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

Phase 3

Recruiting

• Conditions: Dyskinesia; Cerebral Palsy
• Interventions: Drug: Placebo; Drug: Valbenazine

• Registration Number: NCT05206513
• Lead Sponsor: Neurocrine Biosciences

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-01-10
• Study First Submitted QC: 2022-01-12
• Study First Posted: 2022-01-25
• Study Start: 2022-04-15
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-21
• Last Update Posted: 2025-05-25
• Primary Completion: 2025-06
• Study Completion: 2026-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 80

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment. Participants may opt to enter an open-label valbenazine treatment extension phase.
Placebo	Valbenazine	Placebo dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment. Participants may opt to enter an open-label valbenazine treatment extension phase.
Valbenazine	Valbenazine	Valbenazine dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment. Participants may opt to enter an open-label valbenazine treatment extension phase.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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 the Average of the Week 12 and Week 14 Assessments	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	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

Trial Locations

Locations (1)

Neurocrine Clinical Site; 🇪🇸 San Sebastián, Spain