A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA Patients: Single-center, Randomized, Single-blinded, Comparative Study of a Robotically Active Verticalization Model Versus Robotically Assisted Locomotion Model
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 200
- 试验地点
- 2
- 主要终点
- X-ray or ultrasound scan imaging assessment of the spine, hip joints and bone mineral density
研究概览
简要总结
A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization versus Robotically Assisted Locomotion.
The objective of research:
The main goal of the project is to determine the optimal robotically assisted rehabilitation model for people with SMA depending on age and baseline functional status. The study consists of a head-to-head comparison of two rehabilitation models.
- Research period: 4 years
- Patients age: 0-21 y.o.
- Group size: 200 patients (100 patients in each group)
- Assignment of patients to study groups in a randomised manner
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Single (Investigator)
盲法说明
Patients and their parents will not be blinded by the nature of the therapeutic procedure used after random allocation to the treatment group. Study personnel will be divided into Blinded Team and Unblinded Team to ensure objectivity of the assessment of safety and effectiveness of both strategies.
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signing of informed consent to participate in the experiment by the participant or the participant's parent/legal guardian
- •Diagnosis of SMA (spinal muscular atrophy 1-4) confirmed by genetic testing, symptomatic or pre-symptomatic
- •Age between 0 and 21 years of age (will be determined on the day of starting participation in the project based on the date of birth)
- •Treatment under a drug program for spinal muscular atrophy, provided that the patient has a diagnosis of spinal muscular atrophy at the date of eligibility.
排除标准
- •Cardio-respiratory disorders requiring invasive ventilation
- •Advanced osteoporosis with multiple fractures prior to treatment
- •Functional deterioration during the rehabilitation process in the range of scales appropriately selected for the SMA type: Prechtl, HINE, CHOP-INTEND, HFMS, RULM
- •Lack of cooperation with the therapist
- •Other functional indications preventing exercise
结局指标
主要结局
X-ray or ultrasound scan imaging assessment of the spine, hip joints and bone mineral density
时间窗: Once per 1 year
Assessment of the quality of life of patients and their caregivers using Pediatric Quality of Life Inventory ver. 4.0
时间窗: Through study completion, max. for 2.5 year every 6 months
Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale
时间窗: Through study completion, max. for 2.5 year every 6 months
Clinical assessment of patients using Balance test minimum 1 min., Self-walk test of at least 2 meters, 6-minute gait test, Up&Go test, 10 meters gait test, hip range of motion and muscle strength evaluation.
时间窗: Through study completion, max. for 2.5 year every 6 months
Functional assessment of patients using GMFM (Gross Motor Function Measure) scale
时间窗: Through study completion, max. for 2.5 year every 6 months
次要结局
- Occurence of discomfort during therapy requiring abrupt interruption or significant modification(Through study completion, max. for 2.5 year every 6 months)
- Hospitalization during or between rehabilitations that take place each 6 months(Through study completion, max. for 2.5 year)
- Number of fractures(Through study completion, max. for 2.5 year)