Cerebellar AtaxiaNCT03341416

Effects of Deep Brain Stimulation of the Dentate Nucleus on Cerebellar Ataxia

A study testing deep brain stimulation on stimulation - active in people with Cerebellar Ataxia, run by University of Sao Paulo General Hospital at 1 site in Brazil.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Cerebellar Ataxia
What is being tested?: deep brain stimulation on stimulation - active
Type of treatment: Medical device
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 10 participants
Where: 1 siteBrazil
Study dates: 1 Sep 2017to 1 Jan 2019
Phase: Interventional
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients with diagnosis of chronic cerebellar ataxia (> 6 months);
Age ≥18 years;
Be able to understand study protocol;
Signed, written informed consent (approved by the Institutional Ethics Committee) obtained prior to any study procedure;
Refractory symptoms response to first, second and third line pharmacological treatment;
Daily living activities impaired because the ataxia;
Be able to undergo surgery procedures.

Who cannot join

Abuse of alcohol, drugs
Known psychiatric conditions
Contraindications to DBS
Heart failure or cardiac disease that contraindicates surgery procedures;
Pacemaker or other stimulators implanted active;
Positive blood ß-HCG test for women;
Other medical conditions demand hospitalization; Participation in other clinical studies at the same time;

Trial locations and contacts

1 site in 1 country.

Brazil1 site, 1 recruiting

Rubens Cury
São Paulo, São Paulo
Rubens Cury, MD PhD, 5511972838184, rubens_cury@usp.br
carina franca, MD, 1131590293
Recruiting

Other trials in Brazil

About this study

Official title: Deep Brain Stimulation on Cerebellar Ataxia

Cerebellar ataxias are a group of disorders caused by cerebellar affections, for which currently no specific treatment is available. Some limited studies verified the effects of cerebellar transcranial magnetic stimulation (TMS) on ataxic symptoms, with good results. The hypothesis is that cerebellar TMS could improve ataxic symptoms in some patients and in these patients, chronic cerebellar stimulation through deep brain stimulation could be a therapeutic option. The rationale is to stimulate the dentate nucleus of the cerebellum in order to balance the functional asymmetry observed between both motor cortices after chronic cerebellar lesions.

Read the detailed description

Ten patients with cerebellar ataxia will be included in our protocol. Ataxia might be due to several aetiologies, from degenerative to genetic and vascular diseases. The initial focus is vascular and spinal cerebellar ataxias. The participants will be submitted to a neuronavigation protocol for the precise location of the dentate nucleus contralateral to the most symptomatic side. After that, the participants will be randomly assigned to 5 active or 5 placebo sessions of 1Hz TMS over the located area. After the first 5 sessions and a period of at least 4 weeks washout, the participants will cross over and receive other 5 sessions, active or sham. Clinical and video evaluations will be conducted before and after active and sham cluster of sessions. The good responders, i.e., with over than 30% of improvement after the active section will be eligible to bilateral dentate nucleus DBS. After that, again, a cross-over double blind on-off stimulation will be performed. Each period (on or 0ff-stimulation) will last 4 weeks.

Conditions: Cerebellar Ataxia
Being tested: deep brain stimulation on stimulation - active, deep brain stimulation off stimulation - sham
Keywords: cerebellar ataxia, stroke, Spinocerebellar ataxia, deep brain stimulation, dentate nucleus
Medical subject headings: Cerebellar Ataxia, Stroke, Spinocerebellar Ataxias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

deep brain stimulation on stimulation - activeDevice
bilateral deep brain stimulation reaching the dentate nucleus turned ON - phase active
deep brain stimulation off stimulation - shamDevice
bilateral deep brain stimulation reaching the dentate nucleus turned OFF - phase sham

Study groups in NCT03341416
Study group	Role	What they receive
Device - deep brain stimulation ON	Experimental	Intervention type: device (deep brain stimulation of the dentate nucleus in cerebellum). The intervention is a device called deep brain stimulation bilaterally placed in the sub thalamic nucleus. The stimulation will remained turned ON during 3 months - phase 1 - blinded and continuous during the open-label phase Receives: Device: deep brain stimulation on stimulation - active
Device - deep brain stimulation Sham	Sham comparator	Sham stimulation: device (deep brain stimulation of the dentate nucleus in cerebellum). Intervention type: device (deep brain stimulation of the dentate nucleus in cerebellum). The intervention is a device called deep brain stimulation bilaterally placed in the sub thalamic nucleus. During the sham stimulation the intervention will remained turned OFF during 3 months Receives: Device: deep brain stimulation off stimulation - sham

What is being measured

The primary outcome is the main question the study is designed to answer.

Primary outcome - change in Scale for the assessment and rating of ataxia - SARA
To evaluate prospectively the effect of DN-DBS in ataxia. The primary outcome is the change between the Scale for the assessment and rating of ataxia (SARA, ranging from 0-40, higher scores mean more severe ataxia) at baseline and after surgery during the active DBS-ON phase
Measured: change in the SARA score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.

