Interposed Nucleus aDBS for Ataxia

NCT07325487 | Not Yet Recruiting DMC FDA Device

• 1 other identifier: IRB202501191
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-center, open-label study designed to evaluate the feasibility, safety, and preliminary efficacy of cerebellar adaptive deep brain stimulation (aDBS) in adults with spinocerebellar ataxia type 6 (SCA6). A total of 5 participants will be enrolled. Participants will undergo surgical implantation of deep brain stimulation (DBS) leads targeting the motor interposed nucleus of the cerebellum. The leads will be connected to one or two implantable pulse generators capable of delivering stimulation to deep brain structures and recording neural activity. Participants will complete up to 18 in-person study visits over a 24-month follow-up period. During these visits, neural signals will be recorded under varying behavioral tasks and stimulation conditions. Early study visits will be used to identify optimal stimulation parameters and neural biomarkers associated with disease state. These biomarkers will subsequently be used to implement adaptive DBS, in which stimulation amplitude is automatically adjusted in response to recorded neural activity. Study outcomes will include assessments of safety and feasibility of cerebellar aDBS, as well as preliminary evaluation of its effects on clinical measures.

Conditions

Spinocerebellar Ataxia (SCA); Spinocerebellar Ataxia Type 6

Keywords

SCA6; Spinocerebellar Ataxia; adaptive deep brain stimulation; aDBS; Spinocerebellar Ataxia Type 6; DBS; deep brain stimulation

Outcome Measures

Primary Outcomes (2)

• The identification of a physiological signal to use as the aDBS feedback signal

  Local field potentials (LFP) will be recorded in the clinic as well as chronically at home. These recordings will be analyzed to identify electrophysiological markers of disease states and therapeutic effects.

  From baseline through study completion, about 2 years.

• The incidence of unexpected adverse events and serious adverse events with aDBS compared to baseline

  Adverse events are monitored the entire time subject is enrolled in study. Unexpected and serious adverse events from baseline through study completion, about 2 years, is a primary endpoint.

  From baseline through study completion, about 2 years.

Secondary Outcomes (3)

• The Upright Balance Assessment measured by force plate parameters

  From baseline through study completion, about 2 years.

• The Scale for the Assessment and Rating of Ataxia (SARA)

  From baseline through study completion, about 2 years.

• The Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia)

  From baseline through study completion, about 2 years.

Study Arms (1)

Adaptive Deep Brain Stimulation (aDBS)

EXPERIMENTAL

Participants will undergo surgical implantation of deep brain stimulation (DBS) leads targeting the motor interposed nucleus of the cerebellum. Approximately one month after implantation, participants will begin conventional DBS (cDBS) programming to identify optimal stimulation parameters, including amplitude, contact configuration, frequency, and pulse width, and to assess stimulation-related adverse effects and device function. Approximately nine months after implantation, stimulation settings will be transitioned to adaptive DBS (aDBS), in which stimulation amplitude is automatically adjusted based on recorded neural activity. Adaptive DBS will be used to evaluate the feasibility, safety, and tolerability of cerebellar aDBS. Clinical outcomes, symptoms, and potential side effects will be assessed throughout the study using participant self-reports, validated clinical rating scales, and wearable devices to collect movement and sleep data.

Device: Deep Brain Stimulation

Interventions

Deep Brain Stimulation DEVICE

This device will be surgically implanted into the interposed nucleus of the cerebellum.

Also known as: Medtronic Percept

Adaptive Deep Brain Stimulation (aDBS)

Eligibility Criteria

• Age: 21 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A diagnosis of SCA6 by a movement disorders specialist following established criteria recommended by the Movement Disorders Society
• A positive genetic test for SCA6
• A total score ≥ 8 on the Scale of the Assessment and Rating of Ataxia (SARA) rating scale
• Ability to walk with or without support (score \<8 on the 'gait' subsection of the SARA rating scale)
• Age ≥ 21 years and \<89 years
• Ability to give informed consent for the study
• Ability to understand the study protocol

You may not qualify if:

• 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:
• Genetic/inherited disorders other than SCA6
• Acquired causes: traumatic brain injury, multiple sclerosis, paraneoplastic cerebellar degeneration, infections or post-infectious cerebellitis, autoimmune ataxias (e.g., anti-GAD, gluten ataxia)
• Toxic/metabolic causes: alcoholic cerebellar degeneration, vitamin deficiencies
• Structural, vascular, or neoplastic causes: cerebellar stroke, tumors, congenital malformations
• Suspected multiple system atrophy-cerebellar type (MSA-C)
• 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 conditions that are 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
• Requirement of diathermy, electroconvulsive therapy, or transcranial magnetic stimulation
• Pregnancy or lactation
• Active suicidal ideation, defined as a "Yes" response to questions #2-5 on the Columbia Suicide Severity Rating Scale, C-SSRS

Sponsors & Collaborators

• University of Florida: lead
• Raynor Cerebellum Project: collaborator

Study Sites (1)

Norman Fixel Institute for Neurological Diseases

Gainesville, Florida, 32608, United States

Location

MeSH Terms

Conditions

Spinocerebellar Ataxias

Interventions

Deep Brain Stimulation

Condition Hierarchy (Ancestors)

Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Electric Stimulation Therapy; Therapeutics; Surgical Procedures, Operative

Study Officials

• Coralie de Hemptinne, PhD

  University of Florida

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Coralie de Hemptinne, PhD

CONTACT

(352) 733-3048; Coralie.deHemptinne@neurology.ufl.edu

Julia C Gonzalez, BA

CONTACT

Julia.Gonzalez@neurology.ufl.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 5, 2026
• First Posted: January 8, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): March 1, 2031
• Study Completion (Estimated): March 1, 2031
• Last Updated: January 8, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)