tSCS联合5-氮杂胞苷改善脑性瘫痪运动功能
推进神经康复：评估经皮脊髓刺激与5-氮杂胞苷在改善脑瘫患者运动功能中的作用
This is an intervention study to investigate the impact of spinal stimulation on mobility outcomes in children with Cerebral Palsy. Participants will complete a 16-week training program with weekly sessions of spinal stimulation and walking or activity-based training. Participants will also have the option to participate in a randomized control trial to investigate the impact of 5-Azacitidine combined with the spinal stimulation to further affect mobility outcomes.
研究概览
详细说明
This project will investigate the impact of transcutaneous spinal cord stimulation (tSCS) on gait and activity-based training in children with Cerebral Palsy. Through a 1:1 randomization, a subset of participants will also receive a dose of 5-azacitidine (AZA), an FDA-approved drug for pediatric oncology, or a placebo. Research in muscle fibers as shown that AZA aids in muscle growth and regeneration. The use of AZA in this study is an off-label application, employed together with the tSCS intervention to investigate the impact of targeting both muscular and mechanical deficits related to CP. This project will also collect blood samples of participants to perform genetic and epigenetic profiling in order to test for correlation with the severity and trajectory of musculoskeletal impairments and with responses to the tSCS and AZA interventions.
The study team hypothesizes that personalized tSCS combined with targeted gait or activity based training will improve motor outcomes in children with spastic CP by reducing muscle tone, improving gait symmetry and neuromotor function, and increasing functional mobility. Furthermore, the addition of a repurposed anabolic agent (AZA) will augment these gains compared to tSCS and training alone.
Children with CP across GMFCS levels will participate in a three-phase protocol, including: (1) a two week baseline phase, (2) a sixteen week intervention phase during which participants will receive tSCS and training interventions, and (3) a four-week post-intervention phase to examine long term outcomes. At the midpoint, participants will receive a single dose of AZA or a single dose of a placebo through a 1:1 randomization. Participants will have a blood draw in phase 1 for genetic and pre-intervention epigenetic profiling and will have a second blood draw in phase 3 for post-intervention epigenetic profiling.
研究类型
注册 (估计的)
阶段
- 阶段2
联系人和位置
学习联系方式
- 姓名：Audrey Wiesner, BS
- 电话号码：3122388435
- 邮箱：awiesner@sralab.org
研究联系人备份
- 姓名：Tyler Petrie, PhD
- 电话号码：3122380718
- 邮箱：tpetrie@sralab.org
学习地点
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Illinois
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Chicago、Illinois、美国、60611
- 招聘中
- Shirley Ryan AbilityLab
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首席研究员：
- Arun Jayaraman, PhD
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-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- Diagnosis of cerebral palsy (CP) classified as Gross Motor Function Classification System (GMFCS) Levels I-V.
- Between 4 and 17 years old at the time of enrollment/consent.
- Diagnosis of spastic CP hemiplegia, diplegia, or quadriplegia.
- Stable medical condition as determined by the investigator.
- Adequate caregiver support to be able to participate in training and assessment sessions for the duration of the study, at the discretion of the Investigator.
- Capable of performing simple cued motor tasks and can follow 2-3 step commands.
- Capable of communicating an accurate yes or no answer to questions according to parent or guardian.
- Able to localize pain/ discomfort.
- Physician approval for participation.
- Parent/ guardian permission.
Exclusion Criteria:
- Concurrent neurological disease affecting the central nervous system.
- Cardiovascular or musculoskeletal disease or injury that would prevent full participation in physical therapy intervention
- Orthopedic dysfunction, injury, or surgery that would impact an individual's ability to use the upper and/or lower extremity
- Unhealed fracture or other musculoskeletal impairment that might interfere with upper or lower extremity rehabilitation or testing activities
- Implanted stimulator (e.g., epidural stimulator, vagus nerve stimulator, pacemaker, cochlear implant) or drug delivery device (e.g., baclofen pump)
- Dependence on an electro-magnetic medical implant (e.g., cardiac pacemaker, implanted drug pump), ventilation support, or another external device.
