Interposed Nucleus aDBS for Ataxia

Adaptive Deep Brain Stimulation Targeting the Interposed Nucleus to Treat Spinocerebellar Ataxia

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Spinocerebellar Ataxia Type 6; Spinocerebellar Ataxia (SCA)
• Intervention / Treatment: Device: Deep Brain Stimulation

• Study Type: Interventional
• Enrollment (Estimated): 5
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Coralie de Hemptinne, PhD; Phone Number: (352) 733-3048; Email: Coralie.deHemptinne@neurology.ufl.edu
• Study Contact Backup: Name: Julia C Gonzalez, BA; Email: Julia.Gonzalez@neurology.ufl.edu

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32608
      • Norman Fixel Institute for Neurological Diseases
      • Principal Investigator: Coralie de Hemptinne, PhD
      • Contact: Julia C Gonzalez, BA; Email: Julia.Gonzalez@neurology.ufl.edu
      • Contact: Coralie de Hemptinne, PhD; Phone Number: 352-265-0111; Email: Coralie.deHemptinne@neurology.ufl.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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
• Refractory epilepsy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Adaptive Deep Brain Stimulation (aDBS); 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; This device will be surgically implanted into the interposed nucleus of the cerebellum.; Other Names: Medtronic Percept

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Upright Balance Assessment measured by force plate parameters	The Upright Balance Assessment is designed to evaluate walking and balance.	From baseline through study completion, about 2 years.
The Scale for the Assessment and Rating of Ataxia (SARA)	The Scale for the Assessment and Rating of Ataxia (SARA) is an 8-item performance based scale used to determine the severity of ataxia. Scoring ranges from 0 to 40, with a higher score indicating greater ataxia severity.	From baseline through study completion, about 2 years.
The Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia)	The Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia) is a 70-item scale divided into three domains of patient experiences assessing physical function, activities of daily living (ADL), and mental health. Scores range from 0 to 280, with a higher score indicating more severe symptoms and poorer quality of life.	From baseline through study completion, about 2 years.

Collaborators and Investigators

• Sponsor: University of Florida
• Collaborators: Raynor Cerebellum Project
• Investigators: Principal Investigator: Coralie de Hemptinne, PhD, University of Florida

Study record dates

Study Major Dates

• Study Start (Estimated): March 1, 2026
• Primary Completion (Estimated): March 1, 2031
• Study Completion (Estimated): March 1, 2031

Study Registration Dates

• First Submitted: January 5, 2026
• First Submitted That Met QC Criteria: January 6, 2026
• First Posted (Actual): January 8, 2026

Study Record Updates

• Last Update Posted (Actual): January 8, 2026
• Last Update Submitted That Met QC Criteria: January 6, 2026
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: deep brain stimulation; Spinocerebellar Ataxia; DBS; aDBS; Spinocerebellar Ataxia Type 6; adaptive deep brain stimulation; SCA6
• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Dyskinesias; Cerebellar Diseases; Cerebellar Ataxia; Spinocerebellar Degenerations; Ataxia; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Spinocerebellar Ataxias; Therapeutics; Surgical Procedures, Operative; Electric Stimulation Therapy; Deep Brain Stimulation
• Other Study ID Numbers: IRB202501191

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes