DystoniaPhase 2NCT00105430
Deep Brain Stimulation for Cervical Dystonia
A Phase 2 study testing Deep Brain Stimulation in people with Dystonia and Generalized Primary Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
This study will evaluate the effectiveness of deep brain stimulation (DBS) in treating primary generalized dystonia. Patients with dystonia have muscle spasms that cause uncontrolled twisting and repetitive movement or abnormal postures. Read more about this study
- Who is it for?
- Ages 7 to 70 years
- Conditions the study is for
- Dystonia, Generalized Primary Dystonia
- What is being tested?
- Deep Brain Stimulation
- Type of treatment
- Surgery or procedure
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2005to Jul 2006
- Phase
- Phase 2
- 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 7 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be eligible for entry into the study, candidates must meet all the following criteria:
- Patients greater than 7 years of age with generalized primary dystonia.
- Patients who are refractory to medical treatment (including levodopa, anticholinergics, benzodiazepines, Baclofen) who are considered surgical candidates. Patients will be determined to have medical refractory dystonia after having been treated by at least two medications of two different groups at the maximum recommended dose for a duration of at least two months.
- In case of treatment by intrathecal Baclofen delivered by a pump, a change to oral Baclofen must be performed, so that the pump can be taken out.
- Patients must have a minimum BFM score of 20 on optimal medical therapy.
- Adults must be mentally competent to consent for entrance into the protocol at the time of admission.
- Patients must have a signed DPA for research purposes.
Who cannot join
- Candidates will be excluded if:
- The patient has had previous lesioning surgery including radiofrequency lesioning of deep nuclei (thalamus, pallidum, STN).
- The patient currently has a functioning and effective stimulator in deep brain nuclei (thalamus, pallidum, STN).
- The patient is not able to tolerate surgery, as determined by the preoperative evaluation.
- The patient has a coagulopathy demonstrated by an abnormal prothrombin time, activated partial thromboplastin time, or thrombocytopenia (platelet count less than 150,000 platelets/mm(3)).
- The patient has an acute or untreated infection (viral, bacterial or fungal).
Show all 11Show fewer
- The patient has a contraindication to MR-imaging such as previous surgery that involved placement of metal objects that could cause tissue damage or produce image artifacts.
- The patient has another chronic neurologic disorder.
- The patient is pregnant at the time of surgery.
- The patient has epilepsy.
- The patient does not have access to proper follow-up care at home and may not be eligible for the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, 20892
About this study
Official title: Deep Brain Stimulation of the Globus Pallidus Interna or the Subthalamic Nucleus for Treatment of Generalized Primary Dystonia
This study will evaluate the effectiveness of deep brain stimulation (DBS) in treating primary generalized dystonia. Patients with dystonia have muscle spasms that cause uncontrolled twisting and repetitive movement or abnormal postures. Medical therapies are available, but not all patients get adequate relief from the abnormal movements or the pain associated with them. DBS is a surgical procedure that interrupts neuronal circuits in the Gpi and STN, areas of the basal ganglia of the brain that do not work correctly in patients with dystonia. The surgery results in decreased movement and therefore may lessen patients' symptoms and pain.
Patients 7 years of age and older with generalized dystonia that does not respond to medical treatment may be eligible for this study. Candidates are screened with blood and urine tests, chest x-ray, and an electrocardiogram in patients 35 years of age or more.
Read more
Participants undergo the following tests and procedures:
- Magnetic resonance imaging. MRI uses a magnetic field and radio waves to produce images of the brain. The patient lies on a table that is moved into the scanner (a narrow cylinder), wearing earplugs to muffle loud knocking and thumping sounds that occur during the scanning process. The procedure usually lasts about 45 to 90 minutes, during which the patient is asked to lie still for up to 15 minutes at a time.
