Bilateral Internal Pallidum Stimulation in Primary Generalized Dystonia - Full Text View

Sponsors and Collaborators:	Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) Princess Beatrix Fund, The Netherlands
Information provided by:	Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
ClinicalTrials.gov Identifier:	NCT00272246

Purpose

Primary generalized dystonia, also called idiopathic torsion dystonia or dystonia musculorum deformans is a disabling neurological condition which usually starts in childhood, mostly in a lower limb and spreads to other body parts as the disease progresses. Medical treatment is usually ineffective and most patients become wheelchair bound or even bedridden. Recently several case series and one RCT reported favourable results of bilateral deep brain stimulation (DBS) of the globus pallidus internus (GPi) for primary generalized dystonias.

However, the number of patients treated with bilateral GPi stimulation is still limited. Therefore, we propose a RCT to investigate whether bilateral DBS of the GPi is an effective and safe treatment in patients with primary generalized dystonia.

Condition	Intervention	Phase
Dystonia Primary Generalized Dystonia	Procedure: deep brain stimulation of the internal globus pallidus	Phase II Phase III

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Efficacy Study

Resource links provided by NLM:

Genetics Home Reference related topics: early-onset primary dystonia familial paroxysmal nonkinesigenic dyskinesia

MedlinePlus related topics: Dystonia

U.S. FDA Resources

Further study details as provided by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA):

Primary Outcome Measures:

change in BFMDRS-motor score from baseline to six months after stimulation equipment implantation

Secondary Outcome Measures:

BFMDRS-disability score
MOS-SF-36 (Medical Outcome Scale)
GOS (Global Outcome Scale)
Neuropsychological and neuropsychiatric validated scales
Questionnaire designed for the registration of the number and extent of morbidity due to the surgical
procedure, stimulation equipment and active stimulation

Estimated Enrollment:	24
Study Start Date:	February 2005
Estimated Study Completion Date:	December 2007

Detailed Description:

Study design and methods:

This is a randomised, double-blind, placebo-controlled, multicentre study comparing the effect of bilateral DBS of the GPi with placebo in patients with invalidating primary generalized dystonia. After inclusion in the study (see in/exclusioncriteria), a preoperative formal baseline assessment is performed. Within six weeks after the inclusion the patients undergo the stereotactic implantation of the stimulation equipment in the GPi. Immediately after surgery the patients are randomly allocated to direct stimulation (treatment group) or delayed stimulation (control group), in which the stimulator will se switched on 0 Volt before discharge from the hospital by an independent neurologist or neurosurgeon. The patients in both groups are able to control whether the stimulation is active (>0 Volt) or not. In this way the patients and the assessors are blinded. In each centre the postoperative care of the patients will take place according to a stratified stimulation parameter protocol which assures comparable information in both groups. Six months postoperatively, the first formal outcome assessment is performed by the same blinded assessor. After completion of this assessment the stimulators in the control group are activated by an independent neurologist or neurosurgeon. From this moment, all 24 patients have active DBS.

However, the patients and blinded assessor remain unaware of the result of the randomisation. All patients are followed for another six months after which the second outcome assessment is made by the same assessor. Four Dutch centres experienced in DBS have agreed to participate in the study. It is estimated that each centre will include at least 4 patients. Because these centra are geographically spread over the country patients can be operated and controlled nearby.

Eligibility

Ages Eligible for Study:	12 Years to 60 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Invalidating primary generalized dystonia (BFMDRS-motor part >= 35)
Disease duration >= 5 yrs
Age at surgery between 12 and 60
Insufficient improvement with pharmacological and physical therapy
Signed informed consent, for patients < 19 yrs of age a written consent of the parent(s) or caretaker(s) is needed.

Exclusion Criteria:

Mental retardation or dementia (MMSE <24)
Depressive state (HADS>21)
History of former stereotactic brain surgery
Severe brain atrophy
General surgical contraindications, such as uncontrolled hypertension or diabetes mellitus, cardiovascular or pulmonary risks, disturbances of bloodclotting.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00272246

Contacts

Contact: Elisabeth MJ Foncke, MD	0031205669111 ext 58966	e.m.foncke@amc.uva.nl
Contact: Hans Speelman, MD,PhD	0031205669111 ext 63963	j.d.speelman@amc.uva.nl

Locations

Netherlands
Department of Neurology, Movement Disorders Center	Recruiting
Amsterdam, Netherlands, 1100 DD
Contact: Elisabeth MJ Foncke, MD 0031205669111 ext 58966 e.m.foncke@amc.uva.nl
Principal Investigator: Elisabeth MJ Foncke, MD

Sponsors and Collaborators

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Princess Beatrix Fund, The Netherlands

Investigators

Study Chair:

Hans Speelman, PhD

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

More Information

Publications:

Foncke EM, Speelman JD. [Primary generalized dystonia and deep brain stimulation: a randomized, placebo-controlled, double-blind, multicentre study] Ned Tijdschr Geneeskd. 2005 May 14;149(20):1123-4. Dutch.

Study ID Numbers:	DYSPAS
Study First Received:	January 4, 2006
Last Updated:	January 4, 2006
ClinicalTrials.gov Identifier:	NCT00272246 History of Changes
Health Authority:	Netherlands: Medical Ethics Review Committee (METC)

Keywords provided by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA):

dystonia

Study placed in the following topic categories:

Dystonic Disorders
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases

Neurologic Manifestations
Dystonia
Dyskinesias

Additional relevant MeSH terms:

Dystonic Disorders
Signs and Symptoms
Movement Disorders
Nervous System Diseases

Central Nervous System Diseases
Neurologic Manifestations
Dystonia
Dyskinesias

ClinicalTrials.gov processed this record on September 01, 2009