Clinical trial NCT05206513: Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Dyskinesia
Cerebral Palsy
Keywords: Cerebral Palsy
Vesicular monoamine transporter 2 (VMAT2) inhibitor
Dyskinesia
Neurocrine
Valbenazine
NBI-98854
Dystonia
Chorea
Athetosis
Athetoid cerebral palsy
Choreoathetosis
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
First posted: 2022-01-25
Enrollment: 80
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: 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
timeFrame: Baseline, Week 12 and Week 14
Secondary outcomes:
measure: Change in the Clinical Global Impression of Severity (CGI-S) Score from Baseline to Week 14
timeFrame: Baseline, Week 14
measure: 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
timeFrame: Baseline, Week 12 and Week 14
measure: Change in the Total Maximal Dystonia (TMD) Score of the UHDRS from Baseline to the Average of the Week 12 and Week 14 Assessments
timeFrame: Baseline, Week 12 and Week 14
measure: Patient Global Impression of Improvement (PGI-I) Score at Week 14
timeFrame: Week 14
measure: Caregiver Global Impression of Improvement (CaGI-I) Score at Week 14
timeFrame: Week 14
measure: Clinical Global Impression of Improvement (CGI-I) Score at Week 14
timeFrame: Week 14
measure: Goal Attainment Score at Week 14 Using the Goal Attainment Scale (GAS)
timeFrame: Week 14
measure: Change in Pain Assessment from Baseline to Week 14 Using the Faces Pain Scale-Revised (FPS-R)
timeFrame: Baseline, Week 14
measure: Change in the UHDRS Total Motor Score (TMS) from Baseline to the Average of the Week 12 and Week 14 Assessments
timeFrame: Baseline, Week 12 and Week 14
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Neurocrine Clinical Site
state: California
city: Aurora
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Stamford
country: United States
facility: Neurocrine Clinical Site
state: Connecticut
city: Gulf Breeze
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Miami
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Orlando
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Augusta
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Baltimore
country: United States
facility: Neurocrine Clinical Site
state: Maryland
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Farmington Hills
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Grand Rapids
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Saint Paul
country: United States
facility: Neurocrine Clinical Site
state: Minnesota
city: New York
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Cincinnati
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Columbus
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Oklahoma City
country: United States
facility: Neurocrine Clinical Site
state: Oklahoma
city: Portland
country: United States
facility: Neurocrine Clinical Site
state: Oregon
city: Dallas
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Fort Worth
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Houston
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Buenos Aires
country: Argentina
facility: Neurocrine Clinical Site
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Neurocrine Clinical Site
city: Clayton
country: Australia
facility: Neurocrine Clinical Site
city: Bruxelles
country: Belgium
facility: Neurocrine Clinical Site
city: Brasília
country: Brazil
facility: Neurocrine Clinical Site
city: Curitiba
country: Brazil
facility: Neurocrine Clinical Site
city: Porto Alegre
country: Brazil
facility: Neurocrine Clinical Site
city: Porto Alegre
country: Brazil
facility: Neurocrine Clinical Site
city: Zerifin
country: Israel
facility: Neurocrine Clinical Site
city: Calambrone
country: Italy
facility: Neurocrine Clinical Site
city: Firenze
country: Italy
facility: Neurocrine Clinical Site
city: Milano
country: Italy
facility: Neurocrine Clinical Site
city: Aguascalientes
country: Mexico
facility: Neurocrine Clinical Site
city: Gdańsk
country: Poland
facility: Neurocrine Clinical Site
city: Kraków
country: Poland
facility: Neurocrine Clinical Site
city: Oswiecim
country: Poland
facility: Neurocrine Clinical Site
city: Lisboa
country: Portugal
facility: Neurocrine Clinical Site
city: Lisboa
country: Portugal
facility: Neurocrine Clinical Site
city: Porto
country: Portugal
facility: Neurocrine Clinical Site
city: Barcelona
country: Spain
facility: Neurocrine Clinical Site
city: Madrid
country: Spain
facility: Neurocrine Clinical Site
city: San Sebastián
country: Spain
facility: Neurocrine Clinical Site
Study officials:
affiliation: Neurocrine Biosciences
name: Clinical Development Lead
role: STUDY_DIRECTOR
References: