Huntington DiseaseNCT07513844
Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in Huntington's Disease
A study testing Low-Intensity Focused Ultrasound Stimulation（LIFU） in people with Huntington Disease, run by First Affiliated Hospital of Fujian Medical University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- Low-Intensity Focused Ultrasound Stimulation（LIFU）
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteChina
- Study dates
- 20 Apr 2026to 31 Mar 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Department of Rehabilitation Medicine, The First Affiliated Hospital of Fujian Medical University
Fuzhou, Fujian
Xin-Yuan Chen, +86 15005065282, fychenxinyuan@fjmu.edu.cn
About this study
Official title: Low-Intensity Focused Ultrasound Stimulation of the External Globus Pallidus in Huntington's Disease: A Phase I/II Safety and Feasibility Trial
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.
Read the detailed description
Huntington's disease is an autosomal dominant neurodegenerative disorder caused by HTT gene CAG repeat expansion. Early degeneration of striatal neurons projecting to the external globus pallidus (GPe) leads to abnormal basal ganglia circuitry and impaired motor control, resulting in involuntary movements and other motor symptoms. Current treatments are symptomatic only, with no disease-modifying therapies available. Deep brain stimulation targeting the globus pallidus has shown efficacy but is invasive and associated with significant adverse events.
Low-intensity focused ultrasound (LIFU) enables non-invasive, deep, and millimeter-precise neuromodulation. It has been applied in various neurological and psychiatric disorders with favorable safety and efficacy, demonstrating potential for neuromodulation of basal ganglia circuits.
This phase I/II adaptive dose-finding prospective interventional study evaluates the safety and efficacy of LIFU targeting the external globus pallidus in patients with Huntington's disease. Using MRI-derived individualized head models and real-time neuronavigation, the study employs a Bayesian optimal interval (BOIN) design with three pulse repetition frequencies: 10 Hz, 50 Hz, and 100 Hz. Patients are enrolled in sequential cohorts of three, with dose escalation guided by a utility-based approach integrating safety and efficacy data. Each patient receives ten LIFU sessions over five consecutive days, with two sessions daily. Motor function, cognitive function, functional assessments, and magnetic resonance imaging are evaluated before the first treatment session and after the final treatment session.
- Conditions
- Huntington Disease
- Being tested
- Low-Intensity Focused Ultrasound Stimulation（LIFU）
- Keywords
- low intensity focused ultrasound, Huntington Disease, brain stimulation, ultrasonic stimulation
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low-Intensity Focused Ultrasound Stimulation（LIFU）Device
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.
| Study group | Role | What they receive |
|---|---|---|
| 10 Hz LIFU Group | Experimental | Low-Intensity Focused Ultrasound (LIFU), PRF 10 Hz Receives: Device: Low-Intensity Focused Ultrasound Stimulation（LIFU） |
| 50 Hz LIFU Group | Experimental | Low-Intensity Focused Ultrasound (LIFU), PRF 50 Hz Receives: Device: Low-Intensity Focused Ultrasound Stimulation（LIFU） |
| 100 Hz LIFU Group | Experimental | Low-Intensity Focused Ultrasound (LIFU), PRF 100 Hz Receives: Device: Low-Intensity Focused Ultrasound Stimulation（LIFU） |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of Dose-Limiting Toxicity (DLT)
DLT refers to the occurrence of any of the following six events during LIFU treatment:
- Second-degree scalp burn;
- Clinical seizure;
- Worsening of chorea or new movement disorder;
- Significant deterioration of the patient's mood or mental status (e.g., anxiety, depression, hallucinations, excessive sleepiness);
- Severe autonomic dysfunction, such as marked blood pressure fluctuations, abnormal heart rate, or respiratory distress;
- Imaging-related safety events: new or significantly enlarged brain edema, new microbleeds, new infarcts, or structural abnormalities in the treatment area on post-treatment cranial MRI compared with baseline, deemed clinically significant by both radiologists and clinicians.
Measured: At any point during or immediately following intervention on day of LIFUS application
Efficacy: Change in Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS)
The Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS) evaluates motor impairment in Huntington's disease.
Score range: 0-124 Higher scores indicate more severe motor impairment
Response is defined as a reduction of ≥4 points from baseline.
Measured: Baseline and within 2 days after completing the 5-day LIFU treatment
Comprehensive Benefit-Risk: Utility
Utility is a composite measure integrating dose-limiting toxicity (DLT) and efficacy response to quantify the overall benefit-risk balance for each dose group.
During the trial, the Utility value for each dose group is dynamically calculated using the U-BOIN design platform. In Stage II, the Utility value determines dose allocation for subsequent cohorts. At study completion, the dose group with the highest Utility value is identified as the optimal biological dose.
