A Monocentric, Prospective and Longitudinal Study Investigating the Acceptability, Feasibility, Safety and Efficacy of an Optimized Rehabilitation Program for Treated Patients With SMA Compared to the Current Rehabilitation Program in United Kingdom: ACE SMA.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 2
- 主要终点
- Acceptability of an optimized rehabilitation program. Measured as number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate. Endpoint: 70% of acceptability.
研究概览
简要总结
The goal of this study is to investigate the acceptability, feasibility, safety and efficacy of an optimized rehabilitation program for treated patients with spinal muscular atrophy (SMA) compared to the current rehabilitation program in the United Kingdom. The aim is to provide patients with more hands on physiotherapy and access to rehabilitation devices at home to support parents currently providing rehabilitation on their own.
详细描述
The main study objective is to investigate whether an optimized rehabilitation program would be accepted and is feasible within the SMA patient community.
The optimized program will comprise of goal orientated rehabilitation with more frequent hands on physiotherapy (every 2 weeks) as well as a SMA approved rehabilitation device used at home on a weekly basis, to provide the most appropriate therapy for each individual patient. All study participants will receive the optimized rehabilitation program over 12 months. An external control group will be used to determine the potential efficacy.
Research study visits will take place at baseline, month 6 and month 12. At these visits a general physical exam will be undertaken alongside collecting the participant and carer perception, satisfaction and compliancy of the rehabilitation program. Motor function assessments according to age and ambulatory status will be assessed to help evaluate potential therapeutic benefits.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient participant Eligibility Criteria:
- •Inclusion Criteria:
- •Genetically confirmed SMA considered as a non-sitter, sitter or walker
- •Post-symptomatically treated and on stable dose for 12 months with any disease-modifying market-approved drug
- •Patients from 1-10 years of age at baseline
- •Parent(s)/legal guardian(s) of patients less than 16 years of age must provide written informed consent prior to the patient's participation in the study
- •Willing and able to comply with all protocol requirements and procedures.
排除标准
- •The participant may not enter the trial if ANY of the following apply:
- •Any acute or chronic condition which, as assessed by the investigator, significantly interferes with the rehabilitation of the patient
- •Need of diurnal and/or invasive ventilation, naps excluded
- •Currently enrolled in a treatment study; or treatment with an experimental therapy
- •Any surgical and/or medical intervention, according to the investigator, 3 months before baseline and/or during the study participation.
- •Carer (considered as participant) Eligibility Criteria:
- •Inclusion Criteria:
- •Willing and able to comply with all protocol requirements and procedures
- •Carer's child has been included in study.
- •Exclusion Criteria:
- •No exclusion criteria.
研究组 & 干预措施
Treated cohort
干预措施: Optimized rehabilitation program (Other)
结局指标
主要结局
Acceptability of an optimized rehabilitation program. Measured as number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate. Endpoint: 70% of acceptability.
时间窗: Baseline visit
Number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate.
Feasibility of an optimized rehabilitation program. Measured as the number of patients who complete the study compared to the number of included patients. Endpoint: 60% of feasibility.
时间窗: Month 12 (end of study)
Number of patients who complete the study compared to the number of included patients.
次要结局
- Carer perception and satification measured through the Clinical Global Impression scale - Improvement (CGI I). Comparison of CGI-S score with CGI-I score of the treated group.(Month 6 and Month 12 (end of study).)
- Carer perception and satification measured through the Clinical Global Impression scale - Severity (CGI-S). Comparison of CGI-S score with CGI-I score of the treated group.(Baseline visit: CGI-S.)
- Safety of an optimized rehabilitation program. Measured as the number of serious adverse events (SAE) compared to the year before the baseline.(From baseline to month 12 (end of study))