NCT01975909

Brief Summary

Spinocerebellar Ataxia (SCA) refers to a family of genetic diseases that cause progressive problems with gait and balance, as well as other debilitating symptoms. This is a randomized controlled pilot study to test a novel therapeutic intervention that uses noninvasive magnetic brain stimulation to improve functional outcomes in patients with SCA. The study will include quantitative evaluations of gait, balance, and brain physiology to examine possible objective end-points for a future, larger multi-site clinical trial. The investigators anticipate that patients receiving the real intervention will show a functional gain.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Sep 2013

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2013

Completed
23 days until next milestone

First Submitted

Initial submission to the registry

September 24, 2013

Completed
1 month until next milestone

First Posted

Study publicly available on registry

November 5, 2013

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2016

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2016

Completed
7 months until next milestone

Results Posted

Study results publicly available

May 30, 2017

Completed
Last Updated

May 30, 2017

Status Verified

April 1, 2017

Enrollment Period

3.1 years

First QC Date

September 24, 2013

Results QC Date

March 2, 2017

Last Update Submit

April 19, 2017

Conditions

Keywords

Spinocerebellar AtaxiaSCAAtaxiaTranscranial Magnetic StimulationTMS

Outcome Measures

Primary Outcomes (1)

  • Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA)

    Assess 8 items: gait, stance, sitting, speech, dysmetria, kinetic tremor, pro- and supinations of the hand, and the heel-shin slide. Each item is scored by the physician on a 4 to 8 numerical scale based upon the amount of dysfunction observed while performing the task. The maximum possible score for the total scale is 40. Lower scores of SARA represents better task performance.

    Baseline and 1 week post treatment

Secondary Outcomes (2)

  • Percent Change From Baseline to Post Treatment on the Timed 25-Foot Walk

    Baseline and 1 week post treatment

  • Percent Change From Baseline to Post Treatment on the 9-hole Peg Test

    Baseline and 1 week post treatment

Other Outcomes (3)

  • Percent Change From Baseline to Post Treatment on Gait Speed in 90 Second Walking Test

    Baseline and 1 week post treatment

  • Percent Change From Baseline to Post Treatment on Standing Postural Control

    Baseline and 1 week post treatment

  • Percent Change From Baseline to Post Treatment on Mobility and Turning

    Baseline and 1 week post treatment

Study Arms (2)

Transcranial Magnetic Stimulation (TMS)

ACTIVE COMPARATOR

A Magstim 200 (Magstim, UK) and 14cm circular coil positioned tangential to the head will be used to deliver stimuli at 100% of maximum stimulator output. Transcranial Magnetic Stimulation will be applied to three regions: 1) 4cm lateral to the right of the inion, 2) centered on the inion, 3) 4cm lateral to the left of the inion. Five pulses separated by 6 seconds will be delivered with a counter-clockwise current, followed by the same five pulses delivered with a clockwise current, for a total of 10 pulses per region, and 30 pulses per session.

Device: Transcranial Magnetic Stimulation

Sham Transcranial Magnetic Stimulation

SHAM COMPARATOR

A sham condition of Transcranial Magnetic Stimulation will be used and follow the same protocol as the active stimulation; however no magnetic pulses will be delivered through the scalp.

Device: Transcranial Magnetic Stimulation

Interventions

0.2 Hz (5 pulses every six seconds in a counter-clockwise current, followed by the same five pulses in a clockwise current); 10 pulses per region, 30 pulses per session; 5 days a week for 4 weeks.

Also known as: Magstim 200
Sham Transcranial Magnetic StimulationTranscranial Magnetic Stimulation (TMS)

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Outpatients with ataxia as diagnosed by a movement disorder specialist and confirmed by clinically obtained genetic testing of the patient and/or in a first-degree relative of the patient.
  • Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study
  • Stable on doses of all medications for at least 30 days prior to study entry and for the duration of the study
  • The ability to ambulate
  • A score of three or higher (worse) on the 'gait' subsection of the Scale of the Assessment and Rating of Ataxia (SARA) rating scale.

You may not qualify if:

  • Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study, including disorders that may affect gait or balance (i.e., stroke, arthritis, etc).
  • The presence of clinically significant abnormalities on screening CBC, CMP or EKG.
  • Pregnancy or lactation
  • Concurrent participation in another clinical study
  • A history of substance abuse
  • The presence of psychosis, bipolar disorder, untreated depression (BDI greater than or equal to 21), or history of suicide attempt.
  • Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 24).
  • Legal incapacity or limited legal capacity.
  • Ataxia derived from any cause other than genetically-confirmed SCA (including but not limited to alcoholism, head injury, Multiple Sclerosis, olivo-ponto-cerebellar atrophy or multiple system atrophy).
  • The patient's past medical history, drug dose, history of recent medication changes or duration of treatment, and combination with other CNS active drugs.
  • The published TMS guidelines review of medications to be considered with TMS.
  • History of seizures, diagnosis of epilepsy, history of abnormal (epileptiform) EEG.
  • Known metal in the head (such as a surgical aneurysm clip) or a history of prior neurosurgical procedures.
  • Ferromagnetic bioimplants activated by any electronic, mechanical or magnetic means, such as cochlear implants, pacemakers, medication pumps, vagal stimulators, deep brain stimulators, neurostimulators, biostimulators, or ventriculo-peritoneal shunts.
  • Subjects who have or might have bullet fragments or other shrapnel (veterans or workers exposed to metal in their work environment).
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Related Publications (1)

  • Manor B, Greenstein PE, Davila-Perez P, Wakefield S, Zhou J, Pascual-Leone A. Repetitive Transcranial Magnetic Stimulation in Spinocerebellar Ataxia: A Pilot Randomized Controlled Trial. Front Neurol. 2019 Feb 12;10:73. doi: 10.3389/fneur.2019.00073. eCollection 2019.

Related Links

MeSH Terms

Conditions

Spinocerebellar AtaxiasAtaxia

Interventions

Transcranial Magnetic Stimulation

Condition Hierarchy (Ancestors)

Cerebellar AtaxiaCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinocerebellar DegenerationsSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesDyskinesiasNeurologic ManifestationsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSigns and SymptomsPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Magnetic Field TherapyTherapeutics

Results Point of Contact

Title
Brad Manor, Ph.D.
Organization
Beth Israel Deaconess Medical Center

Study Officials

  • Alvaro Pascual-Leone, MD, PhD

    Beth Israel Deaconess Medical Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Neurology

Study Record Dates

First Submitted

September 24, 2013

First Posted

November 5, 2013

Study Start

September 1, 2013

Primary Completion

October 1, 2016

Study Completion

November 1, 2016

Last Updated

May 30, 2017

Results First Posted

May 30, 2017

Record last verified: 2017-04

Locations