Measured: Within 2 days after completing the 5-day LIFU treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2026
- Study start20 Apr 2026
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Fujian Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xinyuan Chen, First Affiliated Hospital of Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07513844
- Sponsor's protocol ID
- MRCTA,ECFAH of FMU[2025]738-1
- Status last verified
- Mar 2026
- First submitted
- 24 Mar 2026
- Last updated
- 29 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Low-Intensity Focused Ultrasound Stimulation of the External Globus Pallidus in Huntington's Disease: A Phase I/II Safety and Feasibility Trial
- ClinicalTrials.gov ID
- NCT07513844
- Acronym / sponsor ID
- MRCTA,ECFAH of FMU[2025]738-1
- Conditions
- Huntington Disease
- Keywords
- low intensity focused ultrasound, Huntington Disease, brain stimulation, ultrasonic stimulation
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Fujian Medical UniversityUniversity, hospital or other
- Study start
- 20 Apr 2026 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Mar 2027 (estimated)
- First posted
- 7 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Huntington's disease is an autosomal dominant neurodegenerative disorder caused by HTT gene CAG repeat expansion. Early degeneration of striatal neurons projecting to the external globus pallidus (GPe) leads to abnormal basal ganglia circuitry and impaired motor control, resulting in involuntary movements and other motor symptoms. Current treatments are symptomatic only, with no disease-modifying therapies available. Deep brain stimulation targeting the globus pallidus has shown efficacy but is invasive and associated with significant adverse events.
Low-intensity focused ultrasound (LIFU) enables non-invasive, deep, and millimeter-precise neuromodulation. It has been applied in various neurological and psychiatric disorders with favorable safety and efficacy, demonstrating potential for neuromodulation of basal ganglia circuits.
This phase I/II adaptive dose-finding prospective interventional study evaluates the safety and efficacy of LIFU targeting the external globus pallidus in patients with Huntington's disease. Using MRI-derived individualized head models and real-time neuronavigation, the study employs a Bayesian optimal interval (BOIN) design with three pulse repetition frequencies: 10 Hz, 50 Hz, and 100 Hz. Patients are enrolled in sequential cohorts of three, with dose escalation guided by a utility-based approach integrating safety and efficacy data. Each patient receives ten LIFU sessions over five consecutive days, with two sessions daily. Motor function, cognitive function, functional assessments, and magnetic resonance imaging are evaluated before the first treatment session and after the final treatment session.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 10 Hz LIFU Group | Experimental | Device: Low-Intensity Focused Ultrasound Stimulation（LIFU）Low-Intensity Focused Ultrasound (LIFU), PRF 10 Hz |
| 50 Hz LIFU Group | Experimental | Device: Low-Intensity Focused Ultrasound Stimulation（LIFU）Low-Intensity Focused Ultrasound (LIFU), PRF 50 Hz |
| 100 Hz LIFU Group | Experimental | Device: Low-Intensity Focused Ultrasound Stimulation（LIFU）Low-Intensity Focused Ultrasound (LIFU), PRF 100 Hz |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Low-Intensity Focused Ultrasound Stimulation（LIFU）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. | Device | 10 Hz LIFU Group, 100 Hz LIFU Group, 50 Hz LIFU Group |
Primary outcome
Safety: Incidence of Dose-Limiting Toxicity (DLT)
Time frame: At any point during or immediately following intervention on day of LIFUS application
DLT refers to the occurrence of any of the following six events during LIFU treatment: 1. Second-degree scalp burn; 2. Clinical seizure; 3. Worsening of chorea or new movement disorder; 4. Significant deterioration of the patient's mood or mental status (e.g., anxiety, depression, hallucinations, excessive sleepiness); 5. Severe autonomic dysfunction, such as marked blood pressure fluctuations, abnormal heart rate, or respiratory distress; 6. Imaging-related safety events: new or significantly enlarged brain edema, new microbleeds, new infarcts, or structural abnormalities in the treatment area on post-treatment cranial MRI compared with baseline, deemed clinically significant by both radiologists and clinicians.
Efficacy: Change in Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS)
Time frame: Baseline and within 2 days after completing the 5-day LIFU treatment
The Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS) evaluates motor impairment in Huntington's disease. Score range: 0-124 Higher scores indicate more severe motor impairment Response is defined as a reduction of ≥4 points from baseline.
Comprehensive Benefit-Risk: Utility
Time frame: Within 2 days after completing the 5-day LIFU treatment
Utility is a composite measure integrating dose-limiting toxicity (DLT) and efficacy response to quantify the overall benefit-risk balance for each dose group. During the trial, the Utility value for each dose group is dynamically calculated using the U-BOIN design platform. In Stage II, the Utility value determines dose allocation for subsequent cohorts. At study completion, the dose group with the highest Utility value is identified as the optimal biological dose.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2026.
- Individual participant dataSharing planned: No
Related trials for Huntington Disease
Recruiting
Feasibility and Acceptability of an LLM-based Chatbot for Family Caregivers: Evaluation Study
Caregivers
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
RecruitingPhase 1
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
Huntington's Disease
RecruitingPhase 3
Pridopidine Phase 3 Study in Huntington's Disease
Huntington Disease
RecruitingPhase 2/3
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Huntington Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Low-Intensity Focused Ultrasound Stimulation（LIFU） in people with Huntington Disease, run by First Affiliated Hospital of Fujian Medical University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xin-Yuan Chen (+86 15005065282, fychenxinyuan@fjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Incidence of Dose-Limiting Toxicity (DLT) (time frame: At any point during or immediately following intervention on day of LIFUS application). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Apr 2026.