2023-508000-37-01招募中4 期
GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
适应症
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up
研究概览
简要总结
The objective of this trial it to characterize how muscle glycogen is affected by enzyme replacement therapy in treatment naïve patients with Pompe Disease.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Subjects with genetically verified Late-onset Pompe Disease
- •Naïve to enzyme replacement therapy
排除标准
- •Ferrous objects in or around the body
- •Pacemaker or other implanted electronic devices
- •Claustrophobia
- •Inability to understand the purpose of the trial or corporate for the conduction of the experiments
- •Participation in other trials that may interfere with the results
- •Competing conditions at risk of compromising the results of the study, evaluated by the investigator
结局指标
主要结局
Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up
Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up
次要结局
- Change in glycogen concentration of the calf, hamstring, anterior thigh, and lumbar muscles over time – both within the first treatment cycle, and at 3, 6 and 12-months follow-up
- Change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 3, 6 and 12-months follow-up.
- Correlations between baseline glycogen levels and change in FVC between baseline and 12-months follow-up.
- Correlations between baseline glycogen levels and change in 6MWT between baseline and 12-months follow-up.
- Correlations between baseline glycogen levels and change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 12-months follow-up.
研究者
Mads Stemmerik
Scientific
Rigshospitalet
研究点 (1)
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