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

Sponsor

Neurocrine Biosciences (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05206513

Collaborator

(none)

144

Enrollment

Location

Arms

27.6

Anticipated Duration (Months)

5.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The primary objective of this study is to evaluate the efficacy of valbenazine versus placebo in participants aged 6 to 70 years for the treatment of dyskinesia due to cerebral palsy (DCP).

Condition or Disease	Intervention/Treatment	Phase
Dyskinesia Cerebral Palsy	Drug: Placebo Drug: Valbenazine	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

144 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Apr 15, 2022

Anticipated Primary Completion Date :

Dec 1, 2023

Anticipated Study Completion Date :

Aug 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo Placebo dosed for 14 weeks followed by open label valbenazine treatment for 32 weeks	Drug: Placebo Capsule, administered orally once daily Drug: Valbenazine Capsule, administered orally once daily Other Names: NBI-98854
Experimental: Valbenazine Valbenazine dosed for 14 weeks followed by open label valbenazine treatment for 32 weeks	Drug: Valbenazine Capsule, administered orally once daily Other Names: NBI-98854

Arm

Intervention/Treatment

Placebo Comparator: Placebo

Placebo dosed for 14 weeks followed by open label valbenazine treatment for 32 weeks

Drug: Placebo

Capsule, administered orally once daily

Drug: Valbenazine

Capsule, administered orally once daily

Other Names:

NBI-98854

Experimental: Valbenazine

Valbenazine dosed for 14 weeks followed by open label valbenazine treatment for 32 weeks

Drug: Valbenazine

Capsule, administered orally once daily

Other Names:

NBI-98854

Outcome Measures

Primary Outcome Measures

The change in the Total Maximal Chorea (TMC) score of the Unified Huntington Disease Rating Scale (UHDRS) from baseline to Week 14. [Baseline and Week 14]
The change in the Total Maximal Dystonia (TMD) score of the UHDRS from baseline to Week 14. [Baseline and Week 14]

Secondary Outcome Measures

Change from Baseline in Clinical Global Impression of Severity (CGI-S) Score to Week 14. [Baseline and Week 14]
The Patient Global Impression of Improvement (PGI-I) score at Week 14. [Week 14]
The Caregiver Global Impression of Improvement (CaGI-I) score at Week 14. [Week 14]
The 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 Movement Disorders Childhood Rating Scale (MD-CRS) Part I score from baseline to Week 14 (ages 6 to 17 years). [Baseline and Week 14]
Change in Quality of Life in Neurological Disorders (Neuro-QoL) (Lower Extremity Function Short Form, Upper Extremity Function Short Form, and Satisfaction with Social Roles and Activities Short Form) from baseline to Week 14 (ages 18 to 70 years). [Baseline and Week 14]
Change in Cerebral Palsy Quality of Life-Teen (CP QoL-Teen) from baseline to Week 14. [Baseline and Week 14]
Change in CP QoL-Teen (caregiver-proxy report) from baseline to Week 14 (ages 13 to 17 years). [Baseline and Week 14]
Change in Cerebral Palsy Quality of Life-Child (CP QoL-Child) from baseline to Week 14 (ages 6 to 12 years). [Baseline and Week 14]
Change in CP QoL-Child (caregiver-proxy report) from baseline to Week 14 (ages 6 to 12 years). [Baseline and Week 14]
Change in pain assessment from baseline to Week 14 using the Faces Pain Scale-Revised (FPS-R). [Baseline and Week 14]
Change in the UHDRS Total Motor Score (TMS) (site rater) from baseline to Week 14. [Baseline and Week 14]

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 70 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Medically confirmed diagnosis of dyskinesia (dystonia and/or choreoathetosis) due to Cerebral palsy (CP). The dyskinetic movements must cause disability and be of at least moderate severity.
Medical conditions are stable and expected to remain stable throughout the study.

Key Exclusion Criteria:

Subjects 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, or requirement to take medications by gastro-enteral 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 ECG abnormalities.