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| Name | Class |
|---|---|
| Raynor Cerebellum Project | UNKNOWN |
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The goal of this clinical trial is to test the safety of placing Deep Brain Stimulators (DBS) in the cerebellum and using electrical stimulation of that part of the brain to treat symptoms related to the participants spinocerebellar ataxia. Five adults diagnosed with spinocerebellar ataxia type 6 (SCA6) with inadequate cerebellar symptom relief will be implanted with a Medtronic Percept Primary Cell Neurostimulator. The device will be implanted into the dentate nucleus, which is a structure located within the cerebellum that is responsible for controlling movement and balance.
Specifically, the investigators will be using adaptive deep brain stimulation (aDBS), which analyzes brain signals and automatically adjusts the strength, timing, and pattern of stimulation according to the patient's needs at any given moment. This study will evaluate the feasibility, safety, and tolerability of aDBS in SCA6 patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adaptive Deep Brain Stimulation (aDBS) | Experimental | One month after patients undergo DBS surgery in the cerebellum, conventional deep brain stimulation (cDBS) programming will begin to identify stimulation parameters (such as amplitude, contact, frequency, and pulse width) and ensure no adverse wide effects are witness. This phase also allows the study team to make sure device and electrode placement are working as intended. About nine months after implantation, the investigators will switch system settings over to aDBS to evaluate the feasibility, safety, and tolerability in the dentate nucleus of the cerebellum. Symptoms and side effects will be assessed by patients' self-reports, validated clinical scales, and wearable devices, which will be used to track movements and sleep data. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deep Brain Stimulation (DBS) | Device | This device will be surgically implanted into the dentate nucleus of the cerebellum. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Average total score for the Assessment and Rating of Ataxia (SARA) | SARA is a clinical scale that assesses a variety of different impairments for individuals with cerebellar ataxia. The scale includes 8 different items that are related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements, and heel-shin test. The total amount of points on SARA ranges from 0 (no ataxia) to 40 (most severe ataxia). This study requires participants to have a minimum SARA score of at least 8 so that people who are relatively early in their disease course with limited cerebellar degeneration can be included. | From baseline through study completion, about 2 years. |
| Average total Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia) Score | PROM-Ataxia is a 3-domain, 70-item questionnaire used for the assessment of ataxia-related symptoms. It incorporates a series of questions ranging to the impact of cerebellar dysfunction on physical abilities, activities of daily living, and cognitive-emotional challenges. The maximum severity score on PROM-Ataxia is 280, where all 70 questions are scored on a 0-4 Likert scale. 0 represents never and 4 represents always. | From baseline through study completion, about 2 years. |
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Inclusion Criteria:
Exclusion Criteria:
Inability or unwillingness to comply with the study protocol.
History of previously implanted neurostimulators, pacemakers, defibrillators, or metallic head implants.
Severe cognitive impairment or dementia, defined as a score < 21 on the Montreal Cognitive Assessment (MoCA).
Evidence of ataxia due to other etiologies, including but not limited to:
The presence of active and untreated psychiatric illness, severe depression (Beck Depression Inventory ≥ 21), or personality disorder at the discretion of the study team.
Coagulopathy, uncontrolled epilepsy, or other medical condition considered to place the patient at elevated risk for surgical complications. Presence of a concomitant medical condition that, in the investigator's opinion, may interfere with the study participation or gait/balance, for example, severe arthritis.
Presence of a concominant medical condition that, in the investigator's opinion, may interfere with the study participation or gait/balance, for example, severe arthritis.
Requirement of diathermy, electroconvulsive therapy, or transcranial magnetic stimulation.
Pregnancy or lactation.
Active suicidal ideation, defined as fined as a "Yes" response to questions #2-5 on the Columbia Suicide Severity Rating Scale, C-SSRS.
Refractory Epilepsy.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Marta San Luciano Palenzuela, MD, PhD | Contact | (415) 353-2311 | Marta.SanLucianoPalenzuela@ucsf.edu | |
| Karson J Franjieh, BS | Contact | 415-755-8954 | karson.franjieh@ucsf.edu |
| Name | Affiliation | Role |
|---|---|---|
| Marta San Luciano Palenzuela, MD, PhD | University of California, San Francisco | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Weill Institute for Neurosciences | Recruiting | San Francisco | California | 94158 | United States | |
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| ID | Term |
|---|---|
| D020754 | Spinocerebellar Ataxias |
| ID | Term |
|---|---|
| D002524 | Cerebellar Ataxia |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| D046690 | Deep Brain Stimulation |
| ID | Term |
|---|---|
| D004599 | Electric Stimulation Therapy |
| D013812 | Therapeutics |
| D013514 | Surgical Procedures, Operative |
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| University of California, San Francisco |
| Not yet recruiting |
| San Francisco |
| California |
| 94158 |
| United States |
|
| D009422 |
| Nervous System Diseases |
| D013132 | Spinocerebellar Degenerations |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D001259 | Ataxia |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |