Spinocerebellar Ataxia Genotype Type 1NCT07752095
Cerebellar DBS in SCA1 and SCA3 Study
A study testing UG3NS140720 Deep Brain Stimulation System in people with Spinocerebellar Ataxia Genotype Type 1 and Spinocerebellar Ataxia Genotype Type 3, run by Gordon H. Baltuch at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 3
- What is being tested?
- UG3NS140720 Deep Brain Stimulation System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 2026to Jun 2030
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Subject is aged >= 18 years and =< 60 years.
- Subject has moderate ataxia severity, as demonstrated by a Scale for Ataxia Rating and Assessment (SARA) score >= 11.
- Subject has genetic confirmation of pathological repeat expansions in ATXN1 (for SCA1, repeat size >= 40) and ATXN3 (for SCA3, repeat size >= 56).
- Subject has been on a stable and optimized standard of care (SoC) regimen for at least 3 months prior to Screening (including physical therapy) and continues to exhibit signs of cerebellar ataxia.
- Subject has a minimum 6th grade reading level, as determined by the Rapid Estimate of Adult Literacy in Medicine (REALM) scale.
- Subject is able and willing to give informed consent and attend all study visits.
- Subject is available for all follow-up visits during the length of the study.
Who cannot join
- Subject is unable to provide informed consent.
- Subject has presence of overt tremor, dystonia, and/or parkinsonism, which are atypical features and could be suggestive of prominent extra-cerebellar circuit involvement that may not respond well to cerebellar DBS.
- Subject is wheelchair dependent and may be too progressed to complete gait assessments.
- Subject is unable to stop anticoagulation or platelet anti-aggregation therapy for study procedures.
- Subject has a Montreal Cognitive Assessment (MoCA) score =< 24.
- Subject has a diagnosis of epilepsy.
Show all 18Show fewer
- Subject has a major active psychiatric illness that may interfere with the study, such as psychiatric disorders or severe personality disorders.
- Subject has an untreated or inadequately treated major depressive episode.
- Subject is pregnant, lactating, or planning to become pregnant during the study.
- For women of childbearing potential, subject is unable or unwilling to use accepted contraception methods.
- Subject is unable to communicate with investigators or staff.
- Subject has contraindications to DN DBS, including significant small vessel ischemic disease, vertebrobasilar vascular disease, and/or any other structural abnormalities of the cerebellum, cerebellar peduncles, and brain stem that would preclude safe placement of the DBS leads.
- Subject has contraindications to magnetic resonance (MR) imaging, e.g., weight incompatible with scanner, implanted metallic devices or electrical devices (pacemaker, defibrillator, spinal cord stimulator), or intolerance to MRI contrast agents.
- Subject is enrolled in another device, biologic or pharmaceutical study within 30 days of consent in the current study (i.e., patients cannot be enrolled if participation in another study was not completed at least 30 days prior to consent).
- Subject has evidence of behavior(s) consistent with alcohol or substance abuse/dependence within the preceding 6 months, as outlined by the DSM-V criteria.
- Subject has a condition that, in the opinion of the investigator, would significantly increase the risk for interference with study compliance, safety, or outcome.
- Subject is at risk for suicide, defined by a total score >= 3 on the Columbia Suicide Severity Rating Scale (C-SSRS).
- Patients who are not able to read, speak and/or understand English.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Gordon Baltuch, MD, PhD contact
United States2 sites
Cleveland Clinic
Cleveland, Ohio, 44195
Jeffrey Negrey, 216-316-6896, negreyj2@ccf.org
Andre Machado, MD
Columbia University Irving Medical Center (CUIMC) / NewYork Presbyterian Hospital (NYP)
New York, New York, 10032
Natasha Desai, 347-882-4315, nd2528@cumc.columbia.edu
Nadia Amokrane, 347-882-4315, na2855@cumc.columbia.edu
About this study
Official title: Deep Brain Stimulation of the Deep Cerebellar Nuclei in Subjects With Spinocerebellar Ataxia: A Safety and Feasibility Study
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).
Read more
Secondary/Exploratory Objectives:
- Evaluation of the effect of Dentate Nucleus (DN) - Deep Brain Stimulation (DBS). Ataxia severity will be quantified using the Scale of Assessment and Rating of Ataxia (SARA), Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia), and kinematics.
- To characterize electrophysiological activity across the cerebellothalamocorticomuscular (CTCM) network at rest and during motor behaviors.
- To examine how DN-DBS modulates oscillatory activity and coupling across the CTCM circuit in relation to improvements in controlling ataxia.
Read the detailed description
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.
