NCT02911103

Deep Brain Stimulation Surgery for Focal Hand Dystonia

Deep Brain Stimulation Surgery for Treatment of Focal Hand Dystonia

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) · NIH
• Sex: All
• Age: 22 Years – 100 Years
• Healthy volunteers: Not accepted

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. 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)

Conditions

• Dystonia
• Focal Dystonia
• Musician's Dystonia

Interventions

Type	Name	Description
PROCEDURE	DBS surgery	Unilateral thalamic DBS therapy
DEVICE	Medtronic Activa Rechargeable System	Used for diagnostic purposes.

Timeline

• Start date: 2018-01-17
• Primary completion: 2029-02-02
• Completion: 2029-02-02
• First posted: 2016-09-22
• Last updated: 2026-03-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study