Risperidone for the Treatment of Huntington's Disease Involuntary Movements

University of Rochester

Status and phase: Completed; Phase 2

Conditions: Chorea; Huntington Disease

Treatments: Drug: Risperidone; Device: BioStamp nPoint device

Study type: Interventional

Funder types: Other

Identifiers

NCT04201834

4443

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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 < 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
• Inability to complete study activities, as determined by the study team
• Clinically significant parkinsonism as determined by expert investigator assessment

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Risperidone

Experimental group

Description:

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.

Treatment:

Device: BioStamp nPoint device

Drug: Risperidone