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| ID | Type | Description | Link |
|---|---|---|---|
| UH3NS128297-01A1 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institutes of Health (NIH) | NIH |
| National Institute of Neurological Disorders and Stroke (NINDS) | NIH |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Effective stimulation | Active Comparator | 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. |
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| Sham stimulation | Sham Comparator | Sham stimulation will be settings at low amplitude (0.1mA) known to be ineffective. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DBS | Device | Implanted in the cerebellum. |
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| Measure | Description | Time Frame |
|---|---|---|
| Average total (global index) Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18-R) scores | 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). | Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation). |
| Average total standardized Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) scores | 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). | Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Marta San Luciano Palenzuela, MD, MS | Contact | 4153532311 | Marta.SanLucianoPalenzuela@ucsf.edu | |
| Sarah Wang, PhD, CCRP | Contact | 4153537885 | Sarah.Wang@ucsf.edu |
| Name | Affiliation | Role |
|---|---|---|
| Marta San Luciano Palenzuela, MD, MS | University of California, San Francisco | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, San Francisco | Recruiting | San Francisco | California | 94158 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38684956 | Derived | San Luciano M, Oehrn CR, Wang SS, Tolmie JS, Wiltshire A, Graff RE, Zhu J, Starr PA. Protocol for combined N-of-1 trials to assess cerebellar neurostimulation for movement disorders in children and young adults with dyskinetic cerebral palsy. BMC Neurol. 2024 Apr 29;24(1):145. doi: 10.1186/s12883-024-03633-z. | |
| 38645256 | Derived | San Luciano M, Oehrn CR, Wang SS, Tolmie JS, Wiltshire A, Graff RE, Zhu J, Starr PA. Protocol for combined N-of-1 trials to assess cerebellar neurostimulation for movement disorders in children and young adults with dyskinetic cerebral palsy. Res Sq [Preprint]. 2024 Apr 1:rs.3.rs-4077387. doi: 10.21203/rs.3.rs-4077387/v1. |
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| ID | Term |
|---|---|
| D002547 | Cerebral Palsy |
| ID | Term |
|---|---|
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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The differences in primary outcome scores are calculated while on intervention (effective stimulation) versus placebo (sham stimulation), using an N-of-1 trial design. Each N-of-1 trial consists of a baseline assessment prior to DBS surgery (4 weeks), DBS surgery and open label phase to determine optimal stimulation settings (20 weeks), followed by a randomized three-cycle sequence of paired 8-week exposure periods, with each pair including intervention then placebo or vice versa (24 weeks). The DBS intervention exposure will be at the personalized optimal stimulation settings determined during the open-label phase.
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