- Transcranial magnetic stimulation. This procedure maps brain function. A wire coil is held on the scalp, and a brief electrical current is passed through the coil, creating a magnetic pulse that stimulates the brain. During the stimulation, the patient may be asked to tense certain muscles slightly or perform other simple actions. The stimulation may cause a twitch in muscles of the face, arm, or leg, and the patient may hear a click and feel a pulling sensation on the skin under the coil. During the stimulation, electrical activity of muscles is recorded with a computer, using electrodes attached to the skin with tape.
- Neurologic evaluation. Before and after DBS, the patient's dystonia, including voice strength and difficulty swallowing, are measured with a standardized rating scale.
- DBS treatment. Patients are randomly assigned to have electrodes implanted in either the Gpi or STN area of the basal ganglia. The electrodes are what stimulate the brain in DBS therapy. Before surgery, a frame is secured to the patient's head, and an MRI scan is done. DBS involves making two small incisions and two small holes in the skull, opening the lining around the brain, locating the Gpi or STN, securing the electrodes in place and connecting them to the pulse generator that is placed under the skin below the collar bone. Additionally, during the surgery, the patient is asked to move certain muscles. The muscle activity is recorded to gain a better understanding of the physiology of movement. After surgery, computed tomography (CT) and MRI scans are done to confirm placement of the electrodes.
- Stimulation and evaluation. After surgery, patients' movements are evaluated during and after stimulation. The changes in movement and function are videotaped and scored according to a rating scale. The optimal stimulation settings are determined and the stimulators are adjusted accordingly.
- Follow-up. Patients are evaluated, with videotaping, at 1, 2, 3, 6, 12, 18 and 24 months after surgery, and the stimulators are adjusted as needed.
Read the detailed description
OBJECTIVE:
For dystonic patients who have failed medical therapy, stereotactic deep brain stimulation (DBS) of the globus pallidus interna (Gpi) or subthalamic nucleus (STN) can provide significant symptom improvement, but it is not known which site provides maximum benefit or the physiologic mechanisms by which these improvements may occur. The study goals are to determine if DBS of Gpi or STN provides significant clinical improvement in patients with primary generalized dystonia, whether the anatomic DBS site (if either) provides optimal improvement, and to gain deeper insight into the underlying pathophysiology of this disorder.
STUDY POPULATION:
Adult and pediatric patients with medically refractory primary generalized dystonia will make-up the study population. Patients with primary generalized dystonia suffer a wide range of symptoms including physical deformity, speech difficulties, swallowing dysfunction, visual problems, and intractable pain, which can result in an inability to care for themselves. Moreover, pediatric primary generalized dystonia patients will often have abnormal skeletal development (scoliosis) resulting from the abnormal forces placed on the skeleton by dystonic muscle contractions.
DESIGN:
We propose placing DBS electrodes (using microelectrode guidance) bilaterally into either the Gpi or STN of medically refractory dystonia patients. Physiological studies will be performed and compared with clinical changes to understand the mechanism of action of DBS and underlying pathophysiology of dystonia.
OUTCOME MEASURES:
Clinical effectiveness of DBS of the Gpi or STN will be measured by the Burke-Fahn-Marsden Scale (BFM) and monitored over time to determine the effectiveness of DBS and if there is an ideal anatomic stimulation site. Electrophysiologic outcome measures will be obtained to determine the underlying pathophysiology of dystonia, and the mechanism of action of DBS.
- Conditions
- Dystonia, Generalized Primary Dystonia
- Being tested
- Deep Brain Stimulation
- Keywords
- Surgery, Treatment, Physiology, Pediatric, Neurosurgery, Generalized Primary 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 StimulationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical effectiveness of DBS of the Gpi or STN will be measured by the Burke-Fahn-Marsden Scale (BFM) and monitored over time to determine the effectiveness DBS and if there is an ideal anatomic stimulation site.@@@@@@@@@
Secondary outcomes (1)
Determine the underlying pathophysiology of dystonia, and the mechanism of action of DBS.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2005 (estimated)
- Study startMar 2005
- Study completionJul 2006
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.
