2024-512828-12-00招募中4 期
An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study)
适应症
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 26
- 试验地点
- 10
- 主要终点
- Number of vertebral fractures per 1000 person-years based on X- ray central reading
研究概览
简要总结
To evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fractures
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Subject and/or subject’s parent(s) or legal guardian has provided written informed consent
- •Subject has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the CUP, NPP or EAP
- •Subject is on vamorolone on the day of enrolment
- •Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements
排除标准
- •Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments
- •Vamorolone treatment discontinued for ≥6 months within the year prior to enrolment for a non-safety reason, or vamorolone treatment previously discontinued at any time for a safety reason
- •Severe hepatic impairment
结局指标
主要结局
Number of vertebral fractures per 1000 person-years based on X- ray central reading
Number of vertebral fractures per 1000 person-years based on X- ray central reading
次要结局
- Time to first vertebral fractures (cumulative incidence)
- Number of non-vertebral fractures per 1000 person-years based on investigator reporting
- Time to first non-vertebral fractures (cumulative incidence)
- Number of cataracts per 1000 person-years based on ophthalmologist assessment
- Number of subjects not reaching Tanner stage 2 by 15 years of age
- Frequency of adverse events (AEs) and serious adverse events (SAEs)
- Change from baseline in body weight, height and body mass index (BMI)
- Number of subjects with clinically relevant laboratory abnormalities including glycosylated haemoglobin (HbA1c), and morning cortisol
- Change from baseline in Time to Stand Test (TTSTAND) velocity
- 6-Minute Walk Test (6MWT) distance
- Change from baseline in 6MWT distance
- NorthStar Ambulatory Assessment (NSAA) scores
- Age at ambulatory and non-ambulatory milestones
研究者
Santhera General Inquiries
Scientific
Santhera Pharmaceuticals (Schweiz) AG
研究点 (10)
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