Deep Brain Stimulation for Ataxia

Overseen By Julia C Gonzalez, BA

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: University of Florida

Disqualifiers: Severe cognitive impairment, Psychiatric illness, Refractory epilepsy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 19

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Who Is on the Research Team?

Coralie de Hemptinne, PhD

Principal Investigator

University of Florida

Are You a Good Fit for This Trial?

This trial is for adults with a specific type of movement disorder called spinocerebellar ataxia type 6 (SCA6). Participants will be part of a small group, only 5 people, to test the safety and potential benefits of a new treatment involving brain stimulation.

Inclusion Criteria

I have tested positive for SCA6.

Ability to give informed consent for the study

Ability to understand the study protocol

Exclusion Criteria

Severe cognitive impairment or dementia, defined as a score <21 on the Montreal Cognitive Assessment (MOCA)

My ataxia is not caused by SCA6 but may be due to another condition.

I do not have any bleeding disorders, uncontrolled seizures, or conditions that increase my risk for surgery.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgical Implantation

Participants undergo surgical implantation of DBS leads targeting the motor interposed nucleus of the cerebellum

1 month

1 visit (in-person)

Conventional DBS Programming

Participants begin conventional DBS programming to identify optimal stimulation parameters

8 months

Multiple visits (in-person)

Adaptive DBS Implementation

Transition to adaptive DBS where stimulation amplitude is automatically adjusted based on recorded neural activity

15 months

Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months

18 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Interposed Nucleus aDBS

Trial Overview

The study tests cerebellar adaptive deep brain stimulation (aDBS), where surgical implants in the brain help control movement. Over two years, participants will visit the clinic up to 18 times for adjustments and monitoring how well the treatment works.

How Is the Trial Designed?

Group I: Adaptive Deep Brain Stimulation (aDBS) Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials

1,428

Recruited

987,000+

Raynor Cerebellum Project

Collaborator