NL-OMON56350招募中2 期
A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy - NMD670/placebo in ambulatory adults with SMA.
MD Pharma0 个研究点目标入组 5 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 5
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
入选标准
- •1. Participant must be 18 to 75 years of age inclusive, at the time of signing
- •the informed
- •2. Participants who are with a clinical diagnosis of Type 3 SMA.
- •3. Participants who are ambulatory, defined as being able to walk at least 50
- •metres without
- •walking aids.
- •4. Participant with genetic confirmation of diagnosis (i.e., homozygous
- •deletion of survival of
- •motor neuron 1 gene [SMN1]).
- •5. Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
- •6. Participants with a MFM-32 dimension 1 score <80% at screening.
- •7. Participants with >=7% CMAP amplitude decrement at screening (recorded from
- •the trapezius
- •muscle during repeated nerve stimulation [RNS]).
排除标准
- •1. Participants with prior surgery or fixed deformity (scoliosis, contractures)
- •which would
- •restrict ability to perform study-related tasks.
- •2. Participants with other significant disease that may interfere with the
- •interpretation of study
- •data (e.g., other neuromuscular or muscular diseases).
- •3. Participant with a clinical diagnosis of gout, or with serum uric acid >6.5
- •mg/dL at screening.
- •4. Participant with clinically significant ECG abnormalities at screening
- •including PR interval
- •>=220 msec, irregular rhythms (other than sinus arrhythmia or occasional, rare
- •supraventricular or rare ventricular ectopic beats) in the judgement of the
- •Investigator, or
- •T-wave configurations are not of sufficient quality for assessing QT interval
研究者
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