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

NCT ID: NCT05206513

Last Updated: 2025-08-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

86 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-15

Study Completion Date

2026-06-30

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 pediatric and adult participants who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.

Detailed Description

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Conditions

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Dyskinesia Cerebral Palsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Valbenazine

Intervention Type DRUG

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

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.

Group Type EXPERIMENTAL

Valbenazine

Intervention Type DRUG

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

Interventions

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Placebo

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

Intervention Type DRUG

Valbenazine

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

Intervention Type DRUG

Other Intervention Names

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NBI-98854

Eligibility Criteria

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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.

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.
Minimum Eligible Age

6 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Neurocrine Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Development Lead

Role: STUDY_DIRECTOR

Neurocrine Biosciences

Locations

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Neurocrine Clinical Site

San Diego, California, United States

Site Status

Neurocrine Clinical Site

Aurora, Colorado, United States

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Neurocrine Clinical Site

Stamford, Connecticut, United States

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Neurocrine Clinical Site

Gulf Breeze, Florida, United States

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Neurocrine Clinical Site

Miami, Florida, United States

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Neurocrine Clinical Site

Orlando, Florida, United States

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Neurocrine Clinical Site

Chicago, Illinois, United States

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Neurocrine Clinical Site

Baltimore, Maryland, United States

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Neurocrine Clinical Site

Boston, Massachusetts, United States

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Neurocrine Clinical Site

Farmington Hills, Michigan, United States

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Neurocrine Clinical Site

Saint Paul, Minnesota, United States

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Neurocrine Clinical Site

New York, New York, United States

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Neurocrine Clinical Site

Cincinnati, Ohio, United States

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Neurocrine Clinical Site

Columbus, Ohio, United States

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Neurocrine Clinical Site

Oklahoma City, Oklahoma, United States

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Neurocrine Clinical Site

Portland, Oregon, United States

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Neurocrine Clinical Site

Dallas, Texas, United States

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Neurocrine Clinical Site

Fort Worth, Texas, United States

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Neurocrine Clinical Site

Houston, Texas, United States

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Neurocrine Clinical Site

Buenos Aires, , Argentina

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Neurocrine Clinical Site

Buenos Aires, , Argentina

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Neurocrine Clinical Site

Brussels, , Belgium

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Neurocrine Clinical Site

Brasília, , Brazil

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Neurocrine Clinical Site

Curitiba, , Brazil

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Neurocrine Clinical Site

Porto Alegre, , Brazil

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Neurocrine Clinical Site

Porto Alegre, , Brazil

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Neurocrine Clinical Site

Ẕerifin, , Israel

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Neurocrine Clinical Site

Calambrone, , Italy

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Neurocrine Clinical Site

Milan, , Italy

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Neurocrine Clinical Site

Aguascalientes, , Mexico

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Neurocrine Clinical Site

Gdansk, , Poland

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Neurocrine Clinical Site

Krakow, , Poland

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Neurocrine Clinical Site

Oświęcim, , Poland

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Neurocrine Clinical Site

Lisbon, , Portugal

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Neurocrine Clinical Site

Lisbon, , Portugal

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Neurocrine Clinical Site

Porto, , Portugal

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Neurocrine Clinical Site

Barcelona, , Spain

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Neurocrine Clinical Site

Madrid, , Spain

Site Status

Countries

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United States Argentina Belgium Brazil Israel Italy Mexico Poland Portugal Spain

Other Identifiers

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EUCT

Identifier Type: OTHER

Identifier Source: secondary_id

NBI-98854-DCP3018

Identifier Type: -

Identifier Source: org_study_id

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