NCT05230459进行中（未招募）1 期
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- AskBio Inc
- 入组人数
- 9
- 试验地点
- 12
- 主要终点
- Adverse Events
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
研究设计
- 研究类型
- Interventional
- 分配方式
- 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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
- •Ability to walk/run 10 meters in <30 seconds.
- •Able to understand and comply with all study procedures.
- •Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.
排除标准
- •Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction <40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF >480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.
- •Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
- •Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
- •History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin >upper limit of normal [ULN] and/or elevated AST and ALT >2 ULN).
- •Abnormal renal function (GFR <60 ml/min, using the Modification of Diet in Renal Disease equation).
- •Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).
- •In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
- •Requirement for daytime ventilatory support.
- •Change in glucocorticosteroid treatment within 3 months prior to screening visit.
- •Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
- •Ongoing participation in any other therapeutic clinical trial.
- •Neutralizing antibody titer to AAV9 >1:
- •Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
研究组 & 干预措施
Placebo (Cohorts 1 and 2)
Placebo Comparator
干预措施: Placebo (Other)
AB-1003 Cohort 1
Experimental
干预措施: AB-1003 dose level 1 (Genetic)
AB-1003 Cohort 2
Experimental
干预措施: AB-1003 dose level 2 (Genetic)
结局指标
主要结局
Adverse Events
时间窗: 0-52 weeks
Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
次要结局
未报告次要终点
研究者
研究点 (12)
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