Deep Brain Stimulation of the Deep Cerebellar Nuclei in Subjects with Spinocerebellar Ataxia: A Safety and Feasibility Study
The goal/purpose of the 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.
Disclaimer: This research opportunity is not affiliated with NAF. We are sharing this information as a resource for the Ataxia community. Sharing this information does not imply endorsement by NAF.
About the Study
Eligible Ataxia Types
SCA1 and SCA3
Type of Study
Interventional Trial
Clinical Trial Phase
Phase 1
Age Range
Adult 18+
Study Start Date
10/01/2026
Estimated Completion Date
10/01/2029
IRB Approval #
IRB-ACYY0046
Location(s)
Columbia University Irving Medical Center
622 W. 168th St
New York, NY
Contact Information
Nadia Amokrane
na2855@cumc.columbia.edu
212-305-2651
Jeff Negrey
negreyj2@ccf.org
216-316-6896
What does participation in the study entail?
All participants who pass screening assessments will have DBS implant surgeries with externalization period in between followed by programming sessions and monthly chronic treatment (stimulation “on”) period and finally, an end of study visit.
What are the potential benefits/risks for participants?
Potential benefits include the possibility that the study treatment may lessen the severity of ataxia, which may give relief from some symptoms or improve quality of life.
This clinical trial carries many potential risks to the subject. The most serious of risks include infection and/or death. Potential sources of risk include:
– Risks associated with brain surgery
– Risks associated with implanting a DBS device
– Risks Associated with Temporary Percutaneous Externalization of New DN DBS Leads
– Risks Associated with DBS Stimulation
– Possible Risks to Stimulating the Specific Region of the Brain (i.e., Deep Cerebellar Nuclei) in this Research Study
– Possible risks caused by the DBS Device (i.e., device complications)
Is there financial compensation?
$150 for each in-person visit or set of visits
Is there travel reimbursement?
Hotel room(s) and parking
Who is eligible?
INCLUSION CRITERIA
1. Subject is aged > 18 years and < 60 years
2. Subject has moderate ataxia severity, as demonstrated by a Scale for Ataxia Rating and
Assessment (SARA) score > 11.
3. Subject has genetic confirmation of pathological repeat expansions in ATXN1 (for SCA1, repeat
size > 40) and ATXN3 (for SCA3, repeat size > 56).
4. 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.
5. Subject has a minimum 6th grade reading level, as determined by the Rapid Estimate of Adult
Literacy in Medicine (REALM) scale.
6. Subject is able and willing to give informed consent and attend all study visits.
7. Subject is available for all follow-up visits during the length of the study.
EXCLUSION CRITERIA
1. Subject is unable to provide informed consent.
2. 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.
3. Subject is wheelchair dependent and may be too progressed to complete gait assessments.
4. Subject is unable to stop anticoagulation or platelet anti-aggregation therapy for study procedures.
5. Subject has a Montreal Cognitive Assessment (MoCA) score < 24.
6. Subject has a diagnosis of epilepsy.
7. Subject has a major active psychiatric illness that may interfere with the study, such as psychiatric disorders or severe personality disorders.
8. Subject has an untreated or inadequately treated major depressive episode.
9. Subject is pregnant, lactating, or planning to become pregnant during the study.
10. For women of childbearing potential, subject is unable or unwilling to use accepted contraception methods.
11. Subject is unable to communicate with investigators or staff.
12. 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.
13. 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.
14. 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.
17. Subject is at risk for suicide, defined by a total score > 3 on the Columbia Suicide Severity Rating Scale (C-SSRS).
18. Patients who are not able to read, speak and/or understand English.