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Focused Ultrasound Unilateral Pallidotomy for Medication-Refractory Limb Dystonia

ConditionsLimb DystoniaIdiopathic DystoniaMedication-refractory DystoniaDystonia, Focal
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-80
SponsorFondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

About this trial

The goal of this clinical trial is to find out if a non-invasive procedure called focused ultrasound can safely and effectively treat limb dystonia in adults who have not improved with medications or botulinum toxin injections.

The main questions it aims to answer are:

* Does focused ultrasound reduce the severity of limb dystonia symptoms? * Is the procedure safe and well tolerated?

Researchers will compare people who receive focused ultrasound treatment to those who receive a sham procedure (a look-alike procedure without active treatment) to see if the treatment works.

Focused ultrasound works by producing a very small (millimeter-size) lesion in a deep brain region. In this study, the lesion is located in a region called globus pallidus, which helps control movement.

Participants will:

* Be randomly assigned to one of two groups: focused ultrasound or sham * Undergo a procedure using MRI and focused ultrasound to target a part of the brain called the globus pallidus * Be followed for 6 months after treatment to assess changes in symptoms, quality of life, and brain function * Participants in the sham group will be offered the real treatment after 3 months

This study may offer a new non-surgical treatment option for people with dystonia affecting the arm or leg, whose pharmacological control is unsatisfactory.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Exablate MR-guided Focused Ultrasound Pallidotomy

    Device

    Active Pallidotomy

  • Sham Sham Exablate MRgFUS Pallidotomy

    Device

    Sham procedure. Identical setup without thermal lesioning. Participants cross over to Active Pallidotomy after 3 months.

Treatment groups

34 Participants
are divided into 2 treatment groups
Group A: Active Pallidotomy (MRgFUS)Experimental treatment 1 intervention
Group B: Sham ProcedureSham comparator 2 interventions

Trial outcomes

Primary outcomes

1

Chenge in the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS)

The BFMDRS has a maximum score of 120 for the movement subscale and 30 for the disability subscale. Higher scores indicating more severe dystonia.

Time frame
Baseline to Week 12

Secondary outcomes

1

Incidence of treatment-related adverse events

Assessment of any treatment-related adverse events throughout the whole study period

Time frame
Baseline to Week 24
2

Changes in the Quick Disabilities of Arm, Shoulder & Hand (Quick-DASH) score

The QuickDASH is a shortened, 11-item version of the DASH Outcome Measure, designed to assess physical function and symptom severity related to the arm, shoulder, and hand. Maximum score 100; the greater the score the grear the disability.

Time frame
Baseline, Week 12 and Week 24
3

Changes in the Clinical Global Impression of Change (CGIC) score

The CGIC is a 7-point scale used to assess the degree of change in a patient's condition since the start of treatment or another defined baseline. Lower scores indicates improvement and higher scores indicating worsening of the patient's condition.

Time frame
Baseline, week 12 and week 24
4

Patient Global Impression of Change (PGIC)

The maximum score of the PGIC is 7: the higher the score the higher the improvement reported by a patient.

Time frame
Baseline, Week 12 and week 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Lead sponsor

Azienda ULSS 3 Serenissima

Collaborator

IRCCS Centro Neurolesi Bonino Pulejo

Collaborator

Azienda Ospedaliera Villa Sofia

Collaborator

Source ClinicalTrials.gov
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