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

Cerebral Palsy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05206513
• Other study ID #: NBI-98854-DCP3018
• Secondary ID: EUCT
• Status: Recruiting
• Phase: Phase 3
• Start date: April 15, 2022
• Est. completion date: January 2026

Study information

• Verified date: March 2024
• Source: Neurocrine Biosciences
• Contact: Neurocrine Medical Information Call Center
• Phone: 877-641-3461
• Email: medinfo[@]neurocrine.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: January 2026
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 70 Years

Eligibility

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 solids, unless medications can be administered via a gastrostomy 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.

Related Conditions & MeSH terms

• Cerebral Palsy
• Dyskinesia
• Dyskinesias
• Paralysis

Intervention

Drug:
• Placebo
Capsule, administered once daily orally or via gastrostomy tube
• Valbenazine
Capsule, administered once daily orally or via gastrostomy tube

Locations

Country	Name	City	State
Argentina	Neurocrine Clincial Site	Ciudad Autónoma de Buenos Aires
Poland	Neurocrine Clinical Site	Gdansk
Poland	Neurocrine Clinical Site	Oswiecim
Spain	Neurocrine Clinical Site	Barcelona
United States	Neurocrine Clinical Site	Augusta	Georgia
United States	Neurocrine Clinical Site	Aurora	Colorado
United States	Neurocrine Clinical Site	Chicago	Illinois
United States	Neurocrine Clinical Site	Columbus	Ohio
United States	Neurocrine Clinical Site	Dallas	Texas
United States	Neurocrine Clinical Site	Farmington Hills	Michigan
United States	Neurocrine Clinical Site	Fort Worth	Texas
United States	Neurocrine Clinical Site	Grand Rapids	Michigan
United States	Neurocrine Clinical Site	Gulf Breeze	Florida
United States	Neurocrine Clinical Site	Houston	Texas
United States	Neurocrine Clinical Site	Miami	Florida
United States	Neurocrine Clinical Site	New York	New York
United States	Neurocrine Clinical Site	Oklahoma City	Oklahoma
United States	Neurocrine Clinical Site	Saint Paul	Minnesota
United States	Neurocrine Clinical Site	San Diego	California
United States	Neurocrine Clinical Site	Stamford	Connecticut

Sponsors (1)

Lead Sponsor	Collaborator
Neurocrine Biosciences

Countries where clinical trial is conducted

United States,  Argentina,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in the Clinical Global Impression of Severity (CGI-S) score from baseline to Week 14		Baseline, Week 14
Secondary	Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I score from baseline to Week 14		Baseline, Week 14
Secondary	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
Secondary	Patient Global Impression of Improvement (PGI-I) score at Week 14		Week 14
Secondary	Caregiver Global Impression of Improvement (CaGI-I) score at Week 14		Week 14
Secondary	Clinical Global Impression of Improvement (CGI-I) score at Week 14		Week 14
Secondary	Goal attainment score at Week 14 using the Goal Attainment Scale (GAS)		Week 14
Secondary	Change in pain assessment from baseline to Week 14 using the Faces Pain Scale-Revised (FPS-R)		Baseline, Week 14
Secondary	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

External Links

•   Study website - Kinect-DCP Study