Unilateral focused ultrasound pallidotomy for medication‑resistant limb dystonia
Focused Ultrasound Pallidotomy for Medication-Refractory Limb Dystonia (FUTURE Study)
This will test whether a non‑invasive focused ultrasound procedure can reduce arm or leg dystonia symptoms in adults who haven't improved with medicines or botulinum toxin.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 34 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta Academic / other |
| Locations | 4 sites (Mestre, (Venice) and 3 other locations) |
| Trial ID | NCT07168850 on ClinicalTrials.gov |
What this trial studies
The FUTURE trial is a prospective, randomized, double‑blind, sham‑controlled, multicenter study comparing unilateral MR‑guided focused ultrasound (MRgFUS) pallidotomy targeting the globus pallidus internus (GPi) to a sham procedure in adults with medication‑refractory limb dystonia. Thirty‑four participants aged 18–80 with idiopathic or genetic focal/segmental limb dystonia who failed oral drugs and botulinum toxin will be randomly assigned to active or sham treatment. The procedure uses MRI guidance and Exablate MRgFUS to create a millimeter‑scale lesion in the GPi while participants are awake and monitored; safety and symptom severity will be tracked over scheduled follow‑up visits. Key exclusions include secondary (acquired) dystonia, prior deep brain stimulation or other brain surgery, MRI contraindications, and low skull density ratio (SDR < 0.40).
Who should consider this trial
Good fit: Adults 18–80 with idiopathic or hereditary focal or segmental limb dystonia that has not improved with oral medications and botulinum toxin, who can tolerate MRI and have sufficient skull density (SDR ≥ 0.40), are ideal candidates.
Not a fit: People with acquired secondary dystonia, dementia or other neurodegenerative disease, prior DBS or brain surgery, contraindications to MRI, or low skull density (SDR < 0.40) are unlikely to benefit or are excluded.
Why it matters
Potential benefit: If successful, the procedure could reduce limb dystonia symptoms and offer a less invasive alternative to deep brain surgery for people who don't respond to medications or botulinum toxin.
How similar studies have performed: MRgFUS has established benefit for essential tremor and Parkinsonian tremor and small uncontrolled reports suggest pallidotomy may help dystonia, but randomized sham‑controlled data in limb dystonia are limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age 18-80 years * Able and willing to consent and attend visits * Idiopathic or hereditary limb dystonia (focal, segmental, or multifocal; isolated or combined) * Refractory to oral therapy and botulinum toxin * Stable medication for ≥30 days and ≥90 days since last BoNT * Capable of communicating during Exablate procedure Exclusion Criteria: * Acquired secondary dystonia * Dementia or other neurodegenerative disorders * Any clinically significant or unstable medical condition, which, in the opinion of the principal investigator or the clinician delegated by the principal investigator, may put the participant at risk when participating in the study (e.g., unstable heart disease or coagulopathy; uncontrolled psychiatric comorbidity) * Prior deep brain stimulation or other brain surgery * Any contraindication to MRI * Skull Density Ratio (SDR) \< 0.40 * Significant brain lesions (eg brain tumor, significant white matter lesions or globus pallidus interna lesions on baseline MRI) * Pregnancy
Where this trial is running
Mestre, (Venice) and 3 other locations
- Azienda ULSS3 Serenissima — Mestre, (Venice), Italy (Recruiting)
- IRCCS Centro Neurolesi "Bonino-Pulejo" — Messina, Italy (Recruiting)
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy (Recruiting)
- Azienda Ospedaliera Ospedali Riuniti Villa Sofia — Palermo, Italy (Recruiting)
Study contacts
- Study coordinator: Roberto Eleopra
- Email: roberto.eleopra@istituto-besta.it
- Phone: +390223942552
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.