DystoniaDBSVopNCT03078816

Thalamic Deep Brain Stimulation for Secondary Dystonia in Children and Young Adults

A study testing Activa PC Primary Cell Neurostimulator - (Model 37601), Activa RC Rechargeable Neurostimulator - (Model 37612) and Activa SC Single Cell Neurostimulator (Models 37602/37603) in people with Dystonia, run by University of California, San Francisco at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Dystonia is a movement disorder seen in both children and adults that is characterized by "sustained or intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both." Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more recalcitrant to… Read more about this study

Who is it for?: Ages 7 to 25 years
Conditions the study is for: Dystonia
What is being tested?: Activa PC Primary Cell Neurostimulator - (Model 37601), Activa RC Rechargeable Neurostimulator - (Model 37612) and Activa SC Single Cell Neurostimulator (Models 37602/37603)
Type of treatment: Medical device
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 5 participants
Where: 1 siteUnited States
Study dates: 3 Mar 2017to 24 Jul 2019
Phase: Interventional
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 7 to 25 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Ability to give informed consent or assent for the study
Dystonia symptoms that are sufficiently severe, in spite of best medical therapy, to warrant surgical implantation of deep brain stimulators according to standard clinical criteria
Age 7-25
Stable doses of anti-dystonia medications (such as levodopa, baclofen, or diazepam) for at least 30 days prior to baseline assessment
If patient receives botulinum toxin injections, patient should be on a stable injection regimen
Intact thalamic anatomy as determined by standard clinical MRI

Who cannot join

Pregnancy or breast feeding
Major comorbidity increasing the risk of surgery (severe hypertension, severe diabetes, or need for chronic anticoagulation other than aspirin)
Inability to comply with study follow-up visits
Any prior intracranial surgery
Uncontrolled epilepsy
Immunocompromised

Show all 10Show fewer

Has an active infection
Requires diathermy, electroconvulsive therapy (ECT) or transcranial magnetic stimulation (TMS) to treat a chronic condition
Has an existing implanted neurostimulator or cardiac pacemaker.
Dystonia caused by known genetic mutation in any DYT genes

Trial locations and contacts

1 site in 1 country.

United States1 site

University of California San Francisco Hospital
San Francisco, California, 94158

Other trials in United States

About this study

Dystonia is a movement disorder seen in both children and adults that is characterized by "sustained or intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both." Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more recalcitrant to standard pharmacologic and surgical treatments including Deep Brain Stimulation (DBS). There exists a large unmet need to develop new therapeutics, treatment strategies, and outcome measures for pediatric secondary dystonia.

The investigators are proposing to investigate the ventralis oralis posterior nucleus (Vop) of the thalamus as a new target for DBS in secondary dystonia. Prior to the development of DBS, the main surgical treatment of dystonia was thalamotomy. Although there were many different targets in the thalamus, often done in staged procedures, the most common and successful targeted nuclei was the Vop, which is traditionally thought to be the pallidal receiving area. Previous lesioning of Vop produced improvements in dystonia but intolerable side effects, especially when implanted bilaterally. However, given that secondary dystonia patients were often reported to have superior results to primary dystonia it is reasonable to believe that if the side effects can be modulated, that targeting of the Vop nucleus with DBS could be a viable alternative to Globus Pallidus interna (GPi). Given that Deep Brain Stimulation is a treatment that is inherently adjustable, it is conceivable that settings on the Deep Brain Stimulation could be adjusted to allow for clinical benefit with minimal side effects. Indeed, there have been several scattered successful case reports attesting to this possibility.

Read the detailed description

Dystonia is a movement disorder seen in both children and adults that is characterized by "sustained or intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both." Secondary dystonia has evolved to refer to dystonia resulting from damage to the nervous system or degenerative disease processes. While primary dystonia is generally thought to arise from genetic causes, secondary dystonias have a variety of causes including perinatal injuries (cerebral palsy), central nervous system infections, traumatic brain injuries, and many different metabolic, neurodegenerative, and mitochondrial conditions. Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more recalcitrant to standard pharmacologic and surgical treatments including Deep Brain Stimulation. Given that most treatments for dystonia are developed for primary dystonia and then applied to secondary dystonia, it is not surprising that this effectiveness gap exists. Thus, there exists a large unmet need to develop new therapeutics, treatment strategies, and outcome measures for pediatric secondary dystonia.

