Mode
Text Size
Log in / Sign up
N/A N=5 Treatment

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

Dystonia

Enrolled (actual)
5
Serious AEs
0.0%
Results posted
Oct 2020
Primary outcome: Primary: Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale — 14.7 change in points on a scale

Study Design & Population

Study type
Interventional
Phase
N/A
Interventions
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) (Device)
Age
Pediatric, Adult · 7+ yrs
Sex
All
Sponsor
University of California, San Francisco
Primary completion
Jul 2019

Outcome Measures

OutcomeResultp-value
PRIMARY
Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
14.7
PRIMARY
Percent Change in Pediatric Quality of Life Inventory (PedsQL)
27
PRIMARY
Change in Barry Albright Dystonia Rating Scale
0.25
PRIMARY
Change in Blinded Burke-Fahn-Marsden Dystonia Rating Scale
4.12
SECONDARY
Change in Modified Ashworth Scale - Upper Limbs
0.29
SECONDARY
Change in Diadochokinetic Syllable Rates
3.25
SECONDARY
Children's Memory Scale
SECONDARY
Change in Behavioral Assessment System, 3rd Edition: Self Report of Personality
SECONDARY
Change in Modified Ashworth Scale Spasticity Ratings - Lower Limbs
0.26
SECONDARY
Change in Kaufman Brief Intelligence Test - Second Addition
6.67
SECONDARY
Change in Burke-Fahn-Marsden Dystonia Disability Subscale
15.7
SECONDARY
Change in Modified Unified Parkinson's Disease Rating Scale - Second Edition
19.25

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 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.

Eligibility Criteria

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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
  • 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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03078816). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

Back to search