DystoniaPhase 1/2NCT02911103
Deep Brain Stimulation Surgery for Focal Hand Dystonia
A Phase 1/2 study testing DBS surgery and Medtronic Activa Rechargeable System in people with Dystonia and Focal Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
Researchers want to test a procedure called deep brain simulation (DBS) to treat focal hand dystonia (FHD). A device called a neurostimulator is placed in the chest. It is attached to wires placed in brain areas that affect movement. Stimulating these areas can help block nerve signals that cause abnormal movements. Read more about this study
- Who is it for?
- Ages 22 to 100 years
- Conditions the study is for
- Dystonia, Focal Dystonia, Musician's Dystonia
- What is being tested?
- DBS surgery and Medtronic Activa Rechargeable System
- Type of treatment
- Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Jan 2018to 2 Feb 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 22 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- FHD diagnosed by a neurologist
- BFM upper extremity subscore of 3 or more (score range 0 to 4) or ADDS difficulty of performing score equal to or more than 3.
- Patients must score 3 or below on the Tubiana and Chamagne scale or inability to perform at the concert level or patients must score a rating of "severe" on at least one item, impacting the patient s employment or essential activities on the Writer s Cramp Impairment scale.
- Symptoms causing significant impairment in quality of life and work or daily activities by patient self-assessment and SF12 scale, as reported by subjects. The subjects should be severely impaired in at least one of the following domains: handwriting, typing or playing an instrument. The impairment needs to have affected employment, manifested by substantive change in, or loss of, employment or career of choice.
- All patients must have failed or achieved inadequate benefit (continuing functional impairment) with at least two trials of botulinum toxin treatments.
- Women of childbearing age will have to agree to use contraception methods for the first 3 post operative months.
Who cannot join
- Subjects younger than 22 years old.
- MRI findings that would make participation and surgery unsafe (such as tumor or other space occupying lesion, stroke, hemorrhage or hematoma, edema, hydrocephalus, abscess) and MRI findings that would confound study outcomes (such as iron deposits in the basal ganglia, demyelinating disease, traumatic brain injury, structural or developmental abnormality, moderate or severe white matter hyperintensities).
- Subjects with prior brain surgery
- Pregnant or nursing women as safety of DBS has not been established in this group.
- Presence of a more widespread neurologic syndrome with the dystonia being part of it, and/or dystonia caused by an ongoing etiology, such as tardive dystonia associated with ongoing psychotropic treatment or an inherited neurodegenerative syndrome associated with intractable dystonia
- Intellectual disability as measured by the estimated General Ability Index (GAI) of the Wechsler Adult Intelligence Scale 4th Edition (WAIS-IV), which would render the participant not able to provide informed consent or to comply with the study procedures (estimated GAI less than 70)
Show all 20Show fewer
- Dementia as evidenced by formal neuropsychological evaluation and Mattis Dementia Rating Scale-2 (DRS-2) score below 128.
- Depressed subjects as determined by the neuropsychology or psychiatry screen, including Beck Depression Inventory (BDI) and psychiatric evaluation. Subjects scoring above 20 on the BDI or exhibiting moderate or severe active depression will be excluded
- Subjects with uncontrolled co-existing medical conditions: uncontrolled systemic hypertension with values above 170/100; active heart disease needing immediate intervention; active respiratory disease needing immediate intervention; uncorrected coagulation abnormalities; any condition that would render the patient unable to safely cooperate with the surgery and study tests as judged by the screening physician
- Presence of a contraindication to undergo a brain MRI
- Metal in the body which would make having an MRI scan unsafe, such as pacemakers, medication pumps, aneurysm clips, metallic prostheses (including metal pins and rods, heart valves or cochlear implants), shrapnel fragments, permanent eye liner or small metal fragments in the eye that welders and other metal workers may have;
- Uncomfortable in small closed spaces (have claustrophobia) such that the participant will be uncomfortable in the MRI machine;
- Unable to lay comfortably flat on their back for up to one hour in the MRI scanner;
- Subjects who require diathermy
- Subjects who require post-op MRIs with full body coil
- -- Subjects with an active systematic infection
- Subjects who are immune-compromised
- Subjects who are on anticoagulation therapy that would preclude their ability to undergo the implant procedure
- Subjects who are allergic or have shown hypersensitivity to any materials of the neurostimulation system which may come in contact with the body
- Subjects that cannot appoint a Durable power of attorney (DPA)
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Deep Brain Stimulation Surgery for Treatment of Focal Hand Dystonia
Researchers want to test a procedure called deep brain simulation (DBS) to treat focal hand dystonia (FHD). A device called a neurostimulator is placed in the chest. It is attached to wires placed in brain areas that affect movement. Stimulating these areas can help block nerve signals that cause abnormal movements.
