Intrathecal Baclofen for the Management of Hereditary Spastic Paraparesis: a Prospective Cohort Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Six-Minute Walk Test(6-MWT)
研究概览
简要总结
The investigators conduct a prospective cohort study to explore the treatment effectiveness of continuous infusion of intrathecal baclofen (ITB) for hereditary spastic paraplegia (HSP) in China, delve into the optimal timing for starting treatment, and investigate the response differences among different subtypes. The ultimate goal is to provide clinical evidence and guidance for the application of ITB in treating HSP in China, as well as improve the life expectancy and quality of life for HSP patients. The main questions it aims to answer are:
- Changes in gait and motor function, as well as spasticity levels, compared to pre-surgery and control group after ITB surgery.
- Changes in quality of life, pain, psychological and emotional status, and cognition compared to pre-surgery and control group after ITB surgery.
- Complications following ITB surgery.
- Impact of ITB surgery on the occurrence and progression of skeletal deformities.
- Subgroup analysis: comparing surgical outcomes between different genotypes and between simple versus complex types.
- Determine the optimal timing for ITB intervention.
详细描述
Hereditary spastic paraplegias (HSPs) are a heterogeneous group of neurodegenerative disorders characterized by progressive spasticity and weakness of the lower limbs due to retrograde axonal degeneration of the corticospinal tract. Baclofen is a selective GABA-B receptor agonist and is commonly used for the treatment of spasticity. Baclofen can be administered orally or intrathecally by the surgical implantation of a specialized pump. Intrathecal baclofen (ITB) is significantly more potent for the treatment of spasticity than the oral form. The goal of this clinical trial is to explore the efficacy and safety of ITB in the treatment of HSP patients. This study is prospective, open-label, single center, and this trial will last for 3 years. A total of 50 patients will participate. Twenty-five patients who will receive ITB and the other 25 control patients will receive oral baclofen. Patients with HSP aged 14 to 70 years, with a Modified Ashworth Scale score of ≥3 in ≥2 joints of lower limb, will be recruited. Patients who do not agree to undergo ITB and/or those who have an inadequate response during intrathecal baclofen test will receive oral baclofen therapy and undergo natural history observation. Professional clinical evaluations are conducted regularly for both groups.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 14 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients meet the clinical and genetic diagnostic criteria of hereditary spastic paraplegia (HSP);
- •Age: 14 to 70 years old
- •Modified Ashworth Score for lower limbs: ≥2 joints with muscle tone ≥grade 3
- •Patients are willing to participate in clinical trials and able to understand and comply with the research program
排除标准
- •Patients are allergic to the baclofen
- •Other neurological diseases likely affecting the evaluation of study treatment
- •Other medical conditions such as: heart disease, tumor, blood disease, liver disease, kidney disease, etc. in the past 1 year
- •Pregnancy or lactating women or subjects who are unable to use appropriate contraception during the trial
- •Participating in another study drug trial and used the investigational drug in the past 30 days
- •Subjects have poor compliance or other factors that are not suitable for participating in the clinical trial
结局指标
主要结局
Six-Minute Walk Test(6-MWT)
时间窗: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
The 6-MWT measures the distance a person can walk in six minutes. A shorter distance indicates lower exercise capacity.
knee flexion angle
时间窗: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Knee flexion angle is measured by gait analysis device.An increase in the knee flexion angle indicates improved motor function and reduced spasticity.
Step length
时间窗: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Step length is measured by gait analysis device. Step length refers to the distance from the heel strike of one foot to the heel strike of the opposite foot. Longer step length indicates that the patient has improved lower limb muscle strength and coordination, and that spastic symptoms have been alleviated.
Range of Motion (ROM) of Joints
时间窗: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
ROM refers to the distance and direction a joint can move to its full potential. Flexion, extension, abduction, adduction of joints are measured.
walking speed
时间窗: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
Walking speed is measured by gait analysis device. Walking speed refers to the distance covered by a person within a unit of time while walking, expressed in meters per minute (m/min). An increase in walking speed indicates an improvement in spasticity and motor function.
Modified Ashworth Scale(MAS)
时间窗: From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively
range: 0-4, higher scores mean more severe spasticity.
次要结局
- MoCA score(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- Patient Global Impression of Change (PGIC) scale(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- Oswestry Disability Index (ODI)(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- MMSE score(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- GMFM-88(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- Numeric Rating Scale (NRS)(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- SDS(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- SAS(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- SPRS score(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- Complications(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- GAS(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- Foot deformity molding(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
- Scoliosis assessment(From the end of treatment to observations at 6 months, 12months, 18months, 24months, 30months, and 36months respectively)
研究者
Li Cao
Professor
Shanghai 6th People's Hospital