DBS Surgery for Hand Dystonia

Overseen By Debra J Ehrlich, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)

Must not be taking: Anticoagulants, Psychotropics

Disqualifiers: Pregnancy, Prior brain surgery, Dementia, Depression, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a procedure called deep brain stimulation (DBS) to assist individuals with severe focal hand dystonia, a condition causing involuntary hand movements. The treatment places a device in the chest connected to wires in the brain to block the nerve signals causing these movements. Researchers aim to gain insights into brain and nerve cell function in those with dystonia. Individuals with severe hand dystonia who have tried botulinum toxin treatment at least twice may be suitable candidates for this trial. As a Phase 1 and Phase 2 trial, the study focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are on anticoagulation therapy (blood thinners), you may not be able to participate in the trial.

What prior data suggests that this procedure is safe for treating focal hand dystonia?

Research has shown that deep brain stimulation (DBS) is generally safe for treating dystonia, a movement disorder. Studies have found that DBS can improve symptoms and enhance the quality of life for people with various types of dystonia. These studies also report that DBS is well-tolerated and has maintained a good safety record over many years.

Although some negative side effects can occur, they are usually mild or manageable. These may include temporary issues at the surgery site or the need to adjust the stimulation settings. Overall, DBS is considered a safe option for those dealing with movement disorders like dystonia. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Deep Brain Stimulation (DBS) surgery is unique because it offers a targeted approach to treating hand dystonia, a condition often managed with medications like botulinum toxin injections or oral drugs that can have systemic effects. Unlike these treatments, DBS involves implanting electrodes in specific areas of the brain to modulate abnormal nerve signals, which could provide more precise symptom control with potentially fewer side effects. Researchers are excited about DBS because it has shown promise in other movement disorders, like Parkinson's disease, suggesting it might offer significant relief for hand dystonia patients who have not responded well to conventional therapies.

What evidence suggests that DBS surgery is effective for focal hand dystonia?

Research has shown that deep brain stimulation (DBS) effectively treats various types of dystonia, including focal hand dystonia (FHD). One study found that DBS improved movement and quality of life for people with dystonia. Another analysis confirmed that DBS is safe and significantly reduces symptoms. This procedure involves placing a small device in the chest, connected to wires in the brain, to help block abnormal nerve signals. Early findings suggest that DBS can lessen the severity of involuntary movements in people with FHD. Participants in this trial will undergo DBS surgery to evaluate its effectiveness specifically for hand dystonia. ^{1 2 6 7 8}

Who Is on the Research Team?

Debra J Ehrlich, M.D.

Principal Investigator

National Institute of Neurological Disorders and Stroke (NINDS)

Are You a Good Fit for This Trial?

Adults over 18 with severe focal hand dystonia (FHD) who haven't improved after two botulinum toxin treatments. They must not have widespread neurological issues, prior brain surgery, or be pregnant/nursing. Participants need to agree to use contraception and should not have metal in their body that's unsafe for MRI scans.

Inclusion Criteria

My condition severely affects my daily activities and work, especially in tasks like writing or playing an instrument.

I have tried botulinum toxin treatments at least twice without enough improvement.

FHD diagnosed by a neurologist

Exclusion Criteria

I currently have an infection that affects my whole body.

I am on blood thinners that prevent me from having surgery.

My MRI results do not show any issues that would make surgery or the study unsafe for me.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Surgery and Initial Recovery

Participants undergo DBS surgery and recover in the hospital for about 1 week

1 week

Inpatient stay

Post-Surgery Adjustment

The neurostimulator is turned on 1-4 weeks after discharge, and participants have regular visits for symptom and side effect checks, MRI, and movement, thinking, and memory tests

4 weeks

Regular visits

Follow-up

Participants are monitored for safety and effectiveness with regular follow-up visits every month for 3 months, then every 3 months for 2 years, and every 6 months until 5 years

5 years

Monthly, then quarterly, then biannual visits

What Are the Treatments Tested in This Trial?

Interventions

• DBS surgery

Trial Overview The trial is testing deep brain stimulation (DBS) surgery for FHD treatment. A neurostimulator device will be implanted in the chest and connected to electrodes in the brain, aiming to block signals causing abnormal movements. The study spans five years with regular follow-ups.

How Is the Trial Designed?

Group I: Active Experimental Treatment 2 Interventions

single arm study

Who Is Running the Clinical Trial?

National Institute of Neurological Disorders and Stroke (NINDS)

Lead Sponsor

Trials

1,403

Recruited

655,000+

Published Research Related to This Trial

Deep brain stimulation (DBS) for Parkinson's disease is generally safe, with a low mortality rate of 0.2% and permanent morbidity at 0.6%, but complications such as intracerebral hemorrhage (ICH) and seizures can occur in a small percentage of cases.

The study found that the type of lead used in DBS can influence complications, with Medtronic's leads associated with higher rates of perielectrode brain edema compared to Boston Scientific's, highlighting the need for further research on hardware design to improve safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Complications of deep brain stimulation in Parkinson's disease: a single-center experience of 517 consecutive cases. Servello, D., Galbiati, TF., Iess, G., et al.[2023]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8395937/

Surgical Management for Dystonia: Efficacy of Deep Brain ... This study illustrates DBS is an established, effective treatment option for patients with different dystonias, such as generalized, cervical, and various ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1353802023000408

Deep brain stimulation for hemidystonia: A meta-analysis ... This meta-analysis aims to summarize the published reports on DBS for hemidystonia of different etiologies, to compare different stimulation targets, and to ...

3. frontiersin.org frontiersin.org/journals/human-neuroscience/articles/10.3389/fnhum.2021.757579/full

Deep Brain Stimulation Treating Dystonia: A Systematic ... GPi-DBS and STN-DBS were both safe and resulted in excellent improvement in efficacy and quality of life in patients with dystonia.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11987142/

Efficacy of deep brain stimulation in treating monogenic ... This protocol presents the methodology of a planned systematic review to assess the efficacy of DBS as a treatment for monogenic dystonia symptoms.

5. sciencedirect.com sciencedirect.com/science/article/pii/S2949669124000150

Directional deep brain stimulation for cervical dystonia Directional DBS significantly improved dystonia severity, pain, disability, and quality of life (p < 0.001), with no significant differences versus conventional ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8663760/

Deep Brain Stimulation Treating Dystonia - PubMed Central Conclusion: GPi-DBS and STN-DBS were both safe and resulted in excellent improvement in efficacy and quality of life in patients with dystonia. Compared with ...

7. movementdisorders.onlinelibrary.wiley.com movementdisorders.onlinelibrary.wiley.com/doi/10.1002/mdc3.70274

One Side or Two? A Systematic Review of Deep Brain ... The results showed that bilateral STN DBS improved the mean total Unified Parkinson's Disease Rating Scale (UPDRS) motor score by 54%, whereas ...

8. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1353802022000347

A randomised double-blind controlled study of Deep Brain ... Our data support a long-term effect of both STN-DBS and GPi-DBS in dystonia with equal effect and safety for up to 15 years.