Cerebellar Deep Brain Stimulation for Severe Combined Movement Disorders and Spasticity in Children and Young Adults With Cerebral Palsy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Average total (global index) Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18-R) scores
研究概览
简要总结
The purpose of this study is to test the safety of placing Deep Brain Stimulators (DBS) in a part of the brain called the cerebellum and using electrical stimulation of that part of the brain to treat movement symptoms related to cerebral palsy. Ten children and young adults with dyskinetic cerebral palsy will be implanted with a Medtronic Percept Primary Cell Neurostimulator. We will pilot videotaped automated movement recognition techniques and formal gait analysis, as well as collect and characterize each subject's physiological and neuroimaging markers that may predict hyperkinetic pathological states and their response to therapeutic DBS.
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
- Triple (Participant, Care Provider, Outcomes Assessor)
入排标准
- 年龄范围
- 7 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of DCP (dystonic and/or choreoathetotic cerebral palsy) with or without comorbid spasticity, with a clear history of hypoxic ischemic brain injury preceding motor symptoms made by a pediatric neurologist, with supporting MRI findings.
- •Age 7-25 at the time of surgery.
- •Gross Motor Function Classification System (GMFCS) Levels II-V.
- •History of appropriate therapy with oral medications with inadequate relief as determined by a movement disorders or pediatric neurologist. Prior history of selective dorsal rhizotomy is allowed.
- •Patient and family have requested surgical intervention with DBS for their movement disorder.
- •No gross cerebellar abnormalities observed and reported on structural MRI.
- •Written informed consent and written/verbal assent for those younger than 18 years of age.
- •Ability to comply with study follow-up visits for brain recordings, neuroimaging and testing of sham and effective stimulation and clinical assessments.
排除标准
- •Coagulopathy, uncontrolled epilepsy, severe cardiopulmonary or gastrointestinal conditions, or other medical conditions considered to place the patient at elevated risk for surgical complications.
- •Pregnancy: all women of child-bearing potential will be required to have a negative urine pregnancy test prior to undergoing their surgical procedure.
- •Exclusion of genetic mimics of cerebral palsy: exclusion of conditions that manifest with a clinical syndrome similar to CP, in the absence of documented risk factors or neuroimaging findings consistent with a history of brain injury or congenital cerebral malformation. Work up may include comparative genomic hybridization (CGH) microarray and multi-gene panel and/or whole genome or whole exome sequencing.)
- •Severe fixed contractions and skeletal deformities that would preclude determination of improvement.
- •Traumatic brain injury (i.e., non-accidental trauma) or history of infectious or autoimmune encephalitis.
- •Requirement of diathermy, electroconvulsive therapy or transcranial magnetic stimulation.
研究组 & 干预措施
Effective stimulation
All participants will receive deep brain stimulation (DBS) in the cerebellum. For the first 20 weeks, every participant undergoes an open label phase to titrate stimulation and determine optimal stimulation settings. Following that phase, each participant starts three cycles of randomized, paired 8-week exposure periods, each pair including effective stimulation followed by sham stimulation, or vice versa. Effective stimulation will be the optimal stimulation settings determined during the open label phase.
干预措施: DBS (Device)
Sham stimulation
Sham stimulation will be settings at low amplitude (0.1mA) known to be ineffective.
干预措施: DBS (Device)
结局指标
主要结局
Average total (global index) Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18-R) scores
时间窗: Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation).
This is the primary motor outcome measure. It is the Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18 R). The MD-CRS 4-18 R is a validated tool aimed to evaluate movement disorders in developmental age. It is particularly useful for rating the severity of movement disorders in dyskinetic cerebral palsy. It scored based on two parts. Part I: General Assessment has a minimum total score of 0 (no impairment) and a maximum total score of 60 (maximal impairment). Part II: MD Assessment has a minimum total score of 0 (movement disorder is absent) and a maximum total score of 28 (maximal movement disorder presence).
Average total standardized Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) scores
时间窗: Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation).
This is the primary Quality of Life (QOL) outcome measure. It is the Caregiver Priorities \& Child Health Index of Life with Disabilities (CPCHILD) scale. The CPCHILD is a reliable and valid measure of caregivers' perspectives on health status, functional limitations, and well-being of patients with severe CP, including those individuals who are non-verbal and non-ambulatory. Because many children suffering with CP are severely affected in many areas of their lives including activities of daily living, communication, mobility and overall health, there is an imperative to evaluate new interventions, especially invasive and resource-intensive ones such as DBS, using outcomes that are more meaningful to patients and their caregivers. Scores for each domain and for the total survey are standardized and range from 0 (worst) to 100 (best).
次要结局
未报告次要终点
研究者
Marta San Luciano Palenzuela, MD
Associate Professor, Neurology
University of California, San Francisco