Objectives:
Read more
To test DBS as treatment for FHD. To learn about brain and nerve cell function in people with dystonia.
Eligibility:
People ages 18 and older with severe FHD who have tried botulinum toxin treatment at least twice
Design:
Participation lasts 5 years.
Participants will be screened with:
Medical history
Physical exam
Videotape of their dystonia
Blood, urine, and heart tests
Brain MRI scan
Chest X-ray
Neuropsychological tests: answering questions, doing simple actions, and taking memory and thinking tests.
Hand movement tests
Participants will have surgery: A frame fixes their head to the operating table. A small hole is made in the skull. Wires are inserted to record brain activity and stimulate the brain while they do simple tasks. The wires are removed and the DBS electrode is inserted into the hole. The neurostimulator is placed under the skin of the chest, with wires running to the electrode in the brain. They will have CT and MRI scans during surgery.
Participants will recover in the hospital for about 1 week.
The neurostimulator will be turned on 1 4 weeks after discharge. Participants will have regular visits until the study ends. Visits include:
Checking symptoms and side effects
MRI
Movement, thinking, and memory tests
If the neurostimulator s battery runs out, participants will have surgery to replace it.
Read the detailed description
Objectives
To confirm the safety and possible trends toward efficacy of thalamic deep brain stimulation (DBS) targeting the ventralis oralis anterior/ventralis oralis posterior (VOA/VOP) nuclear complex in the treatment of subjects with focal hand dystonia (FHD); and to study the electrophysiologic properties of neurons in the VOA/VOP complex. DBS is FDA approved for Parkinson's disease, Essential tremor, Dystonia (humanitarian exemption), Obsessive- Compulsive disorder (humanitarian exemption) but not for focal hand dystonia.
Study population
Five adults with focal hand dystonia will be studied.
Design
This is a pilot study of 5 subjects with severe intractable FHD whose activities of daily living and quality of life are severely impaired. They will be recruited from our clinic and from referring practices. The subjects will undergo unilateral VOA/VOP DBS implantation, followed by programming to optimal parameters. The subjects will be followed at monthly intervals for 3 months, then every 3 months for 2 years, and then every 6 months until 5 years from the start. The safety of the procedure will be the primary outcome of the study. Trends toward efficacy will be assessed in several secondary outcomes, the most important ones being the change in dystonia severity and a quality of life scores. The electrophysiologic features of the motor ventral thalamic neurons will be recorded intraoperatively.
Outcome measures
Primary
-Number and severity of adverse events in the 5-year follow-up period.
Secondary
- Upper extremity sub-score of the Burke-Fahn-Marsden (BFM) scale and Arm Dystonia Disability Scale (ADDS) at baseline and every follow up visit
- For subjects with FHD type musician's dystonia: Tubiana and Chamagne scale at every follow up visit
- For subjects with FHD type Writer's cramp: Writer's Cramp Impairment Scale
- Improvement on patient reported outcomes evaluated by the SF-12 Health Status Survey at baseline and at every follow up visit
- Neuropsychological evaluation at baseline, 3 and 12 months post DBS surgery
- Dose of botulinum toxin injection required at baseline, 1 and 5 years.
Exploratory
- Intraoperatory electrophysiologic characteristics of the motor ventral thalamic neurons
- Local field potentials and their response to stimulation (in participants with implanted pulse generator capable of sensing capabilities)
- Conditions
- Dystonia, Focal Dystonia, Musician's Dystonia
- Being tested
- DBS surgery, Medtronic Activa Rechargeable System
- Keywords
- Electrophysiology, Neurosurgery, Dystonia, Deep Brain Stimulation
- Medical subject headings
- Dystonia, Dystonic Disorders, Dystonia, Focal, Task-Specific
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DBS surgeryProcedure or surgery
Unilateral thalamic DBS therapy
Medtronic Activa Rechargeable SystemDevice
Used for diagnostic purposes.
| Study group | Role | What they receive |
|---|---|---|
| Active | Experimental | single arm study Receives: Procedure: DBS surgery; Device: Medtronic Activa Rechargeable System |
What is being measured
The primary outcome is the main question the study is designed to answer.
To confirm the safety of VOA/VOP thalamic DBS in FHD
Number and severity of adverse events in the 5-year follow-up period.