- Being tested
- UG3NS140720 Deep Brain Stimulation System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UG3NS140720 Deep Brain Stimulation SystemDevice
Also called DBS 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.
| Study group | Role | What they receive |
|---|---|---|
| DBS Intervention | Experimental | Participants will undergo surgical implantation of the DBS leads, followed by a second surgery to implant a pulse generator (IPG) which generates electricity. Receives: Device: UG3NS140720 Deep Brain Stimulation System |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Up to 72 months
Secondary outcomes (9)
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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, Month 11
Mean EQ-5D-3L Score
The EQ-5D-3L 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.
Measured: Baseline, Month 11
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2026
- Study startDec 2026 (estimated)
- Primary completionDec 2029 (estimated)
- Study completionJun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Genotype Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gordon H. Baltuch
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Sponsor-investigator, Gordon H. Baltuch, Columbia University
- Investigators
- Gordon Baltuch, MD, Principal investigator, CUIMCSheng-Han Kuo, MD, Study director, CUIMC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07752095
- Sponsor's protocol ID
- ACYY0046
- Other IDs
- 1UG3NS140720-01
- Status last verified
- Oct 2026
- First submitted
- 29 Jul 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Deep Brain Stimulation of the Deep Cerebellar Nuclei in Subjects With Spinocerebellar Ataxia: A Safety and Feasibility Study
- ClinicalTrials.gov ID
- NCT07752095
- Acronym / sponsor ID
- ACYY0046
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Gordon H. BaltuchUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Dec 2026 (estimated)
- Primary completion
- Dec 2029 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 7 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Evaluation of the effect of Dentate Nucleus (DN) - Deep Brain Stimulation (DBS). Ataxia severity will be quantified using the Scale of Assessment and Rating of Ataxia (SARA), Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia), and kinematics.
- To characterize electrophysiological activity across the cerebellothalamocorticomuscular (CTCM) network at rest and during motor behaviors.
- To examine how DN-DBS modulates oscillatory activity and coupling across the CTCM circuit in relation to improvements in controlling ataxia.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DBS Intervention | Experimental | Device: UG3NS140720 Deep Brain Stimulation SystemParticipants will undergo surgical implantation of the DBS leads, followed by a second surgery to implant a pulse generator (IPG) which generates electricity. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Subject is aged >= 18 years and =< 60 years.
- Subject has moderate ataxia severity, as demonstrated by a Scale for Ataxia Rating and Assessment (SARA) score >= 11.
- Subject has genetic confirmation of pathological repeat expansions in ATXN1 (for SCA1, repeat size >= 40) and ATXN3 (for SCA3, repeat size >= 56).
- Subject has been on a stable and optimized standard of care (SoC) regimen for at least 3 months prior to Screening (including physical therapy) and continues to exhibit signs of cerebellar ataxia.
- Subject has a minimum 6th grade reading level, as determined by the Rapid Estimate of Adult Literacy in Medicine (REALM) scale.
Key exclusion criteria (18 listed)
- Subject is unable to provide informed consent.
- Subject has presence of overt tremor, dystonia, and/or parkinsonism, which are atypical features and could be suggestive of prominent extra-cerebellar circuit involvement that may not respond well to cerebellar DBS.
- Subject is wheelchair dependent and may be too progressed to complete gait assessments.
- Subject is unable to stop anticoagulation or platelet anti-aggregation therapy for study procedures.
- Subject has a Montreal Cognitive Assessment (MoCA) score =< 24.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UG3NS140720 Deep Brain Stimulation SystemAlso: DBS SystemThe 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. | Device | DBS Intervention |
Primary outcome
Incidence of treatment-related adverse events
Time frame: Up to 72 months
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).
9 secondary outcomes
Mean Scale for Assessing and Rating of Ataxia (SARA) Score
Time frame: Baseline, Month 11
Mean Patient-Reported Outcome Measures - Ataxia (PROM-Ataxia) Scale Score
Time frame: Baseline, Month 11
Mean Cerebellar Cognitive Affective Syndrome Scale (CCAS) Score
Time frame: Baseline, Month 11
Mean Patient Health Questionnaire (PHQ-9) Score
Time frame: Baseline, Month 11
Mean Generalized Anxiety Disorder (GAD) Scale Score
Time frame: Baseline, Month 11
Mean Patient Global Impression (PGI) Score
Time frame: Baseline, Month 11
Mean Gait Measure of Ataxia
Time frame: Baseline, Month 11
Mean Postural Kinematic Measure of Ataxia
Time frame: Baseline, Month 11
Mean EQ-5D-3L Score
Time frame: Baseline, Month 11
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2026.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Gordon Baltuch, MD, PhD contact
Gordon Baltuch, MD principal investigator
CUIMC
Sheng-Han Kuo, MD study director
CUIMC
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing UG3NS140720 Deep Brain Stimulation System in people with Spinocerebellar Ataxia Genotype Type 1 and Spinocerebellar Ataxia Genotype Type 3, run by Gordon H. Baltuch at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Natasha Desai (347-882-4315, nd2528@cumc.columbia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-related adverse events (time frame: Up to 72 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2026.