Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy

Study Overview
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Brief Summary
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The primary objective of this study is to evaluate the efficacy of valbenazine versus placebo on improving chorea in participants aged 6 to 70 years who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.

Official Title
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy

Conditions
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Dyskinesia, Cerebral Palsy

Intervention / Treatment
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* Drug: Placebo
* Drug: Valbenazine


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Medically confirmed diagnosis of DCP (that is, a hyperkinetic movement disorder due to cerebral palsy [CP]) with choreiform movements. Medical conditions are stable and expected to remain stable throughout the study. Key Exclusion Criteria: Participants will be excluded from the study if they meet any of the following criteria: Are pregnant or breastfeeding. Have a clinical diagnosis or history of dyskinesia due to condition other than CP. Have inability to swallow soft solids, unless medications can be administered via a gastrostomy tube. Have any suicidal behavior or suicidal ideation in the year prior to screening or on Day 1. Is a substance abuser of any compound. Known history of long QT syndrome or cardiac tachyarrhythmia, or clinically significant electrocardiogram (ECG) abnormalities.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Placebo dosed for 14 weeks followed by open-label valbenazine treatment for 32 weeks | Drug: Placebo<br>* Capsule, administered once daily orally or via gastrostomy tube<br>Drug: Valbenazine<br>* Capsule, administered once daily orally or via gastrostomy tube<br>* Other names: NBI-98854;|
| Experimental: Valbenazine<br>Valbenazine dosed for 14 weeks followed by open-label valbenazine treatment for 32 weeks | Drug: Valbenazine<br>* Capsule, administered once daily orally or via gastrostomy tube<br>* Other names: NBI-98854;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the Total Maximal Chorea (TMC) score of the Unified Huntington Disease Rating Scale (UHDRS) from baseline to the average of the Week 12 and Week 14 assessments |  | Baseline, Week 12 and Week 14 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the Clinical Global Impression of Severity (CGI-S) score from baseline to Week 14 |  | Baseline, Week 14 | 
| Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I score from baseline to Week 14 |  | Baseline, Week 14 | 
| Change in the Total Maximal Dystonia (TMD) score of the UHDRS from baseline to the average of the Week 12 and Week 14 assessments |  | Baseline, Week 12 and Week 14 | 
| Patient Global Impression of Improvement (PGI-I) score at Week 14 |  | Week 14 | 
| Caregiver Global Impression of Improvement (CaGI-I) score at Week 14 |  | Week 14 | 
| Clinical Global Impression of Improvement (CGI-I) score at Week 14 |  | Week 14 | 
| Goal attainment score at Week 14 using the Goal Attainment Scale (GAS) |  | Week 14 | 
| Change in pain assessment from baseline to Week 14 using the Faces Pain Scale-Revised (FPS-R) |  | Baseline, Week 14 | 
| Change in the UHDRS Total Motor Score (TMS) from baseline to the average of the Week 12 and Week 14 assessments |  | Baseline, Week 12 and Week 14 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cerebral Palsy, Vesicular monoamine transporter 2 (VMAT2) inhibitor, Dyskinesia, Neurocrine, Valbenazine, NBI-98854, Dystonia, Chorea, Athetosis, Athetoid cerebral palsy, Choreoathetosis