- 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
- Primary purpose
- Treatment
- Enrolment
- 40 participants
Sponsor and investigators
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00105430
- Sponsor's protocol ID
- 050111
- Other IDs
- 05-N-0111
- Status last verified
- Jul 2006
- First submitted
- 11 Mar 2005
- Last updated
- 6 Jul 2006
- Results posted
- No
Publications
Sanger TD, Garg RR, Chen R. Interactions between two different inhibitory systems in the human motor cortex. J Physiol. 2001 Jan 15;530(Pt 2):307-17. doi: 10.1111/j.1469-7793.2001.0307l.x.
PubMed 11208978
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Deep Brain Stimulation of the Globus Pallidus Interna or the Subthalamic Nucleus for Treatment of Generalized Primary Dystonia
- ClinicalTrials.gov ID
- NCT00105430
- Acronym / sponsor ID
- 050111
- Conditions
- Dystonia, Generalized Primary Dystonia
- Keywords
- Surgery, Treatment, Physiology, Pediatric, Neurosurgery, Generalized Primary Dystonia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- Mar 2005
- Study completion
- Jul 2006
- First posted
- 14 Mar 2005 (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.
This study will evaluate the effectiveness of deep brain stimulation (DBS) in treating primary generalized dystonia. Patients with dystonia have muscle spasms that cause uncontrolled twisting and repetitive movement or abnormal postures. Medical therapies are available, but not all patients get adequate relief from the abnormal movements or the pain associated with them. DBS is a surgical procedure that interrupts neuronal circuits in the Gpi and STN, areas of the basal ganglia of the brain that do not work correctly in patients with dystonia. The surgery results in decreased movement and therefore may lessen patients' symptoms and pain.
Patients 7 years of age and older with generalized dystonia that does not respond to medical treatment may be eligible for this study. Candidates are screened with blood and urine tests, chest x-ray, and an electrocardiogram in patients 35 years of age or more.
Participants undergo the following tests and procedures:
- Magnetic resonance imaging. MRI uses a magnetic field and radio waves to produce images of the brain. The patient lies on a table that is moved into the scanner (a narrow cylinder), wearing earplugs to muffle loud knocking and thumping sounds that occur during the scanning process. The procedure usually lasts about 45 to 90 minutes, during which the patient is asked to lie still for up to 15 minutes at a time.
- Transcranial magnetic stimulation. This procedure maps brain function. A wire coil is held on the scalp, and a brief electrical current is passed through the coil, creating a magnetic pulse that stimulates the brain. During the stimulation, the patient may be asked to tense certain muscles slightly or perform other simple actions. The stimulation may cause a twitch in muscles of the face, arm, or leg, and the patient may hear a click and feel a pulling sensation on the skin under the coil. During the stimulation, electrical activity of muscles is recorded with a computer, using electrodes attached to the skin with tape.
- Neurologic evaluation. Before and after DBS, the patient's dystonia, including voice strength and difficulty swallowing, are measured with a standardized rating scale.
- DBS treatment. Patients are randomly assigned to have electrodes implanted in either the Gpi or STN area of the basal ganglia. The electrodes are what stimulate the brain in DBS therapy. Before surgery, a frame is secured to the patient's head, and an MRI scan is done. DBS involves making two small incisions and two small holes in the skull, opening the lining around the brain, locating the Gpi or STN, securing the electrodes in place and connecting them to the pulse generator that is placed under the skin below the collar bone. Additionally, during the surgery, the patient is asked to move certain muscles. The muscle activity is recorded to gain a better understanding of the physiology of movement. After surgery, computed tomography (CT) and MRI scans are done to confirm placement of the electrodes.
- Stimulation and evaluation. After surgery, patients' movements are evaluated during and after stimulation. The changes in movement and function are videotaped and scored according to a rating scale. The optimal stimulation settings are determined and the stimulators are adjusted accordingly.
- Follow-up. Patients are evaluated, with videotaping, at 1, 2, 3, 6, 12, 18 and 24 months after surgery, and the stimulators are adjusted as needed.
