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Phase 2 N=6 Treatment

Risperidone for the Treatment of Huntington's Disease Involuntary Movements

Huntington Disease · Chorea

Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Mar 2025
Primary outcome: Primary: Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) Score — -2.4 score on a scale — p=0.27

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Risperidone (Drug); BioStamp nPoint device (Device)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
University of Rochester
Primary completion
Dec 2023

Outcome Measures

OutcomeResultp-value
PRIMARY
Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) Score
-2.4 0.27
SECONDARY
Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Motor Score
-.02 0.92
SECONDARY
Change From Baseline in Mean Epworth Sleepiness Scale (ESS)
1.2 0.36
SECONDARY
Change From Baseline in Mean Barnes Akathisia Scale Global Clinical Assessment of Akathisia Item Score
-0.2 0.62
SECONDARY
Number of Participants With a Change in Clinical Global Impression of Change (CGI)
3; 1; 1
SECONDARY
Number of Participants With a Change in Patient Global Impression of Change (PGI)
3; 1
SECONDARY
Change From Baseline in Mean Quantitative Motor (Q-Motor) Right Hand Speeded Finger Tapping Variability
-0.04 0.11
SECONDARY
Change From Baseline in Mean Short Problem Behavior Assessment (Short PBA-S) Irritability and Aggression Score
3.4 0.02 sig
SECONDARY
Columbia Suicide Severity Rating Scale( C-SSRS)
0; 5
SECONDARY
Change From Baseline in Mean Apathy Scale Score
0.8 0.86
SECONDARY
Change From Baseline in Mean Hospital Anxiety and Depression Scale-Depression Score
-1.0 0.27
SECONDARY
Change From Baseline in Mean Montreal Cognitive Assessment
-0.6 0.61
SECONDARY
Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Independence Scale Score
4.0 0.37
SECONDARY
Change From Baseline in Mean Hospital Anxiety and Depression Scale-Anxiety Score
-1.4 0.30
SECONDARY
Change From Baseline in MC10-Assessed Mean Gait Cadence
0.6 0.82

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.

Eligibility Criteria

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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 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
  • Inability to complete study activities, as determined by the study team
  • Clinically significant parkinsonism as determined by expert investigator assessment
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04201834). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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