要结局
Safety and tolerability
时间窗: Baseline through 12 months
Incidence of Adverse Events and significant laboratory changes
次要结局
- Combined endpoint (global testing)(Baseline through 12 months)
- Timed Up and Go (TUG) test(Baseline through 12 months)
- Change from baseline in velocity as measured by 10MWT(Baseline through 12 months)
- 2-minute walk distance test(Baseline through 12 months)
- Cardiac MRI(Baseline through 12 months)
- Muscle MRI(Baseline through 12 months)
- Muscle Biopsy(Baseline through 12 months)
- Patient reported outcome and quality of life assessment(Baseline through 12 months)