Deep Brain Stimulation (DBS) is one such therapeutic intervention that has potential to improve secondary dystonia. DBS is a surgical treatment for several different movement disorders that evolved from functional stereotactic neurosurgery techniques initially used to lesion specific deep brain structures. While Essential Tremor and Idiopathic Parkinson's Disease have predictable and consistent response rates to DBS in carefully selected patients, response rates of dystonia have been much more inconsistent. One predictor of success has been the presence of DYT-1 mutation, the most common known genetic cause of primary dystonia. Success rates in DYT-1 dystonia are consistently high with reductions in dystonia typically greater than 80%. However, the results in secondary dystonia have been much more modest and inconsistent. A recent meta-analysis found that on average, dystonia symptoms as measured by common rating scales improve 23% following DBS for dystonic cerebral palsy (the most common cause of secondary dystonia), however there are frequent cases of non-responders. Additionally, there have been very few examination, radiological or laboratory predictors of good response to DBS, except for genetic confirmation of DYT-119. However, across both primary and secondary dystonia, younger age at the time of surgery (less than 21 years old) and shorter duration of symptoms (less than 15 years) have been shown to be the most likely predictive factors for a good postoperative outcome. This has led many to suggest that DBS should be offered earlier in the course of intractable dystonia, prior to the development of permanent complications such as orthopedic contractures. Thus, we are setting an upper age limit of 25 to account for the concern that earlier implantation leads to improved outcomes. The lower age limit of 7 reflects the fact that the current humanitarian exemption for DBS for dystonia currently goes down to age 7. Thus, there exists a need to both improve patient selection as well as application of DBS for secondary dystonia in children.

Conditions: Dystonia
Being tested: Activa PC Primary Cell Neurostimulator - (Model 37601), Activa RC Rechargeable Neurostimulator - (Model 37612), Activa SC Single Cell Neurostimulator (Models 37602/37603), DBS Lead - (Model 3387, DBS Extension - (Models 37085/6)
Medical subject headings: Dystonia

What participants receive

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

Activa PC Primary Cell Neurostimulator - (Model 37601)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa SC Single Cell Neurostimulator (Models 37602/37603)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
DBS Lead - (Model 3387Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
DBS Extension - (Models 37085/6)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Patient Programmer - (Model 37642)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Test Stimulator - (Model 3625)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
N'Vision Clinician Programmer - (Model 8840)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
N'Vision Software Application Card - (Model 8870)Device
Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.

Study groups in NCT03078816
Study group	Role	What they receive
DBS active	Experimental	All participants will be enrolled in DBS placement and active stimulation. The following components will be used: * Activa PC Primary Cell Neurostimulator - (Model 37601) * Activa RC Rechargeable Neurostimulator - (Model 37612) * Activa SC Single Cell Neurostimulator (Models 37602 and 37603) * DBS Lead - (Model 3387) * DBS Extension - (Models 37085/6) * Patient Programmer - (Model 37642) * Test Stimulator - (Model 3625) * N'Vision Clinician Programmer - (Model 8840) * N'Vision Software Application Card - (Model 8870) Receives: Device: Activa PC Primary Cell Neurostimulator - (Model 37601); Device: Activa RC Rechargeable Neurostimulator - (Model 37612); Device: Activa SC Single Cell Neurostimulator (Models 37602/37603); Device: DBS Lead - (Model 3387; Device: DBS Extension - (Models 37085/6); Device: Patient Programmer - (Model 37642); Device: Test Stimulator - (Model 3625); Device: N'Vision Clinician Programmer - (Model 8840); Device: N'Vision Software Application Card - (Model 8870)

What is being measured

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

Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia
Change from Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Measured: Change from baseline to 12 months postoperatively
Percent Change in Pediatric Quality of Life Inventory (PedsQL)
Quality of life measure, scored 0-100, larger scores indicate greater hinderance (ie. lower quality of life)
Measured: baseline to 12 months postoperatively
Change in Barry Albright Dystonia Rating Scale
Severity scale for secondary dystonia, range 0-32, higher scores indicates more severe dystonia
Measured: Change from baseline to 12 months postoperatively
Change in Blinded Burke-Fahn-Marsden Dystonia Rating Scale
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia. These ratings were carried out retroactively by a neurologist who was unfamiliar with the four study participants and who had no knowledge of their unblinded scores.
Measured: change from baseline to 12 months postoperatively

Secondary outcomes (8)

