DystoniaPhase 2/3NCT00272246
Bilateral Internal Pallidum Stimulation in Primary Generalized Dystonia
A Phase 2/3 study testing deep brain stimulation of the internal globus pallidus in people with Dystonia and Primary Generalized Dystonia, run by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) at 1 site in Netherlands.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Dystonia, Primary Generalized Dystonia
- What is being tested?
- deep brain stimulation of the internal globus pallidus
- Type of treatment
- Surgery or procedure
- How many people
- 24 participants
- Where
- 1 siteNetherlands
- Study dates
- Feb 2005to Dec 2007
- Phase
- Phase 2/3
- 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 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Netherlands1 site, 1 recruiting
- Recruiting
Department of Neurology, Movement Disorders Center
Amsterdam, 1100 DD
Elisabeth MJ Foncke, MD, 0031205669111 ext. 58966, e.m.foncke@amc.uva.nl
Elisabeth MJ Foncke, MD
About this study
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.
Read the 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.
- Conditions
- Dystonia, Primary Generalized Dystonia
- Being tested
- deep brain stimulation of the internal globus pallidus
- Keywords
- dystonia
- Medical subject headings
- Dystonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deep brain stimulation of the internal globus pallidusProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
change in BFMDRS-motor score from baseline to six months after stimulation equipment implantation
Secondary outcomes (6)
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2006 (estimated)
- Study startFeb 2005
- Study completionDec 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for DystoniaStudies of the same condition, open and under way.
- More trials run by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)The 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 24 participants
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Princess Beatrix Muscle Foundation
- Investigators
- Hans Speelman, PhD, Study chair, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00272246
- Sponsor's protocol ID
- DYSPAS
- Status last verified
- Jan 2006
- First submitted
- 4 Jan 2006
- Last updated
- 5 Jan 2006
- Results posted
- No
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.
PubMed 15932141
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bilateral Internal Pallidum Stimulation in Primary Generalized Dystonia
- ClinicalTrials.gov ID
- NCT00272246
- Acronym / sponsor ID
- DYSPAS
- Conditions
- Dystonia, Primary Generalized Dystonia
- Keywords
- dystonia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 24 participants
- Lead sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)University, hospital or other
- Collaborators
- Princess Beatrix Muscle Foundation
- Study start
- Feb 2005
- Study completion
- Dec 2007
- First posted
- 5 Jan 2006 (estimated)
- 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.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
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 of the internal globus pallidus | Procedure or surgery | – |
Primary outcome
change in BFMDRS-motor score from baseline to six months after stimulation equipment implantation
6 secondary outcomes
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2006.
- Background: PubMed 15932141Foncke 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.
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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.
Hans Speelman, PhD study chair
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing deep brain stimulation of the internal globus pallidus in people with Dystonia and Primary Generalized Dystonia, run by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) at 1 site in Netherlands.
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 5 inclusion and 5 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 5 Jan 2006. 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 Netherlands. 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.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Elisabeth MJ Foncke, MD (0031205669111 ext. 58966, e.m.foncke@amc.uva.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
change in BFMDRS-motor score from baseline to six months after stimulation equipment implantation. 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 NCT00272246, which also lists 1 linked publication.
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 5 Jan 2006.