An Open-Label, Single Center, Phase 1/2 Study to Evaluate the Safety and Efficacy of DANAGALEX (scAAV9/AGA) in Participants With Aspartylglucosaminuria (AGU)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 9
- 主要终点
- Safety and tolerability of a single dose of scAAV9/AGA in participants with aspartylglucosaminuria (AGU)
研究概览
简要总结
The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).
详细描述
This study is a first in human (FIH) open-label study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding aspartylglucosaminidase (AGA) in participants with Aspartylglucosaminuria (AGU). Safety will be monitored continuously throughout the study for adverse / serious adverse events and dose limiting toxicities. All outcomes (primary, secondary, exploratory) will be assessed at 52 and 104 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented molecular diagnosis of AGU (e.g., whole exome sequencing, whole genome sequencing, gene panel, single-gene sequencing, or chromosomal microarray) identifying two pathogenic and/or likely pathogenic variants in the AGA gene
- •The participant, or the participant's parent or legal guardian, is registered in the AGU Natural History Study
- •The participant, or the participant's parent, legal guardian, or caregiver are willing and able to travel to the study site and complete all aspects of the study, adhere to the study visit schedule, and comply with all assessments.
排除标准
- •Any prior or ongoing medical condition, clinical history, physical examination finding, cardiovascular or ECG abnormality, or laboratory result that may: (1) place the participant at undue risk during administration; (2) interfere with study treatment or follow-up compliance; or (3) confound the interpretation of study data.
- •Acute illness requiring hospitalization within 6 weeks prior to Screening
- •Contraindications to or unwillingness to undergo MRI, lumbar puncture (LP) or other study procedures;
- •Chronic requirement for respiratory support, including invasive or non-invasive ventilation;
- •Known bleeding disorders (e.g., hemophilia, von Willebrand disease) or any medical condition or treatment associated with increased bleeding risk;
- •Prior treatment with a gene, cell therapy, or bone marrow replacement;
- •Treatment with any investigational product (IP) within 90 days or 5 half-lives of the IP, whichever is longer, prior to screening period;
- •Any condition that in the opinion of the investigator or the study medical monitor would prevent the patient from fully complying with the requirements of the study (including the corticosteroid treatment) and/or would impact or interfere with the evaluation and interpretation of patient safety or efficacy results.
研究组 & 干预措施
Treatment
Cohort 1: 18+ years old, Cohort 2: 12-17 years old, then Cohort 3: 4-11 years old
干预措施: scAAV9/AGA (Genetic)
结局指标
主要结局
Safety and tolerability of a single dose of scAAV9/AGA in participants with aspartylglucosaminuria (AGU)
时间窗: Through Day 720
Incidence, severity, and causality of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and adverse events of special interest (AESIs)
次要结局
- Biologic activity following a single dose of scAAV9/AGA in participants with aspartylglucosaminuria (AGU)(Days 360 and 720)
- Preliminary efficacy following a single dose of scAAV9/AGA in participants with aspartylglucosaminuria (AGU)(Days 360 and 720)