An interventional study of UG3NS140720 Deep Brain Stimulation System in Spinocerebellar Ataxia Genotype Type 1 and Spinocerebellar Ataxia Genotype Type 3, sponsored by Gordon H. Baltuch. Not yet recruiting at 2 sites in United States. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-10-29.
Sponsored by Gordon H. Baltuch · Not applicable, Interventional, and Treatment
The purpose of this study is to evaluate the safety and feasibility of deep brain stimulation (DBS) in the deep cerebellar nuclei (DCN) in patients with Spinocerebellar Ataxia (SCA) types 1 and 3.
Primary Objective: The incidence and nature of treatment-related adverse events across the entire study, including serious treatment-related adverse events such as intracerebral hemorrhage, infection, and serious or unanticipated adverse device effects (SADEs and UADEs).
Secondary/Exploratory Objectives:
People with spinocerebellar ataxia (SCA) face progressively disabling symptoms such as poor balance, frequent falls, and a loss of muscle control. Currently, there are no approved treatments for these symptoms.
Developing medications to treat SCA is incredibly difficult because the brain's movement center-the cerebellum-has complex, damaged wiring that varies between patients. However, all signals from this complex brain region flow through a single exit point called the deep cerebellar nuclei (DCN). This makes the DCN (specifically, the dentate nucleus or DN) a perfect target for brain stimulation. Over the past five years, animal studies have shown that using a device to stimulate this area (called deep brain stimulation, or DBS) can significantly improve movement control. Recently, a small study showed promising early results in humans, particularly in two patients with SCA type 3.
To translate these findings into a standard treatment that has the potential to improve patients' lives, the investigators must first determine whether this is feasible and safe. The investigators will also need to see if this treatment works for different types of SCA, since cerebellar damage varies from person to person. Therefore, the aim of this study is to test and safety, feasibility, and early effectiveness of DBS on both sides of the brain in 12 people with SCA types 1 and 3. During the study, the investigators will use temporary external wires and a brain-recording device to listen to the brain's signals. By matching these signals with standard brain wave tests (EEGs), the investigators will hopefully understand exactly how this stimulation fixes the brain's movement network.
94 studies on the registry are indexed under Spinocerebellar Ataxias; 24 are open to participants now.
This study's planned enrollment of 12 is below the median of 31 across 60 interventional studies indexed under Spinocerebellar Ataxias.
Browse Spinocerebellar Ataxias studies →This is the only study on the registry with Gordon H. Baltuch as lead sponsor.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The American Psychiatric Association’s manual of mental health diagnoses. DSM-5 (2013, revised as DSM-5-TR in 2022) replaced DSM-IV, and some studies still name the older edition. “Meets DSM criteria for …” means diagnosed by that edition’s diagnostic criteria, usually by a clinician in an interview.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
MMSE and MoCA
Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
DSM diagnostic criteria
The American Psychiatric Association’s manual of mental health diagnoses. DSM-5 (2013, revised as DSM-5-TR in 2022) replaced DSM-IV, and some studies still name the older edition. “Meets DSM criteria for …” means diagnosed by that edition’s diagnostic criteria, usually by a clinician in an interview.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will undergo surgical implantation of the DBS leads, followed by a second surgery to implant a pulse generator (IPG) which generates electricity.
Device: UG3NS140720 Deep Brain Stimulation System
The UG3NS140720 Deep Brain Stimulation System is primarily comprised of components from the Medtronic Percept™ system, which is approved in the U.S. by the FDA for unilateral or bilateral stimulation of the ventral intermediate nucleus (VIM) of the thalamus as adjunctive therapy for ET, cerebellar outflow tremor, and MS-related tremor. This system is also approved to be used on patients with neurologic disorders such as Parkinson's disease, dystonia, or epilepsy. The FDA has not approved any DBS system for the stimulation of the Deep Cerebellar Nuclei (DCN) for this patient population. Deep brain stimulation (DBS) utilizes surgically implanted electrodes and a neurostimulator to deliver electrical energy to specific targeted structures deep within either one or both hemispheres of the brain.
Also known as: DBS System
Incidence of treatment-related adverse events
To further understand the safety and optimize a novel therapeutic target to achieve ataxia control for individuals with SCA. The adverse events will be evaluated and tallied if they met the criteria as Serious Adverse Events (SAE), Serious Adverse Device Effects (SADE), and Unanticipated (Serious) Adverse Device Effects (UADE).
