Cerebellar DBS in SCA1 and SCA3 Study

Starting soon · Not applicable

Conditions studied: Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 3

In brief

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.

Key facts

Study ID

NCT07752095

Run by

Gordon H. Baltuch

People needed

12

Starts

2026-10-01

Expected to finish

2030-06-01

Last updated by the study team

2026-08-07

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.
• 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.

Where it is running

• Columbia University Irving Medical Center — New York, New York, United States
• Cleveland Clinic — Cleveland, Ohio, United States

Full record on ClinicalTrials.gov

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