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

Neurocrine Biosciences

Status and phase: Active, not recruiting; Phase 3

Conditions: Cerebral Palsy; Dyskinesia

Treatments: Drug: Placebo; Drug: Valbenazine

Study type: Interventional

Funder types: Industry

Identifiers

NCT05206513

NBI-98854-DCP3018

EUCT (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 86 patients

Sex: All

Ages: 6 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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86 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Valbenazine

Drug: Placebo

Valbenazine

Experimental group

Description:

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.

Treatment:

Drug: Valbenazine