- History of uncontrolled seizures
- Unexplained presence of persistent complaints of pain of any kind
- Unable to localize pain/discomfort
- Severe cortico-visual impairment
- Active pressure sores
- Active cancer or cancer in remission less than 5 years
- Clinically significant depression, psychiatric disorders, or ongoing drug abuse
- Immunodeficiency or hematologic condition
- Allergy to AZA or mannitol
- Plans to receive a vaccine within 30 days of the planned AZA or placebo injection to take place at week 10
- Pregnancy
- Orthopedic surgery completed in the prior 12 months
- Intrathecal medication titration that may affect muscle spasticity
- Botulinum toxin treatment within 3 months preceding enrollment unless given approval by the treating physician
- Current enrollment in a conflicting research study
- Any other health condition or diagnosis not listed above that may put the participant at risk as determined by the investigator or treating physician
学习计划
研究是如何设计的？
设计细节
- 主要用途：其他
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：三倍
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
有源比较器：tSCS + Functional Activity Training
Transcutaneous spinal cord stimulation will be paired with training based on Aim. Participants in Aim 1(GMFCS levels 1-3) will engage in three sessions a week for 16 weeks of treadmill and overground gait training using tSCS. Participants in Aim 2(GMFCS levels 4-5) will engage in three sessions a week for 16 weeks of functional activity based training as appropriate. |
Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation and activity based training. Stimulation intensity will be based on involuntary motor threshold, determined during a MEPs assessment.
其他名称：
All participants will engage in functional activity training based on Aim. Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation (tSCS) and activity based training. Participants in Aim 1 will complete gait training. Participants in Aim 2 will complete activity-based training. |
|
实验性的：tSCS + Functional Activity Training + 5-Azacitidine
Transcutaneous spinal cord stimulation will be paired with training based on Aim. Participants in Aim 1(GMFCS levels 1-3) will engage in three sessions a week for 16 weeks of treadmill and overground gait training using tSCS. Participants in Aim 2(GMFCS levels 4-5) will engage in three sessions a week for 16 weeks of functional activity based training as appropriate. A subset of opt-in participants will also be randomized to receive either 5-Azacitidine (VIDAZA) or placebo (Mannitol) at midpoint. A registered nurse will deliver a single subcutaneous injection of either AZA or placebo at a dose of 75 mg/m² to the participants lower abdomen or medial thigh. |
Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation and activity based training. Stimulation intensity will be based on involuntary motor threshold, determined during a MEPs assessment.
其他名称：
All participants will engage in functional activity training based on Aim. Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation (tSCS) and activity based training. Participants in Aim 1 will complete gait training. Participants in Aim 2 will complete activity-based training. A subset of opt-in participants will receive a single subcutaneous injection of 5-Azacitidine at a dose of 75 mg/m²
其他名称：
|
|
有源比较器：tSCS + Functional Activity Training + Mannitol (Placebo)
Transcutaneous spinal cord stimulation will be paired with training based on Aim. Participants in Aim 1(GMFCS levels 1-3) will engage in three sessions a week for 16 weeks of treadmill and overground gait training using tSCS. Participants in Aim 2(GMFCS levels 4-5) will engage in three sessions a week for 16 weeks of functional activity based training as appropriate. A subset of opt-in participants will also be randomized to receive either 5-Azacitidine (VIDAZA) or placebo (Mannitol) at midpoint. A registered nurse will deliver a single subcutaneous injection of either AZA or placebo at a dose of 75 mg/m² to the participants lower abdomen or medial thigh. |
Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation and activity based training. Stimulation intensity will be based on involuntary motor threshold, determined during a MEPs assessment.
其他名称：
All participants will engage in functional activity training based on Aim. Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation (tSCS) and activity based training. Participants in Aim 1 will complete gait training. Participants in Aim 2 will complete activity-based training. A subset of opt-in participants will receive a single subcutaneous injection of Mannitol (placebo) at a dose of 75 mg/m².
其他名称：
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Spatiotemporal Gait Symmetry via Instrumented Gait-Mat
大体时间：Baseline to Follow-Up, approximately 22 weeks
|
For Aim 1 participants (GMFCS levels 1-3), an instrumented gait-mat will be used to assess spatiotemporal gait symmetry. |
Baseline to Follow-Up, approximately 22 weeks
|
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Modified Tardieu Scale
大体时间：Baseline to Follow-Up, approximately 22 weeks
|
For Aim 2 participants (GMFCS levels 4-5), muscle spasticity will be assessed using the Modified Tardieu Scale. |
Baseline to Follow-Up, approximately 22 weeks
|
合作者和调查者
调查人员
- 首席研究员：Arun Jayaraman, PhD、Shirley Ryan AbilityLab
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.