Change in Modified Ashworth Scale - Upper Limbs
Measure of spasticity, range 0-32, higher values indicate more spasticity
Measured: Change from baseline to 12 months postoperatively
Change in Diadochokinetic Syllable Rates
Articulation, range (min 6- no upper limit), longer times indicate less articulation/more difficulty with speech
Measured: Change from baseline to 12 months postoperatively
Children's Memory Scale
Will include the following subtests: Memory for Faces, Dot Locations, and Digit Span
Measured: Change from baseline to 12 months postoperatively
Change in Behavioral Assessment System, 3rd Edition: Self Report of Personality
Mood and behavior assessment, main use as a screening tool for depression.
Measured: Change from baseline to 12 months postoperatively
Change in Modified Ashworth Scale Spasticity Ratings - Lower Limbs
This scale is used to measure spasticity, which is a velocity-dependent increase in muscle stretch reflexes associated with increased muscle tone as a component of upper motor neuron syndrome. It is scored 0-4 with higher scores indicating greater severity.
Measured: Change from baseline to 12 months postoperatively
Change in Kaufman Brief Intelligence Test - Second Addition
Kaufman Brief Intelligence Test Second Edition (KBIT-2) is a brief measure of verbal and nonverbal intelligence used with individuals ages 4 through 90 years, raw scores 0 - unlimited, with higher scores indicating higher ability.
Measured: baseline to 12 months postoperatively
Change in Burke-Fahn-Marsden Dystonia Disability Subscale
This scale is a measurement of quality of life related to dystonia, with lower scores indicating greater quality of life and high scores indicating more hinderance. It is scored 0-100.
Measured: baseline to 12 months postoperatively
Change in Modified Unified Parkinson's Disease Rating Scale - Second Edition
This scale is a measurement of quality of life related to dystonia, with lower scores indicating greater quality of life and high scores indicating more hinderance. It is scored 0-199
Measured: baseline to 12 months posoperatively

Timeline

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

3 Mar 201724 Jul 2019

First posted13 Mar 2017
Study start3 Mar 2017
Primary completion24 Jul 2019
Study completion24 Jul 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (18)Papers the sponsor has linked to this study.
Other trials for DystoniaStudies of the same condition, open and under way.
More trials run by University of California, San FranciscoThe 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 5 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03078816
Sponsor's protocol ID: 123822A
Acronym: DBSVop
Status last verified: Sep 2020
First submitted: 26 Apr 2016
Last updated: 8 Oct 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: Yes
Individual data sharing: No

