Dystonia causes involuntary muscle contractions that twist the body into abnormal postures or repetitive movements. It can affect one area like the neck or hand, or spread to multiple body parts. Current treatments include botulinum toxin injections, oral medications that rarely provide complete relief, and deep brain stimulation for severe cases.
What's actually going on in research
Trials are testing new gene therapies for inherited forms, medications that target different neurotransmitter systems, and refined deep brain stimulation approaches. Researchers are also studying the brain circuits involved in dystonia to identify better treatment targets, and testing drugs already approved for other movement disorders to see if they help with dystonia.
Gene therapy for DYT1
A gene therapy delivered directly to the brain aims to correct the genetic mutation causing DYT1 dystonia, the most common inherited form. Early trials are testing safety and whether it can reduce symptoms in young people with this form.
Targeted medications
Several trials are testing drugs that work on specific neurotransmitter systems beyond the ones current medications target. These include drugs affecting adenosine receptors and phosphodiesterase enzymes that may calm abnormal muscle signals.
Deep brain stimulation refinement
Researchers are testing personalized stimulation patterns and new brain targets to improve outcomes. Studies are also examining which dystonia types respond best to specific stimulation settings.
What to know before you search
Eligibility typically depends on dystonia type and location, genetic testing results for inherited forms, previous treatments tried, and symptom severity.
What types of trials are currently open
- Gene therapy trials — Testing one-time treatments that deliver corrected genes to brain cells, currently focused on inherited forms like DYT1 dystonia.
- Medication trials — Testing new oral drugs or repurposed medications to reduce muscle contractions, often comparing them to placebo or current treatments.
- Device trials — Studies of deep brain stimulation programming, new electrode designs, or other neuromodulation approaches.
- Botulinum toxin trials — Testing new formulations, injection techniques, or combinations with other treatments to improve how long relief lasts.
- Natural history studies — Following people with dystonia over time to understand how different forms progress and what factors predict treatment response.
Recently added Dystonia trials
Receive focused ultrasound stimulation to treat cervical dystonia
This study is a single-blinded, randomized control study that aims to recruit 48 participants. Participants will be "randomized" into one of the study arms of the study: active stimulation to GPi, active stimulation to DN, or sham stimulation. The purpose of the study is to look into the effects of LIFUS in patients with cervical dystonia. The participation will include seven study visits, at least 24 hours apart. All study visits will take place at TWH. The first visit is a screening visit. Visits 2-6 will be on consecutive days. Visit 7 will be a week from Visit 6 or from your last day of LIFUS, whichever comes first.
Noninvasive Brain Stimulation for Cervical Dystonia
The primary aim of this study is to assess whether continuous theta-burst stimulation (cTBS) can be helpful in reducing symptoms of idiopathic cervical dystonia. This will be a parallel-group, randomised, sham-controlled, double blind clinical trial. Patients and investigators measuring patient symptoms clinical scales will be blinded from allocation group, investigators delivering the intervention will not be blinded, as this is not feasible. Real cTBS will be compared to sham cTBS, with patients allocated to these two groups with a 1:1 recruitment ratio. Bilateral cTBS will be delivered four times daily, over eight days, for a total of 32 bilateral cTBS sessions. The primary outcome is the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS). This will be administered at T1 (pre-cTBS), T2 (day 10, following days 2-9 where patients received the intervention), T3 (2 weeks post the final session of cTBS), and T4 (4-5 weeks post the final session of cTBS). Secondary outcomes will be the TWSTRS subscales (severity, disability, pain), Quality of Life scale, Global dystonia severity rating scale (GDS), Fahn Tolosa Marin (FTM) modified (to include only questions relevant to head/neck tremor) tremor scale, kinematic data of patient's movement patterns, motor evoked potentials (MEPs), and resting-state fMRI data (fMRI).
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