Interposed Nucleus aDBS for Ataxia
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age21-89
SponsorUniversity of Florida
About 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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
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)
Genetic/inherited disorders other than SCA6
Trial design
Design model
Single group
Treatments tested in this trial
Deep Brain Stimulation
DeviceThis device will be surgically implanted into the interposed nucleus of the cerebellum.
Treatment groups
5 Participants
are divided into 1 treatment groupGroup A: Adaptive Deep Brain Stimulation (aDBS)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
From baseline through study completion, about 2 years.2
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.
Time frame
From baseline through study completion, about 2 years.Secondary outcomes
1
The Upright Balance Assessment measured by force plate parameters
The Upright Balance Assessment is designed to evaluate walking and balance.
Time frame
From baseline through study completion, about 2 years.2
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.
Time frame
From baseline through study completion, about 2 years.3
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.
Time frame
From baseline through study completion, about 2 years.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
University of Florida
Lead sponsor
Raynor Cerebellum Project
Collaborator
Source ClinicalTrials.gov