Publications

Mink JW. Special concerns in defining, studying, and treating dystonia in children. Mov Disord. 2013 Jun 15;28(7):921-5. doi: 10.1002/mds.25548.
PubMed 23893449
Sironi VA. Origin and evolution of deep brain stimulation. Front Integr Neurosci. 2011 Aug 18;5:42. doi: 10.3389/fnint.2011.00042. eCollection 2011.
PubMed 21887135
Franzini A, Cordella R, Messina G, Marras CE, Romito LM, Albanese A, Rizzi M, Nardocci N, Zorzi G, Zekaj E, Villani F, Leone M, Gambini O, Broggi G. Targeting the brain: considerations in 332 consecutive patients treated by deep brain stimulation (DBS) for severe neurological diseases. Neurol Sci. 2012 Dec;33(6):1285-303. doi: 10.1007/s10072-012-0937-9. Epub 2012 Jan 24.
PubMed 22271259
Panov F, Gologorsky Y, Connors G, Tagliati M, Miravite J, Alterman RL. Deep brain stimulation in DYT1 dystonia: a 10-year experience. Neurosurgery. 2013 Jul;73(1):86-93; discussion 93. doi: 10.1227/01.neu.0000429841.84083.c8.
PubMed 23615098
Cif L, Vasques X, Gonzalez V, Ravel P, Biolsi B, Collod-Beroud G, Tuffery-Giraud S, Elfertit H, Claustres M, Coubes P. Long-term follow-up of DYT1 dystonia patients treated by deep brain stimulation: an open-label study. Mov Disord. 2010 Feb 15;25(3):289-99. doi: 10.1002/mds.22802.
PubMed 20063427
Air EL, Ostrem JL, Sanger TD, Starr PA. Deep brain stimulation in children: experience and technical pearls. J Neurosurg Pediatr. 2011 Dec;8(6):566-74. doi: 10.3171/2011.8.PEDS11153.
PubMed 22132914
Koy A, Hellmich M, Pauls KA, Marks W, Lin JP, Fricke O, Timmermann L. Effects of deep brain stimulation in dyskinetic cerebral palsy: a meta-analysis. Mov Disord. 2013 May;28(5):647-54. doi: 10.1002/mds.25339. Epub 2013 Feb 13.
PubMed 23408442
Vidailhet M, Jutras MF, Grabli D, Roze E. Deep brain stimulation for dystonia. J Neurol Neurosurg Psychiatry. 2013 Sep;84(9):1029-42. doi: 10.1136/jnnp-2011-301714. Epub 2012 Nov 15.
PubMed 23154125
Hyam JA, Owen SL, Kringelbach ML, Jenkinson N, Stein JF, Green AL, Aziz TZ. Contrasting connectivity of the ventralis intermedius and ventralis oralis posterior nuclei of the motor thalamus demonstrated by probabilistic tractography. Neurosurgery. 2012 Jan;70(1):162-9; discussion 169. doi: 10.1227/NEU.0b013e3182262c9a.
PubMed 22158304
Andrew J, Fowler CJ, Harrison MJ. Stereotaxic thalamotomy in 55 cases of dystonia. Brain. 1983 Dec;106 ( Pt 4):981-1000. doi: 10.1093/brain/106.4.981.
PubMed 6360306
Burchiel KJ. Thalamotomy for movement disorders. Neurosurg Clin N Am. 1995 Jan;6(1):55-71.
PubMed 7696875
Cardoso F, Jankovic J, Grossman RG, Hamilton WJ. Outcome after stereotactic thalamotomy for dystonia and hemiballismus. Neurosurgery. 1995 Mar;36(3):501-7; discussion 507-8. doi: 10.1227/00006123-199503000-00009.
PubMed 7753350
Kim JP, Chang WS, Chang JW. The long-term surgical outcomes of secondary hemidystonia associated with post-traumatic brain injury. Acta Neurochir (Wien). 2012 May;154(5):823-30. doi: 10.1007/s00701-012-1306-4. Epub 2012 Feb 27.
PubMed 22367408
Vidailhet M, Yelnik J, Lagrange C, Fraix V, Grabli D, Thobois S, Burbaud P, Welter ML, Xie-Brustolin J, Braga MC, Ardouin C, Czernecki V, Klinger H, Chabardes S, Seigneuret E, Mertens P, Cuny E, Navarro S, Cornu P, Benabid AL, Le Bas JF, Dormont D, Hermier M, Dujardin K, Blond S, Krystkowiak P, Destee A, Bardinet E, Agid Y, Krack P, Broussolle E, Pollak P; French SPIDY-2 Study Group. Bilateral pallidal deep brain stimulation for the treatment of patients with dystonia-choreoathetosis cerebral palsy: a prospective pilot study. Lancet Neurol. 2009 Aug;8(8):709-17. doi: 10.1016/S1474-4422(09)70151-6. Epub 2009 Jul 1.
PubMed 19576854
Binder DK, Rau GM, Starr PA. Risk factors for hemorrhage during microelectrode-guided deep brain stimulator implantation for movement disorders. Neurosurgery. 2005 Apr;56(4):722-32; discussion 722-32. doi: 10.1227/01.neu.0000156473.57196.7e.
PubMed 15792511

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

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

Recruiting
Impact of rTMS on Abnormal Cortical fMRI in Patients With Dystonia
Dystonia Disorder
Recruiting
Intrathecal Baclofen and Pediatric Dystonia
Dystonic Cerebral Palsy
Recruiting
Focused Ultrasound Unilateral Pallidotomy for Medication-Refractory Limb Dystonia
Limb Dystonia, Idiopathic Dystonia
Recruiting
Deep Brain Stimulation Surgery for Movement Disorders
Parkinson's Disease, Essential Tremor
Active, not recruitingPhase 1/2
Deep Brain Stimulation Surgery for Focal Hand Dystonia
Dystonia, Focal Dystonia

All Dystonia trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Thalamic Deep Brain Stimulation for Secondary Dystonia in Children and Young Adults
ClinicalTrials.gov ID: NCT03078816
Acronym / sponsor ID: DBSVop / 123822A
Conditions: Dystonia
Study type: Interventional
Recruitment status: Completed
Enrolment: 5 participants (actual)
Lead sponsor: University of California, San FranciscoUniversity, hospital or other
Study start: 3 Mar 2017 (actual)
Primary completion: 24 Jul 2019 (actual)
Study completion: 24 Jul 2019 (actual)
First posted: 13 Mar 2017 (actual)
Results: Posted 8 Oct 2020

Clinical summary

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

Dystonia is a movement disorder seen in both children and adults that is characterized by "sustained or intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both." Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more recalcitrant to standard pharmacologic and surgical treatments including Deep Brain Stimulation (DBS). There exists a large unmet need to develop new therapeutics, treatment strategies, and outcome measures for pediatric secondary dystonia.