Time frame: Up to 72 months
Mean Scale for Assessing and Rating of Ataxia (SARA) Score
To characterize the effect of DN-DBS on disease-related impairments, the study will measure ataxia severity as assessed by the Scale for Assessing and Rating of Ataxia (SARA). SARA is a clinical scale that includes 8 different domains of neurological exams, including gait, stance, sitting, speech, and hand dexterity. The total amount of points on SARA ranges from 0 (no ataxia) to 40 (most severe ataxia).
Time frame: Baseline, Month 11
Mean Patient-Reported Outcome Measures - Ataxia (PROM-Ataxia) Scale Score
To characterize the effect of DN DBS on ataxia severity and related cognitive and emotional symptoms in SCA, the study will utilize the Patient-Reported Outcome Measures - Ataxia (PROM-Ataxia) scale. PROM-Ataxia is a 3-domain, 70-item questionnaire which assesses 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.
Time frame: Baseline, Month 11
Mean Cerebellar Cognitive Affective Syndrome Scale (CCAS) Score
SCA patients may have cognitive impairment, presumed to come from the dysfunctional cerebellum. Therefore, cognition status will be measured by the Cerebellar Cognitive Affective Syndrome Scale (CCAS). The CCAS evaluates multiple cognitive domains such as executive function, attention, memory, language, visual-spatial skills, abstract reasoning, and neuropsychiatric features. The total possible score is 120 points; the Pass / Fail measure provides a maximum fail score of 10 (i.e., 10 failed tests). A fail score of 0 is normal. In a patient with cerebellar disease, a fail score of 1 indicates Possible CCAS, a fail score of 2 indicates Probable CCAS, and a fail score of 3 or more indicates Definite CCAS.
Time frame: Baseline, Month 11
Mean Patient Health Questionnaire (PHQ-9) Score
To assess depression symptoms, the Patient Health Questionnaire assessment will be used. The PHQ is a 9-item patient-reported assessment which asks how often a patient has experienced a symptom in the prior 2 weeks. Responses range from "0" (Not at all) to "3" (nearly every day). Scores range from 0 to 27, with total scores over 10 indicating a presence of depression.
Time frame: Baseline, Month 11
Mean Generalized Anxiety Disorder (GAD) Scale Score
To assess anxiety symptoms, the GAD scale will be used. The Generalized Anxiety Disorder (GAD) scale is a 7-item patient-reported assessment which assesses how often a patient has experienced a symptom of anxiety in the last 2 weeks. To assess depression symptoms, the Patient Health Questionnaire assessment will be used. Responses range from "0" (Not at all) to "3" (nearly every day). Scores range from 0 to 21, with total scores over 10 indicating a presence of anxiety.
Time frame: Baseline, Month 11
Mean Patient Global Impression (PGI) Score
The Patient Global Impression (PGI) scores are single-item patient-reported assessments. This study will use the severity (PGI-S) and change (PGI-C) scores.
Time frame: Baseline, Month 11
Mean Gait Measure of Ataxia
Kinematic measures of ataxia will be measured by 6 wearable sensors from the APDM Mobility Lab System. The system has recently been validated to track cerebellar ataxia severity in SCA patients. SCA patients have increased postural sway, increased toe-out angle variability, and increased double-support time variability. APDM sensors will also be used to study different gait cycles and finger-to-target movements as indicators for gait and hand ataxia, respectively.
Time frame: Baseline, Month 11
Mean Postural Kinematic Measure of Ataxia
Kinematic measures of ataxia will be measured by 6 wearable sensors from the APDM Mobility Lab System. The system has recently been validated to track cerebellar ataxia severity in SCA patients. SCA patients have increased postural sway, increased toe-out angle variability, and increased double-support time variability. APDM sensors will also be used to study different gait cycles and finger-to-target movements as indicators for gait and hand ataxia, respectively.
Time frame: Baseline, Month 11
Mean EQ-5D-5L Score
The EQ-5D-5L is a self-assessed, health related, quality of life questionnaire. The scale measures quality of life on a 5-component scale including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.
Time frame: Baseline, Month 11
Number of evaluable recordings completed by participants
EEG, EMG, and LFP recordings will be collected at rest and during ataxia-eliciting behaviors at specified study visits to characterize activity, as well as assess how DN-DBS modulates oscillatory activity and coupling, across the cerebellothalamocorticomuscular (CTCM) network.
Time frame: From Implant to End of Study (Approximately 11 Months)
Plan to share: Undecided
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From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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