An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 4
- 主要终点
- Incidence and severity of adverse events
研究概览
简要总结
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement
详细描述
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement
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
- None
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 or female ≥ 18 to ≤65 years of age
- •Pathogenic GLA mutation consistent with Fabry Disease
- •Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement
- •Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat
- •Individuals receiving ERT, must be on a stable dose for at least 6 months
- •Agree to use highly effective contraception
排除标准
- •Presence of pre-existing antibodies to 4D-310 capsid or to AGA
- •eGFR <65 mL/min/1.73 m2
- •Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
- •HIV, active or chronic hepatitis B or C,
- •Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control
- •History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)
- •Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
- •Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.
- •Moderately severe to severe cardiovascular disease or uncontrolled hypertension
- •Left ventricular ejection fraction of <45% on echocardiogram (ECHO)
- •Currently receiving investigational drug, device or therapy or having ever received gene therapy
- •History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation
- •History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)
- •Pregnant or breast-feeding
- •Note: Other inclusion and exclusion criteria apply
研究组 & 干预措施
4D-310 Dose Level 1 (No longer enrolling)
No longer enrolling - Single IV administration of 4D-310 at Dose Level 1
干预措施: 4D-310 (Biological)
4D-310 Dose Level -1
Single IV administration of 4D-310 Dose Level -1
干预措施: 4D-310 (Biological)
4D-310 Dose Level 2
Single IV administration of 4D-310 Dose Level 2
干预措施: 4D-310 (Biological)
4D-310 Dose Level 3
Single IV administration of 4D-310 at Dose Level 3
干预措施: 4D-310 (Biological)
结局指标
主要结局
Incidence and severity of adverse events
时间窗: 1 year
Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose
次要结局
未报告次要终点