Risperidone for the Treatment of Huntington's Disease Involuntary Movements

NCT04201834 | Completed Phase 2 Results FDA Drug FDA Device

• 1 other identifier: 4443
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the safety and benefit of risperidone for the treatment of chorea (involuntary movements) in Huntington's disease. Risperidone is commonly used in clinical practice to treat chorea, however, it has not been approved by the Food and Drug Administration (FDA) to treat chorea. This study will examine 1) whether the investigators see MRI changes with risperidone treatment and 2) whether sensors applied to the participants body can measure chorea and detect changes in chorea.

Conditions

Huntington Disease; Chorea

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) Score

  The UHDRS is a validated assessment of HD. The TMC score is a subset of the overall motor assessment and measures maximal chorea in seven different body regions. The TMC score ranges from 0 to 28 with higher scores indicating more chorea.

  Baseline to week 12

Secondary Outcomes (14)

• Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Motor Score

  Baseline to week 12

• Change From Baseline in Mean Epworth Sleepiness Scale (ESS)

  Baseline to week 12

• Change From Baseline in Mean Barnes Akathisia Scale Global Clinical Assessment of Akathisia Item Score

  Baseline to week 12

• Number of Participants With a Change in Clinical Global Impression of Change (CGI)

  Baseline to week 12

• Number of Participants With a Change in Patient Global Impression of Change (PGI)

  Baseline to week 12

Study Arms (1)

Risperidone

EXPERIMENTAL

Participants will initiate risperidone 0.5 mg nightly the day after the baseline visit. Dose assessment will occur at pre-specified intervals during the titration phase (week 2, 3, 4, 6, 7). The investigator will increase the dose by 0.5 mg at the week 2, week 3, week 4, and week 6 visits until either optimal chorea benefit has been obtained, an intolerable adverse event occurs, or the maximum allowable dose (3.0 mg) is reached.

Drug: Risperidone; Device: BioStamp nPoint device

Interventions

Risperidone DRUG

capsule or tablet, 0.5 mg

Risperidone

BioStamp nPoint device DEVICE

MC10 has developed the BioStamp nPointTM, a FDA 510(k) cleared medical device. This multimodal, reusable and rechargeable biosensor uses flexible and stretchable electronics to enable unobtrusive wear on the body and monitoring in the home. The sensors have accelerometry, gyroscopy, and ECG/EMG capabilities. A docking station enables wireless recharging and data collection.

Risperidone

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Manifest HD (Diagnostic Confidence Level 4 + CAG repeat ≥ 37 or family history of HD)
• UHDRS Total Maximal Chorea (TMC) ≥ 8
• UHDRS Total Functional Capacity ≥ 5
• Subject willing and able to provide written informed consent OR legally authorized representative provides written informed consent and subject provides assent\*
• Between 18 and 65 years of age

You may not qualify if:

• Use of antipsychotic, levodopa, dopamine agonist, monoamine oxidase inhibitor or other disallowed medication in the 30 days prior to the baseline visit (see Section 4.2.5)\*
• Prior non-response to risperidone or intolerability to risperidone (in the investigator's opinion)
• Allergy or hypersensitivity to risperidone
• Dysphagia that in the investigator's opinion would preclude participation in the study
• Active suicidal ideation or psychiatric condition that in the investigator's opinion would preclude study participation
• QTc \> 460 msec for women and QTc \> 450 msec for men on 12-lead EKG
• History of cardiac arrhythmia or congenital long QT syndrome
• Significant renal impairment (creatinine clearance \< 30 mL/min as estimated by the Cockgroft-Gault formula) or hepatic impairment (AST or ALT \> 2.5 times upper limit of normal OR alkaline phosphatase or total bilirubin \> 2 times upper limit of normal)
• Active drug or alcohol abuse or dependence
• Pregnant or breast-feeding
• Any contraindication to MRI (e.g. pacemakers, aneurysm clips, metallic prostheses, shrapnel fragments, claustrophobia)
• History of active (clinically significant) skin disorder that would interfere with sensor adherence
• History of allergic response to adhesives
• Pacemaker, AICD, or other implantable stimulator
• Use of an investigational drug in the 30 days prior to the baseline visit

Sponsors & Collaborators

• University of Rochester: lead

Study Sites (1)

URMC Neurology; 919 Westfall Rd, Building C, Suite 100

Rochester, New York, 14618, United States

Location

MeSH Terms

Conditions

Huntington Disease; Chorea

Interventions

Risperidone

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pyrimidinones; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Limitations and Caveats

Small number of subjects.

Results Point of Contact

• Title: Ruth Schneider, MD
• Organization: University of Rochester

Ruth_schneider@urmc.rochester.edu

585-341-7500

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Masking Details: Outcomes assessor will be masked to the visit.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: December 13, 2019
• First Posted: December 17, 2019
• Study Start: August 13, 2020
• Primary Completion: December 30, 2023
• Study Completion: December 30, 2023
• Last Updated: March 21, 2025
• Results First Posted: March 21, 2025

Record last verified: 2025-03

Locations

US (1)