Secondary outcomes (2)

Secondary outcome - change in tremor score using the Fahn Tolosa Marin Scale
To evaluate prospectively the effect of DN-DBS on tremor. The secondary outcome is the change between the Fahn Tolosa Marin Scale (ranging from 0-40, higher scores mean worse tremor) at baseline and after surgery during the active DBS-ON phase
Measured: change in the Fahn Tolosa Marin Scale score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.
Secondary outcome - quality of life
To evaluate prospectively the effect of DN-DBS on quality of life. The secondary outcome is the change between the World Health Organization Quality of Life (WHOQOL-bref, 26 items, which measure the following broad domains: physical health, psychological health, social relationships, and environment, higher scores mean better quality of life) at baseline and after surgery during the active DBS-ON phase
Measured: change in the WHOQOL-bref score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Sep 20171 Jan 2019

First posted14 Nov 2017
Study start1 Sep 2017
Primary completion1 Jul 2018 (estimated)
Study completion1 Jan 2019 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Cerebellar AtaxiaStudies of the same condition, open and under way.
More trials run by University of Sao Paulo General HospitalThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Model description: After surgery patients will undergo a first washout period intended to allow their ataxia return to baseline (preoperative levels), thus avoiding post surgery effects bias. Phase 1: Participants will be randomized to either ON or OFF stimulation. The assigned stimulation will be performed for one week. Phase 2: Open label phase. All patients are in the ON stimulation condition and will be followed for 6 months. Duration: Phase 1 will last from one to 2 months and phase 2 will last six months.
Masking: Triple blind
Who is masked: Participant, Care provider, Outcomes assessor
Enrolment: 10 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03341416
Sponsor's protocol ID: 102178/2015
Status last verified: Nov 2017
First submitted: 30 Oct 2017
Last updated: 17 Nov 2017
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

Each site lists its own contact. Pick the site nearest to you.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
RecruitingEarly Phase 1
Aprepitant for the Treatment of CLIFAHDD
CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders

All Cerebellar Ataxia trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Deep Brain Stimulation on Cerebellar Ataxia
ClinicalTrials.gov ID: NCT03341416
Acronym / sponsor ID: 102178/2015
Conditions: Cerebellar Ataxia
Keywords: cerebellar ataxia, stroke, Spinocerebellar ataxia, deep brain stimulation, dentate nucleus
Study type: Interventional
Recruitment status: Unknown status
Enrolment: 10 participants (estimated)
Lead sponsor: University of Sao Paulo General HospitalUniversity, hospital or other
Study start: 1 Sep 2017 (actual)
Primary completion: 1 Jul 2018 (estimated)
Study completion: 1 Jan 2019 (estimated)
First posted: 14 Nov 2017 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Triple blind (participant, care provider, outcomes assessor)
Number of arms: 2
Model description: After surgery patients will undergo a first washout period intended to allow their ataxia return to baseline (preoperative levels), thus avoiding post surgery effects bias. Phase 1: Participants will be randomized to either ON or OFF stimulation. The assigned stimulation will be performed for one week. Phase 2: Open label phase. All patients are in the ON stimulation condition and will be followed for 6 months. Duration: Phase 1 will last from one to 2 months and phase 2 will last six months.

Arm	Type	Receives
Device - deep brain stimulation ON	Experimental	Device: deep brain stimulation on stimulation - activeIntervention type: device (deep brain stimulation of the dentate nucleus in cerebellum). The intervention is a device called deep brain stimulation bilaterally placed in the sub thalamic nucleus. The stimulation will remained turned ON during 3 months - phase 1 - blinded and continuous during the open-label phase
Device - deep brain stimulation Sham	Sham comparator	Device: deep brain stimulation off stimulation - shamSham stimulation: device (deep brain stimulation of the dentate nucleus in cerebellum). Intervention type: device (deep brain stimulation of the dentate nucleus in cerebellum). The intervention is a device called deep brain stimulation bilaterally placed in the sub thalamic nucleus. During the sham stimulation the intervention will remained turned OFF during 3 months

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Patients with diagnosis of chronic cerebellar ataxia (> 6 months);
Age ≥18 years;
Be able to understand study protocol;
Signed, written informed consent (approved by the Institutional Ethics Committee) obtained prior to any study procedure;
Refractory symptoms response to first, second and third line pharmacological treatment;

Key exclusion criteria (7 listed)

Abuse of alcohol, drugs
Known psychiatric conditions
Contraindications to DBS
Heart failure or cardiac disease that contraindicates surgery procedures;
Pacemaker or other stimulators implanted active;

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
deep brain stimulation on stimulation - activebilateral deep brain stimulation reaching the dentate nucleus turned ON - phase active	Device	Device - deep brain stimulation ON
deep brain stimulation off stimulation - shambilateral deep brain stimulation reaching the dentate nucleus turned OFF - phase sham	Device	Device - deep brain stimulation Sham

Primary outcome

Primary outcome - change in Scale for the assessment and rating of ataxia - SARA
Time frame: change in the SARA score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.
To evaluate prospectively the effect of DN-DBS in ataxia. The primary outcome is the change between the Scale for the assessment and rating of ataxia (SARA, ranging from 0-40, higher scores mean more severe ataxia) at baseline and after surgery during the active DBS-ON phase

2 secondary outcomes

Secondary outcome - change in tremor score using the Fahn Tolosa Marin Scale
Time frame: change in the Fahn Tolosa Marin Scale score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.
Secondary outcome - quality of life
Time frame: change in the WHOQOL-bref score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2017.
Individual participant dataSharing planned: No

Related trials for Cerebellar Ataxia

RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
RecruitingEarly Phase 1
Aprepitant for the Treatment of CLIFAHDD
CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Rubens Cury

São Paulo, Brazil

5511972838184

rubens_cury@usp.br

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A study testing deep brain stimulation on stimulation - active in people with Cerebellar Ataxia, run by University of Sao Paulo General Hospital at 1 site in Brazil.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 17 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Primary outcome - change in Scale for the assessment and rating of ataxia - SARA (time frame: change in the SARA score between the baseline versus after surgery during ON-stimulation deep brain stimulation phase. This phase lasts 1 month, so the evaluation will be performed 01 months after the stimulation is turned on.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03341416.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2017.