Detailed description
OBJECTIVE:
For dystonic patients who have failed medical therapy, stereotactic deep brain stimulation (DBS) of the globus pallidus interna (Gpi) or subthalamic nucleus (STN) can provide significant symptom improvement, but it is not known which site provides maximum benefit or the physiologic mechanisms by which these improvements may occur. The study goals are to determine if DBS of Gpi or STN provides significant clinical improvement in patients with primary generalized dystonia, whether the anatomic DBS site (if either) provides optimal improvement, and to gain deeper insight into the underlying pathophysiology of this disorder.
STUDY POPULATION:
Adult and pediatric patients with medically refractory primary generalized dystonia will make-up the study population. Patients with primary generalized dystonia suffer a wide range of symptoms including physical deformity, speech difficulties, swallowing dysfunction, visual problems, and intractable pain, which can result in an inability to care for themselves. Moreover, pediatric primary generalized dystonia patients will often have abnormal skeletal development (scoliosis) resulting from the abnormal forces placed on the skeleton by dystonic muscle contractions.
DESIGN:
We propose placing DBS electrodes (using microelectrode guidance) bilaterally into either the Gpi or STN of medically refractory dystonia patients. Physiological studies will be performed and compared with clinical changes to understand the mechanism of action of DBS and underlying pathophysiology of dystonia.
OUTCOME MEASURES:
Clinical effectiveness of DBS of the Gpi or STN will be measured by the Burke-Fahn-Marsden Scale (BFM) and monitored over time to determine the effectiveness of DBS and if there is an ideal anatomic stimulation site. Electrophysiologic outcome measures will be obtained to determine the underlying pathophysiology of dystonia, and the mechanism of action of DBS.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 7 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Patients greater than 7 years of age with generalized primary dystonia.
- Patients who are refractory to medical treatment (including levodopa, anticholinergics, benzodiazepines, Baclofen) who are considered surgical candidates. Patients will be determined to have medical refractory dystonia after having been treated by at least two medications of two different groups at the maximum recommended dose for a duration of at least two months.
- In case of treatment by intrathecal Baclofen delivered by a pump, a change to oral Baclofen must be performed, so that the pump can be taken out.
- Patients must have a minimum BFM score of 20 on optimal medical therapy.
- Adults must be mentally competent to consent for entrance into the protocol at the time of admission.
Key exclusion criteria (11 listed)
- The patient has had previous lesioning surgery including radiofrequency lesioning of deep nuclei (thalamus, pallidum, STN).
- The patient currently has a functioning and effective stimulator in deep brain nuclei (thalamus, pallidum, STN).
- The patient is not able to tolerate surgery, as determined by the preoperative evaluation.
- The patient has a coagulopathy demonstrated by an abnormal prothrombin time, activated partial thromboplastin time, or thrombocytopenia (platelet count less than 150,000 platelets/mm(3)).
- The patient has an acute or untreated infection (viral, bacterial or fungal).
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 | Procedure or surgery | – |
Primary outcome
Clinical effectiveness of DBS of the Gpi or STN will be measured by the Burke-Fahn-Marsden Scale (BFM) and monitored over time to determine the effectiveness DBS and if there is an ideal anatomic stimulation site.@@@@@@@@@
1 secondary outcome
Determine the underlying pathophysiology of dystonia, and the mechanism of action of DBS.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2006.
- Background: PubMed 11208978Sanger TD, Garg RR, Chen R. Interactions between two different inhibitory systems in the human motor cortex. J Physiol. 2001 Jan 15;530(Pt 2):307-17. doi: 10.1111/j.1469-7793.2001.0307l.x.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deep Brain Stimulation in people with Dystonia and Generalized Primary Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
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 11 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 6 Jul 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 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.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Clinical effectiveness of DBS of the Gpi or STN will be measured by the Burke-Fahn-Marsden Scale (BFM) and monitored over time to determine the effectiveness DBS and if there is an ideal anatomic stimulation site.@@@@@@@@@. 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 NCT00105430, 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 6 Jul 2006.