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

Phase 3Active Not RecruitingNCT05206513

Neurocrine Biosciences86 enrolled

Overview

The primary objective of this study is to evaluate the efficacy of valbenazine versus placebo on improving chorea in pediatric and adult participants who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Dyskinesia Cerebral Palsy

Interventions

PlaceboDRUG

Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube

ValbenazineDRUG

Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube

Eligibility

Sex: ALLMin age: 6 YearsMax age: 70 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: 1. Medically confirmed diagnosis of DCP (that is, a hyperkinetic movement disorder due to cerebral palsy \[CP\]) with choreiform movements. 2. 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: 1. Are pregnant or breastfeeding. 2. Have a clinical diagnosis or history of dyskinesia due to condition other than CP. 3. Have inability to swallow soft foods, unless medications can be administered via gastrostomy/gastrojejunostomy tube. 4. Have any suicidal behavior or suicidal ideation in the year prior to screening or on Day 1. 5. Is a substance abuser of any compound. 6. Known history of long QT syndrome or cardiac tachyarrhythmia, or clinically significant electrocardiogram (ECG) abnormalities.

Locations (38)

Outcomes

Primary Outcomes

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

Time frame: Baseline, Week 12 and Week 14

Secondary Outcomes

Change in the Clinical Global Impression of Severity (CGI-S) Score from Baseline to Week 14

Time frame: Baseline, Week 14

Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I Score from Baseline to the Average of the Week 12 and Week 14 Assessments

Time frame: Baseline, Week 12 and 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

Time frame: Baseline, Week 12 and Week 14

Patient Global Impression of Improvement (PGI-I) Score at Week 14

Time frame: Week 14

Caregiver Global Impression of Improvement (CaGI-I) Score at Week 14

Time frame: Week 14

Clinical Global Impression of Improvement (CGI-I) Score at Week 14

Time frame: Week 14

Goal Attainment Score at Week 14 Using the Goal Attainment Scale (GAS)

Time frame: Week 14

Change in Pain Assessment from Baseline to Week 14 Using the Faces Pain Scale-Revised (FPS-R)

Time frame: Baseline, Week 14

Change in the UHDRS Total Motor Score (TMS) from Baseline to the Average of the Week 12 and Week 14 Assessments

Data from ClinicalTrials.gov

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Neurocrine Clinical Site

San Diego, California, United States

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Aurora, Colorado, United States

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Stamford, Connecticut, United States

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Gulf Breeze, Florida, United States

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Miami, Florida, United States

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Orlando, Florida, United States

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Chicago, Illinois, United States

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Baltimore, Maryland, United States

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Boston, Massachusetts, United States

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Farmington Hills, Michigan, United States

...and 28 more locations