ClinicalTrials.gov record NCT07752095

Cerebellar DBS in SCA1 and SCA3 Study

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Deep Brain Stimulation of the Deep Cerebellar Nuclei in Subjects With Spinocerebellar Ataxia: A Safety and Feasibility Study

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Age18 years old to 60 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This interventional study tests the safety and feasibility of bilateral deep cerebellar nucleus (dentate nucleus) deep brain stimulation (DBS) in people with SCA types 1 and 3. Twelve participants will receive surgical implantation of DBS leads and a pulse generator; temporary external wires and a brain-recording device will be used to record brain signals. The study will track treatment-related adverse events (up to 72 months) and measure ataxia severity, cognition, mood, patient-reported outcomes, and movement kinematics.

What it is studying. The study is testing whether bilateral deep cerebellar nucleus (dentate nucleus) DBS can be safely and feasibly implanted and used in people with SCA1 or SCA3. It also explores how stimulation changes brain electrical activity and how those changes relate to clinical measures of ataxia, cognition, mood, patient-reported functioning, and movement.

What the researchers hope to learn. Primary scientific goal: measure the incidence and nature of treatment-related adverse events associated with DN-DBS. Secondary/exploratory goals: characterize changes in ataxia severity (SARA), patient-reported ataxia impact (PROM-Ataxia), cognitive status (CCAS), mood (PHQ-9, GAD), patient global impression (PGI), gait and postural kinematics (APDM Mobility Lab sensors), and quality of life (EQ-5D). Additional goals: record and describe electrophysiological activity across the cerebellothalamocorticomuscular network and examine how DBS modulates oscillatory activity and coupling in relation to ataxia measures.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Device
  • UG3NS140720 Deep Brain Stimulation System

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participation involves neurosurgery to implant deep brain stimulation (DBS) leads in the deep cerebellar nuclei and a second surgery to implant a pulse generator (IPG). Temporary external wires and a brain-recording device will be used to record brain activity, and standard brain‑wave tests (EEGs) will be performed at rest and during movement. Clinical assessments at baseline and Month 11 include ataxia rating scales (SARA), patient questionnaires (PROM‑Ataxia, PHQ‑9, GAD, PGI), cognitive testing (CCAS), and wearable-sensor gait and posture measurements; safety and adverse-event monitoring continue for up to 72 months. You will attend multiple study visits for the surgeries, recordings, assessments, and follow-up; the registry record does not list other participant services.

How the study is structured. This is a single-group, non-masked interventional device study (treatment purpose). All enrolled participants receive bilateral DBS targeting the deep cerebellar nuclei. Procedures named in the record include surgical implantation of DBS leads, a second surgery to implant a pulse generator (IPG), use of temporary external wires and a brain-recording device to record signals, and follow-up visits with clinical ratings, questionnaires, cognitive and mood assessments, and wearable motion-sensor measurements. Safety (treatment-related adverse events) will be recorded; specific outcome assessments are scheduled at baseline and at follow-up time points including Month 11, with adverse-event monitoring up to 72 months.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on spinocerebellar ataxia

Clinical trials30 trials listed for spinocerebellar ataxiaEvery trial the registry lists for spinocerebellar ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in spinocerebellar ataxia researchLatest monthly update: September 2026. Includes: NBP trial result. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
October 2026 (estimated)
Primary completion
December 2029 (estimated)
Study completion
June 2030 (estimated)
Target enrollment
12 participants (estimated)
Last updated on registry
Aug 7, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Gordon H. Baltuch
Collaborators
  • National Institute of Neurological Disorders and Stroke (NINDS)

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (2)

Terms used on this page · explanations by MedicalUpdates.ai

Primary Objective
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
Outcome Measure
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
standard of care
Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
prior to Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
women of childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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