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

NCT05206513 | Completed Phase 3 DMC FDA Drug

• 2 other identifiers: NBI-98854-DCP3018EUCT
• Study Type: interventional
• Target: 86
• Locations: 10 countries
• Sites: 38

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.

Conditions

Cerebral Palsy; Dyskinesia

Keywords

Cerebral Palsy; Vesicular monoamine transporter 2 (VMAT2) inhibitor; Dyskinesia; Neurocrine; Valbenazine; NBI-98854; Dystonia; Chorea; Athetosis; Athetoid cerebral palsy; Choreoathetosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (9)

• 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

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo; Drug: Valbenazine

Valbenazine

EXPERIMENTAL

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.

Drug: Valbenazine

Interventions

Placebo DRUG

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

Placebo

Valbenazine DRUG

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

Also known as: NBI-98854

Placebo; Valbenazine

Eligibility Criteria

• Age: 6 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Medically confirmed diagnosis of DCP (that is, a hyperkinetic movement disorder due to cerebral palsy \[CP\]) with choreiform movements.
• Medical conditions are stable and expected to remain stable throughout the study.

You may not qualify if:

• Participants 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 foods, unless medications can be administered via gastrostomy/gastrojejunostomy 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 electrocardiogram (ECG) abnormalities.

Sponsors & Collaborators

• Neurocrine Biosciences: lead

Study Sites (38)

Neurocrine Clinical Site

San Diego, California, 92123, United States

Location

Neurocrine Clinical Site

Aurora, Colorado, 80045, United States

Location

Neurocrine Clinical Site

Stamford, Connecticut, 06905, United States

Location

Neurocrine Clinical Site

Gulf Breeze, Florida, 32561, United States

Location

Neurocrine Clinical Site

Miami, Florida, 33155, United States

Location

Neurocrine Clinical Site

Orlando, Florida, 32806, United States

Location

Neurocrine Clinical Site

Chicago, Illinois, 60611, United States

Location

Neurocrine Clinical Site

Baltimore, Maryland, 21205, United States

Location

Neurocrine Clinical Site

Boston, Massachusetts, 02115, United States

Location

Neurocrine Clinical Site

Farmington Hills, Michigan, 48334, United States

Location

Neurocrine Clinical Site

Saint Paul, Minnesota, 55101, United States

Location

Neurocrine Clinical Site

New York, New York, 10003, United States

Location

Neurocrine Clinical Site

Cincinnati, Ohio, 45229, United States

Location

Neurocrine Clinical Site

Columbus, Ohio, 43205, United States

Location

Neurocrine Clinical Site

Oklahoma City, Oklahoma, 73120, United States

Location

Neurocrine Clinical Site

Portland, Oregon, 97239, United States

Location

Neurocrine Clinical Site

Dallas, Texas, 75390-8857, United States

Location

Neurocrine Clinical Site

Fort Worth, Texas, 76104, United States

Location

Neurocrine Clinical Site

Houston, Texas, 77030, United States

Location

Neurocrine Clinical Site

Buenos Aires, C1060AAF, Argentina

Location

Neurocrine Clinical Site

Buenos Aires, C1425EFD, Argentina

Location

Neurocrine Clinical Site

Brussels, 1200, Belgium

Location

Neurocrine Clinical Site

Brasília, 70684-831, Brazil

Location

Neurocrine Clinical Site

Curitiba, 80250-060, Brazil

Location

Neurocrine Clinical Site

Porto Alegre, 90035-001, Brazil

Location

Neurocrine Clinical Site

Porto Alegre, 90050-170, Brazil

Location

Neurocrine Clinical Site

Ẕerifin, 7030000, Israel

Location

Neurocrine Clinical Site

Calambrone, 56128, Italy

Location

Neurocrine Clinical Site

Milan, 3. 20162, Italy

Location

Neurocrine Clinical Site

Aguascalientes, Mexico

Location

Neurocrine Clinical Site

Gdansk, 80-389, Poland

Location

Neurocrine Clinical Site

Krakow, 30-539, Poland

Location

Neurocrine Clinical Site

Oświęcim, 32-600, Poland

Location

Neurocrine Clinical Site

Lisbon, 1169-045, Portugal

Location

Neurocrine Clinical Site

Lisbon, 1649-035, Portugal

Location

Neurocrine Clinical Site

Porto, 4050-651, Portugal

Location

Neurocrine Clinical Site

Barcelona, 08038, Spain

Location

Neurocrine Clinical Site

Madrid, 28006, Spain

Location

MeSH Terms

Conditions

Dyskinesias; Cerebral Palsy; Dystonia; Chorea; Athetosis

Interventions

valbenazine

Condition Hierarchy (Ancestors)

Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Brain Damage, Chronic; Brain Diseases

Study Officials

• Clinical Development Lead

  Neurocrine Biosciences

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2022
• First Posted: January 25, 2022
• Study Start: April 15, 2022
• Primary Completion: October 16, 2025
• Study Completion: March 31, 2026
• Last Updated: April 29, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (19); BR (4); BE (1); AR (2); ES (2); IT (2); PL (3); PT (3); IL (1); ME (1)