Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation 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
First Affiliated Hospital of Fujian Medical University
24 participants
Apr 20, 2026
INTERVENTIONAL
Conditions
Summary
The purpose of this research study is to determine the optimal pulse repetition frequency of low-intensity focused ultrasound that is safe and effective in improving motor symptoms in patients with Huntington's disease.
Eligibility
Inclusion Criteria7
- Aged 18 to 75 years (inclusive)
- Genetically confirmed Huntington's disease with HTT CAG repeat length of 40 or greater
- 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
- Huntington's Disease Integrated Staging System stage 2 or 3
- Willing to participate and provide informed consent
- Have a reliable caregiver available
- No severe cognitive impairment that would preclude reliable reporting of adverse events or efficacy during treatment
Exclusion Criteria9
- History of self-injury, aggressive behavior, or unstable psychiatric disorders
- History of head injury or neurosurgical procedure
- Presence of intracranial implants, metallic foreign bodies, cochlear implants, or cardiac pacemakers
- Severe systemic disease, pregnancy, or breastfeeding
- Contraindications to magnetic resonance imaging, ultrasound, or anesthesia
- 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
- Severe brain atrophy with unclear external globus pallidus structure on MRI
- Concurrent participation in another interventional clinical trial
- Any other condition that, in the opinion of the investigator, would preclude participation
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Interventions
This is not a traditional three-arm parallel trial but a Stage I/II dose-finding study using an adaptive design. Participants are dynamically assigned to three pulse repetition frequency levels (10 Hz, 50 Hz, 100 Hz) based on predefined rules, rather than fixed randomization. The study adopts a two-stage utility-based Bayesian optimal interval (U-BOIN) design: Stage I: Dose decisions are based solely on dose-limiting toxicity (DLT) incidence. Participants are enrolled in cohorts of three. Based on observed DLTs, the next cohort's dose is determined (escalate/stay/de-escalate). Stage II: When any dose group reaches 6 participants or the highest dose is explored, safety and efficacy data are integrated to calculate a utility value. Subsequent cohorts are assigned to the dose group with the highest utility value. The study stops when any dose group reaches 12 participants or total enrollment reaches 24. Not all three dose levels may be utilized. Allocation is not fixed a priori.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT07513844