Low-intensity focused ultrasound targeting the globus pallidus to improve movement in Huntington's disease
Low-Intensity Focused Ultrasound Stimulation of the External Globus Pallidus in Huntington's Disease: A Phase I/II Safety and Feasibility Trial
This trial tests whether different pulse frequencies of low-intensity focused ultrasound (LIFU) aimed at a deep brain target can safely reduce chorea and other motor symptoms in adults with Huntington's disease.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | First Affiliated Hospital of Fujian Medical University Academic / other |
| Locations | 1 site (Fuzhou, Fujian) |
| Trial ID | NCT07513844 on ClinicalTrials.gov |
What this trial studies
This phase I/II adaptive dose-finding study delivers LIFU to the external globus pallidus using MRI-derived individualized head models and real-time neuronavigation. Three pulse repetition frequencies (10 Hz, 50 Hz, 100 Hz) are evaluated using a Bayesian optimal interval design with sequential cohorts of three and utility-driven dose escalation that integrates safety and early efficacy data. Each participant receives ten LIFU sessions over five consecutive days (two sessions per day) while motor function, cognitive measures, and adverse events are monitored. The primary goal is to identify the pulse frequency that best balances safety with improvement in motor symptoms.
Who should consider this trial
Good fit: Adults 18–75 years with genetically confirmed Huntington's disease (HTT CAG ≥40), in stage 2 or 3 with a Total Maximal Chorea score of 8 or higher, able to consent and with a reliable caregiver are the intended participants.
Not a fit: Patients with severe cognitive impairment, unstable psychiatric disorders, prior neurosurgery or intracranial implants, contraindications to MRI/ultrasound/anesthesia, pregnancy, or breastfeeding are unlikely to be eligible or to benefit from this protocol.
Why it matters
Potential benefit: If successful, this could offer a noninvasive way to reduce chorea and improve motor control for people with Huntington's disease.
How similar studies have performed: Early clinical and preclinical LIFU work demonstrates promising safety and the ability to modulate deep brain circuits, but targeted pallidal LIFU for Huntington's disease is a novel application that has not yet been proven.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Aged 18 to 75 years (inclusive) 2. Genetically confirmed Huntington's disease with HTT CAG repeat length of 40 or greater 3. Unified Huntington's Disease Rating Scale Total Maximal Chorea score of 8 or higher on a scale of 0 to 28, with higher scores indicating more severe chorea 4. Huntington's Disease Integrated Staging System stage 2 or 3 5. Willing to participate and provide informed consent 6. Have a reliable caregiver available 7. No severe cognitive impairment that would preclude reliable reporting of adverse events or efficacy during treatment Exclusion Criteria: 1. History of self-injury, aggressive behavior, or unstable psychiatric disorders 2. History of head injury or neurosurgical procedure 3. Presence of intracranial implants, metallic foreign bodies, cochlear implants, or cardiac pacemakers 4. Severe systemic disease, pregnancy, or breastfeeding 5. Contraindications to magnetic resonance imaging, ultrasound, or anesthesia 6. Prior treatment with electroconvulsive therapy, transcranial magnetic stimulation, transcranial direct current stimulation, transcranial alternating current stimulation, or other neuromodulation therapies within the past 3 years 7. Severe brain atrophy with unclear external globus pallidus structure on MRI 8. Concurrent participation in another interventional clinical trial 9. Any other condition that, in the opinion of the investigator, would preclude participation
Where this trial is running
Fuzhou, Fujian
- Department of Rehabilitation Medicine, The First Affiliated Hospital of Fujian Medical University — Fuzhou, Fujian, China (Recruiting)
Study contacts
- Study coordinator: Xin-Yuan Chen
- Email: fychenxinyuan@fjmu.edu.cn
- Phone: +86 15005065282
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.