Cerebral Palsy, Dyskinesia
Conditions
Keywords
Cerebral Palsy, Vesicular monoamine transporter 2 (VMAT2) inhibitor, Dyskinesia, Neurocrine, Valbenazine, NBI-98854, Dystonia, Chorea, Athetosis, Athetoid cerebral palsy, Choreoathetosis
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.
Interventions
DRUGPlacebo
Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube
DRUGValbenazine
Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube
Sponsors
Neurocrine Biosciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 70 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| 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 Pain Assessment from Baseline to Week 14 Using the Faces Pain Scale-Revised (FPS-R) | Baseline, Week 14 |
| Change in the UHDRS Total Motor Score (TMS) from Baseline to the Average of the Week 12 and Week 14 Assessments | Baseline, Week 12 and Week 14 |
Countries
Argentina, Belgium, Brazil, Israel, Italy, Mexico, Poland, Portugal, Spain, United States
Contacts
STUDY_DIRECTORClinical Development Lead
Neurocrine Biosciences
Outcome results
None listed