Detailed description

Dystonia is a movement disorder seen in both children and adults that is characterized by "sustained or intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both." Secondary dystonia has evolved to refer to dystonia resulting from damage to the nervous system or degenerative disease processes. While primary dystonia is generally thought to arise from genetic causes, secondary dystonias have a variety of causes including perinatal injuries (cerebral palsy), central nervous system infections, traumatic brain injuries, and many different metabolic, neurodegenerative, and mitochondrial conditions. Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more recalcitrant to standard pharmacologic and surgical treatments including Deep Brain Stimulation. Given that most treatments for dystonia are developed for primary dystonia and then applied to secondary dystonia, it is not surprising that this effectiveness gap exists. Thus, there exists a large unmet need to develop new therapeutics, treatment strategies, and outcome measures for pediatric secondary dystonia.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
DBS active	Experimental	Device: Activa PC Primary Cell Neurostimulator - (Model 37601), Device: Activa RC Rechargeable Neurostimulator - (Model 37612), Device: Activa SC Single Cell Neurostimulator (Models 37602/37603), Device: DBS Lead - (Model 3387, Device: DBS Extension - (Models 37085/6), Device: Patient Programmer - (Model 37642), Device: Test Stimulator - (Model 3625), Device: N'Vision Clinician Programmer - (Model 8840), Device: N'Vision Software Application Card - (Model 8870)All participants will be enrolled in DBS placement and active stimulation. The following components will be used: * Activa PC Primary Cell Neurostimulator - (Model 37601) * Activa RC Rechargeable Neurostimulator - (Model 37612) * Activa SC Single Cell Neurostimulator (Models 37602 and 37603) * DBS Lead - (Model 3387) * DBS Extension - (Models 37085/6) * Patient Programmer - (Model 37642) * Test Stimulator - (Model 3625) * N'Vision Clinician Programmer - (Model 8840) * N'Vision Software Application Card - (Model 8870)

Who may qualify

Ages: 7 to 25 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (6 listed)

Ability to give informed consent or assent for the study
Dystonia symptoms that are sufficiently severe, in spite of best medical therapy, to warrant surgical implantation of deep brain stimulators according to standard clinical criteria
Age 7-25
Stable doses of anti-dystonia medications (such as levodopa, baclofen, or diazepam) for at least 30 days prior to baseline assessment
If patient receives botulinum toxin injections, patient should be on a stable injection regimen

Key exclusion criteria (10 listed)

Pregnancy or breast feeding
Major comorbidity increasing the risk of surgery (severe hypertension, severe diabetes, or need for chronic anticoagulation other than aspirin)
Inability to comply with study follow-up visits
Any prior intracranial surgery
Uncontrolled epilepsy

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
Activa PC Primary Cell Neurostimulator - (Model 37601)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
Activa RC Rechargeable Neurostimulator - (Model 37612)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
Activa SC Single Cell Neurostimulator (Models 37602/37603)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
DBS Lead - (Model 3387Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
DBS Extension - (Models 37085/6)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
Patient Programmer - (Model 37642)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
Test Stimulator - (Model 3625)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
N'Vision Clinician Programmer - (Model 8840)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active
N'Vision Software Application Card - (Model 8870)Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.	Device	DBS active

Primary outcome

Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Time frame: Change from baseline to 12 months postoperatively
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia Change from Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Percent Change in Pediatric Quality of Life Inventory (PedsQL)
Time frame: baseline to 12 months postoperatively
Quality of life measure, scored 0-100, larger scores indicate greater hinderance (ie. lower quality of life)
Change in Barry Albright Dystonia Rating Scale
Time frame: Change from baseline to 12 months postoperatively
Severity scale for secondary dystonia, range 0-32, higher scores indicates more severe dystonia
Change in Blinded Burke-Fahn-Marsden Dystonia Rating Scale
Time frame: change from baseline to 12 months postoperatively
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia. These ratings were carried out retroactively by a neurologist who was unfamiliar with the four study participants and who had no knowledge of their unblinded scores.