Measured: 5 years
Secondary outcomes (6)
Neuropsychological Evaluation
To perform a neuropsychological evaluation at baseline, 3 and 12 months post DBS surgery of the VOA/VOP complex in subjects with FHD
Measured: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in clinical improvement of severe intractable FHD
Burke-Fahn-Marsden (BFM) scale and Arm Dystonia Disability Scale (ADDS) at baseline and every follow up visit
Measured: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improved patient reported outcomes
Neuropsychological evaluation at baseline, 3 and 12 months post DBS surgery
Measured: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improvement of severe intractable FHD in subjects with musician s dystonia
For subjects with FHD type Musician s Dystonia: Tubiana and Chamagne scale at every follow up visit
Measured: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improvement of severe intractable FHD in subjects with Writer s cramp
For subjects with FHD type writer s cramp: Writer s Cramp Impairment Scale
Measured: 5 years
To evaluate the botulinum toxin dose required for the treatment of FHD at baseline and 1 and 5 years after DBS therapy of the VOA/VOP complex
Dose of Botulinum Toxin at 1 and 5 years
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2016 (estimated)
- Study start17 Jan 2018
- Primary completion2 Feb 2029 (estimated)
- Study completion2 Feb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DystoniaStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Debra J Ehrlich, M.D., Principal investigator, National Institute of Neurological Disorders and Stroke (NINDS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02911103
- Sponsor's protocol ID
- 160166
- Other IDs
- 16-N-0166
- Status last verified
- 24 Sep 2026
- First submitted
- 21 Sep 2016
- Last updated
- 5 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided: It has not been determined whether sharing IPD will be feasible for this study.
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Deep Brain Stimulation Surgery for Treatment of Focal Hand Dystonia
- ClinicalTrials.gov ID
- NCT02911103
- Acronym / sponsor ID
- 160166
- Conditions
- Dystonia, Focal Dystonia, Musician's Dystonia
- Keywords
- Electrophysiology, Neurosurgery, Dystonia, Deep Brain Stimulation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 5 participants (actual)
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- 17 Jan 2018 (actual)
- Primary completion
- 2 Feb 2029 (estimated)
- Study completion
- 2 Feb 2029 (estimated)
- First posted
- 22 Sep 2016 (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.
Researchers want to test a procedure called deep brain simulation (DBS) to treat focal hand dystonia (FHD). A device called a neurostimulator is placed in the chest. It is attached to wires placed in brain areas that affect movement. Stimulating these areas can help block nerve signals that cause abnormal movements.
Objectives:
To test DBS as treatment for FHD. To learn about brain and nerve cell function in people with dystonia.
Eligibility:
People ages 18 and older with severe FHD who have tried botulinum toxin treatment at least twice
Design:
Participation lasts 5 years.
Participants will be screened with:
Medical history
Physical exam
Videotape of their dystonia
Blood, urine, and heart tests
Brain MRI scan
Chest X-ray
Neuropsychological tests: answering questions, doing simple actions, and taking memory and thinking tests.
Hand movement tests
Participants will have surgery: A frame fixes their head to the operating table. A small hole is made in the skull. Wires are inserted to record brain activity and stimulate the brain while they do simple tasks. The wires are removed and the DBS electrode is inserted into the hole. The neurostimulator is placed under the skin of the chest, with wires running to the electrode in the brain. They will have CT and MRI scans during surgery.
Participants will recover in the hospital for about 1 week.
The neurostimulator will be turned on 1 4 weeks after discharge. Participants will have regular visits until the study ends. Visits include:
Checking symptoms and side effects
MRI
Movement, thinking, and memory tests
If the neurostimulator s battery runs out, participants will have surgery to replace it.
Detailed description
Objectives
To confirm the safety and possible trends toward efficacy of thalamic deep brain stimulation (DBS) targeting the ventralis oralis anterior/ventralis oralis posterior (VOA/VOP) nuclear complex in the treatment of subjects with focal hand dystonia (FHD); and to study the electrophysiologic properties of neurons in the VOA/VOP complex. DBS is FDA approved for Parkinson's disease, Essential tremor, Dystonia (humanitarian exemption), Obsessive- Compulsive disorder (humanitarian exemption) but not for focal hand dystonia.
Study population
Five adults with focal hand dystonia will be studied.
