12-Week Strength and Functional Exercise Protocol for Treating Motor Symptoms in Individuals With Hereditary Spastic Paraplegia: Adaptive Randomized Crossover Clinical Trial
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Spastic Paraplegia Rating Scale (SPRS)
研究概览
简要总结
This study aims to evaluate whether a 12-week program of strength and functional exercises can improve movement and daily function in people with Hereditary Spastic Paraplegia (HSP). Participants will take part in supervised exercise sessions twice a week, delivered by videoconference, with guidance from a physiotherapist. The exercises will be tailored to each participant's abilities and goals and will focus on improving muscle strength, balance, and walking.
The study is a randomized crossover trial, meaning that all participants will experience both the exercise program and a comparison period without the intervention.
Eligible participants are individuals aged 16 to 70 years with a clinical or genetically confirmed diagnosis of HSP, who are able to walk independently (with or without assistive devices) and have stable treatment for spasticity.
Researchers will measure changes in walking ability, balance, muscle strength, and quality of life using clinical scales and functional tests. These include walking tests, mobility assessments, and questionnaires about daily activities and confidence in movement. To reduce bias, outcome assessments will be performed by an evaluator who is blinded to the participants' group allocation.
The results of this study will help determine whether a structured exercise program is safe, feasible, and effective for improving motor symptoms in people with HSP, and may support its use as part of routine care.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Investigator, Outcomes Assessor)
盲法说明
Assessments will be conducted by two evaluators blinded to group allocation and will take place in person at two distinct time points: pre-intervention and immediately post-intervention.
入排标准
- 年龄范围
- 16 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of hereditary spastic paraplegia confirmed by genetic testing or clinical diagnosis with a positive family history
- •Age between 16 and 70 years
- •Independent ambulation, with or without assistive devices
- •Access to an electronic device (smartphone, tablet, or computer) with camera and internet connection
- •Stable therapeutic regimen for spasticity management (pharmacological and/or physiotherapy) for at least 6 months prior to enrollment
排除标准
- •Presence of other neurological or systemic conditions causing additional motor impairment or musculoskeletal injury limiting movement
- •Inability to perform performance outcomes even with assistive devices
- •Planned or ongoing botulinum toxin injections or surgical interventions for spasticity reduction during the 12-week intervention period
研究组 & 干预措施
Individualized Functional and Strength Exercise Intervention
Individualized functional and strength exercise intervention delivered via videoconference and supervised by a physiotherapist.
干预措施: Individualized Functional and Strength Exercises (Other)
No Intervention
No individualized prescription or real-time supervision will be provided during this phase. They will receive printed educational materials containing general guidance on performing strength and functional exercises.
结局指标
主要结局
Spastic Paraplegia Rating Scale (SPRS)
时间窗: Baseline, 12 and 24 weeks
Clinician-reported outcome used to assess disease severity in hereditary spastic paraplegia (HSP). It consists of 13 items evaluating gait, lower limb strength and spasticity, joint contractures, pain, and bladder and bowel function. Total scores range from 0 to 52, with higher scores indicating greater disease severity.
Timed Up and Go Test (TUG) - maximum walking speed
时间窗: Baseline, 12 and 24 weeks
Performance outcome in which participants are instructed to stand up from a seated position, walk 3 meters, turn around, walk back, and sit down at maximum walking speed. The time to complete the task is recorded.
Goal Attainment Scaling (GAS)
时间窗: Baseline, 12 and 24 weeks
individualized patient reported outcome measure used to assess the extent to which patient-specific goals are achieved during the intervention. Each participant will define four main individualized goals at baseline. Goals and expected levels of performance are defined individually for each participant and scored on a 5-point scale ranging from -2 to +2. A score of 0 represents the expected level of achievement, positive scores (+1, +2) indicate better-than-expected outcomes, and negative scores (-1, -2) indicate less-than-expected outcomes.
次要结局
- 10-Meter Walk Test (10MWT) - self-selected walking speed(Baseline, 12 and 24 weeks)
- 10-Meter Walk Test (10MWT) - maximum walking speed(Baseline, 12 and 24 weeks)
- Timed Up and Go (TUG) - self-selected walking speed(Baseline, 12 and 24 weeks)
- Falls Efficacy scale - International (Brazilian Portuguese)(Baseline, 12 and 24 weeks)
- 36-Item Short Form Health Survey (SF-36) - Brazillian Portuguese(Baseline, 12 and 24 weeks)
- Medical Research Council (MRC) Muscle Strength Scale(Baseline, 12 and 24 weeks)
- Modified Ashworth Scale (MAS)(Baseline, 12 and 24 weeks)
- Patient Global Impression of Change (PGI-C)(Baseline, 12 and 24 weeks)