8 secondary outcomes

Change in Modified Ashworth Scale - Upper Limbs
Time frame: Change from baseline to 12 months postoperatively
Change in Diadochokinetic Syllable Rates
Time frame: Change from baseline to 12 months postoperatively
Children's Memory Scale
Time frame: Change from baseline to 12 months postoperatively
Change in Behavioral Assessment System, 3rd Edition: Self Report of Personality
Time frame: Change from baseline to 12 months postoperatively
Change in Modified Ashworth Scale Spasticity Ratings - Lower Limbs
Time frame: Change from baseline to 12 months postoperatively
Change in Kaufman Brief Intelligence Test - Second Addition
Time frame: baseline to 12 months postoperatively
Change in Burke-Fahn-Marsden Dystonia Disability Subscale
Time frame: baseline to 12 months postoperatively
Change in Modified Unified Parkinson's Disease Rating Scale - Second Edition
Time frame: baseline to 12 months posoperatively

Results and safety

Results posted to the registry on 8 Oct 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
DBS Active	5	4

Primary outcome: Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale

Time frame: Change from baseline to 12 months postoperatively. Mean (Standard Error). Unit: change in points on a scale

Measure	DBS Active n = 4
Value	14.7 (11.8)

Primary outcome: Percent Change in Pediatric Quality of Life Inventory (PedsQL)

Time frame: baseline to 12 months postoperatively. Mean (Full Range). Unit: percent change

Measure	DBS Active n = 4
Value	27 (0 to 54.8)

Primary outcome: Change in Barry Albright Dystonia Rating Scale

Time frame: Change from baseline to 12 months postoperatively. Mean (Standard Error). Unit: average change of points on a scale

Measure	DBS Active n = 4
Value	0.25 (3.2)

Primary outcome: Change in Blinded Burke-Fahn-Marsden Dystonia Rating Scale

Time frame: change from baseline to 12 months postoperatively. Mean (Standard Error). Unit: change in points on a scale

Measure	DBS Active n = 4
Value	4.12 (3.96)

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Patients were monitored for two years following surgery.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
DBS Active	0 of 4 (0%)	4 of 4 (100%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 23893449Mink JW. Special concerns in defining, studying, and treating dystonia in children. Mov Disord. 2013 Jun 15;28(7):921-5. doi: 10.1002/mds.25548.
Background: PubMed 21887135Sironi VA. Origin and evolution of deep brain stimulation. Front Integr Neurosci. 2011 Aug 18;5:42. doi: 10.3389/fnint.2011.00042. eCollection 2011.
Background: PubMed 22271259Franzini A, Cordella R, Messina G, Marras CE, Romito LM, Albanese A, Rizzi M, Nardocci N, Zorzi G, Zekaj E, Villani F, Leone M, Gambini O, Broggi G. Targeting the brain: considerations in 332 consecutive patients treated by deep brain stimulation (DBS) for severe neurological diseases. Neurol Sci. 2012 Dec;33(6):1285-303. doi: 10.1007/s10072-012-0937-9. Epub 2012 Jan 24.
Background: PubMed 23615098Panov F, Gologorsky Y, Connors G, Tagliati M, Miravite J, Alterman RL. Deep brain stimulation in DYT1 dystonia: a 10-year experience. Neurosurgery. 2013 Jul;73(1):86-93; discussion 93. doi: 10.1227/01.neu.0000429841.84083.c8.
Background: PubMed 20063427Cif L, Vasques X, Gonzalez V, Ravel P, Biolsi B, Collod-Beroud G, Tuffery-Giraud S, Elfertit H, Claustres M, Coubes P. Long-term follow-up of DYT1 dystonia patients treated by deep brain stimulation: an open-label study. Mov Disord. 2010 Feb 15;25(3):289-99. doi: 10.1002/mds.22802.
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Individual participant dataSharing planned: No

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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.

Marta San Luciano Palenzuela, MD principal investigator

University of California, San Francisco

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

Questions about this trial

What is this clinical trial studying?

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 6 inclusion and 10 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: finished, as of the sponsor's last update on 8 Oct 2020. 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 United States. 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. 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?

Yes. Results are posted in the registry (first posted 8 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale (time frame: Change from baseline to 12 months postoperatively). The study lists 4 primary outcomes in all. 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 NCT03078816, which also lists 18 linked publications.

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 8 Oct 2020.