Design
This is a pilot study of 5 subjects with severe intractable FHD whose activities of daily living and quality of life are severely impaired. They will be recruited from our clinic and from referring practices. The subjects will undergo unilateral VOA/VOP DBS implantation, followed by programming to optimal parameters. The subjects will be followed at monthly intervals for 3 months, then every 3 months for 2 years, and then every 6 months until 5 years from the start. The safety of the procedure will be the primary outcome of the study. Trends toward efficacy will be assessed in several secondary outcomes, the most important ones being the change in dystonia severity and a quality of life scores. The electrophysiologic features of the motor ventral thalamic neurons will be recorded intraoperatively.
Outcome measures
Primary
-Number and severity of adverse events in the 5-year follow-up period.
Secondary
- Upper extremity sub-score of the Burke-Fahn-Marsden (BFM) scale and Arm Dystonia Disability Scale (ADDS) at baseline and every follow up visit
- For subjects with FHD type musician's dystonia: Tubiana and Chamagne scale at every follow up visit
- For subjects with FHD type Writer's cramp: Writer's Cramp Impairment Scale
- Improvement on patient reported outcomes evaluated by the SF-12 Health Status Survey at baseline and at every follow up visit
- Neuropsychological evaluation at baseline, 3 and 12 months post DBS surgery
- Dose of botulinum toxin injection required at baseline, 1 and 5 years.
Exploratory
- Intraoperatory electrophysiologic characteristics of the motor ventral thalamic neurons
- Local field potentials and their response to stimulation (in participants with implanted pulse generator capable of sensing capabilities)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Active | Experimental | Procedure: DBS surgery, Device: Medtronic Activa Rechargeable Systemsingle arm study |
Who may qualify
- Ages
- 22 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- FHD diagnosed by a neurologist
- BFM upper extremity subscore of 3 or more (score range 0 to 4) or ADDS difficulty of performing score equal to or more than 3.
- Patients must score 3 or below on the Tubiana and Chamagne scale or inability to perform at the concert level or patients must score a rating of "severe" on at least one item, impacting the patient s employment or essential activities on the Writer s Cramp Impairment scale.
- Symptoms causing significant impairment in quality of life and work or daily activities by patient self-assessment and SF12 scale, as reported by subjects. The subjects should be severely impaired in at least one of the following domains: handwriting, typing or playing an instrument. The impairment needs to have affected employment, manifested by substantive change in, or loss of, employment or career of choice.
- All patients must have failed or achieved inadequate benefit (continuing functional impairment) with at least two trials of botulinum toxin treatments.
Key exclusion criteria (20 listed)
- Subjects younger than 22 years old.
- MRI findings that would make participation and surgery unsafe (such as tumor or other space occupying lesion, stroke, hemorrhage or hematoma, edema, hydrocephalus, abscess) and MRI findings that would confound study outcomes (such as iron deposits in the basal ganglia, demyelinating disease, traumatic brain injury, structural or developmental abnormality, moderate or severe white matter hyperintensities).
- Subjects with prior brain surgery
- Pregnant or nursing women as safety of DBS has not been established in this group.
- Presence of a more widespread neurologic syndrome with the dystonia being part of it, and/or dystonia caused by an ongoing etiology, such as tardive dystonia associated with ongoing psychotropic treatment or an inherited neurodegenerative syndrome associated with intractable dystonia
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 |
|---|---|---|
| DBS surgeryUnilateral thalamic DBS therapy | Procedure or surgery | Active |
| Medtronic Activa Rechargeable SystemUsed for diagnostic purposes. | Device | Active |
Primary outcome
To confirm the safety of VOA/VOP thalamic DBS in FHD
Time frame: 5 years
Number and severity of adverse events in the 5-year follow-up period.
6 secondary outcomes
Neuropsychological Evaluation
Time frame: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in clinical improvement of severe intractable FHD
Time frame: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improved patient reported outcomes
Time frame: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improvement of severe intractable FHD in subjects with musician s dystonia
Time frame: 5 years
To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improvement of severe intractable FHD in subjects with Writer s cramp
Time frame: 5 years
To evaluate the botulinum toxin dose required for the treatment of FHD at baseline and 1 and 5 years after DBS therapy of the VOA/VOP complex
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. It has not been determined whether sharing IPD will be feasible for this study.
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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.
Debra J Ehrlich, M.D. principal investigator
National Institute of Neurological Disorders and Stroke (NINDS)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing DBS surgery and Medtronic Activa Rechargeable System in people with Dystonia and Focal 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 6 inclusion and 20 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: under way, not looking for participants, as of the sponsor's last update on 5 Nov 2026. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
This study is still under way. Results are not available in the source record yet.
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?
To confirm the safety of VOA/VOP thalamic DBS in FHD (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Nov 2026.