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
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
- 试验地点
- 27
- 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
This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.
详细描述
All subjects in this study have completed previous studies with vamorolone and continued to receive vamorolone under special programs: Compassionate Use Program [CUP], Named Patient Program [NPP] or Expanded Access Protocol [EAP]. All subjects will continue treatment with vamorolone under Guardian protocol instead. The primary objective of this study is to evaluate the safety of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy regarding vertebral fractures. Secondary study objectives will evaluate the safety of long-term treatment with vamorolone on non-vertebral fractures, cataracts, delayed puberty, overall safety as well as ambulatory and non-ambulatory function.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
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 Compassionate Use Program, Named Patient Program or Expanded Acess Protocol
- •Subject is on vamorolone on 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
研究组 & 干预措施
vamorolone
On Day 1, Subjects will roll over from a previous vamorolone program and continue treatment with vamorolone under this protocol. During the study, vamorolone will be administered at a dose range between 2 mg/kg/day and 6 mg/kg/day for boys weighing <40 kg. For boys weighing 40 kg or above, the dose range will be 80 mg to 240 mg once daily.
干预措施: vamorolone 40 mg/mL oral suspension (Drug)
结局指标
主要结局
Number of vertebral fractures per 1000 person-years based on X-ray central reading.
时间窗: At Enrolment and every 2 years during a Full visit
Lateral thoracolumbar spine X-Rays will be collected and sent to a central reader for evaluation of vertebral fractures
次要结局
- Time to first vertebral fractures (cumulative incidence)(From enrolment up to at least 2 years)
- Number of non-vertebral fractures per 1000 person-years based on investigator reporting(From enrolment until up to at least 3 years)
- Time to first non-vertebral fractures (cumulative incidence)(From enrolment until up to at least 3 years)
- Number of cataracts per 1000 person-years based on ophthalmologist assessment(From enrolment until up to at least 3 years)
- Number of subjects not reaching Tanner stage 2 by 15 years of age(From enrolment until up to at least 3 years)
- Frequency of adverse events (AEs) and serious adverse events (SAEs)(From enrolment until up to at least 3 years)
- Change from baseline in body weight(From enrolment until up to at least 3 years)
- Number of subjects with clinically relevant laboratory abnormalities(From enrolment until up to at least 3 years)
- Change from baseline in Time to Stand (TTSTAND) velocity(From enrolment until up to at least 3 years)
- Six-minute Walk Test (6MWT)(From enrolment until up to at least 3 years)
- Change from baseline in 6MWT distance(From enrolment until up to at least 3 years)
- Age at ambulatory and non-ambulatory milestones(From enrolment until up to at least 3 years)
- North Star Ambulatory Assessment (NSAA) scores(At enrolment and each Full visit (Month 12, 24, 36, etc / End-of -Treatment) until End of Study)
- Change from baseline in body height(From enrolment until up to at least 3 years)
- Change from baseline in Body Mass Index (BMI)(From enrolment until up to at least 3 years)