EUCTR2023-000785-33-Outside-EU/EEA进行中（未招募）1 期
A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects with Propionic or Methylmalonic Acidemia Followed by a 6-Month, Randomized, Double-blind, Placebo-controlled, 2-Period Crossover Study and an Open-label, Long-term Extension Study
HemoShear Therapeutics0 个研究点开始时间: 2023年8月18日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •* Confirmed diagnosis of symptomatic PA or MMA (Mutase)
- •* Ages = 2 years old.
- •* History of Inadequate metabolic control while receiving standard of care (SoC).
- •* Plasma MCA concentration > 3x upper limit of normal of the reference range at screening.
- •* Stable supplementation dose of carnitine for at least 1 week prior to the entry in the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 8
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 4
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •* Moderate-to-severely impaired cardiac function with LVEF < 40% by ECHO.
- •* Clinically significant arrhythmia by Holter monitor.
- •* QTcF > 450 msec
- •* Moderate to severe chronic kidney disease with estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2.
- •* Exposure to any investigational therapy within the past 6 months prior to study entry.
- •* Exposure to gene therapy for PA or MMA at any time prior to study entry.
- •* History of organ transplantation
- •* History of severe allergic or anaphylactic reactions to any of the components of HST5040.
研究者
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