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| ID | Type | Description | Link |
|---|---|---|---|
| EUCT | Other Identifier | 2022-502713-29-00 |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube |
| |
| Measure | Description | 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 |
| Measure | Description | 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 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Participants will be excluded from the study if they meet any of the following criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Development Lead | Neurocrine Biosciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurocrine Clinical Site | San Diego | California | 92123 | United States | ||
| Neurocrine Clinical Site |
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| Valbenazine |
| Drug |
Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube |
|
|
| 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 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Neurocrine Clinical Site | Stamford | Connecticut | 06905 | United States |
| Neurocrine Clinical Site | Gulf Breeze | Florida | 32561 | United States |
| Neurocrine Clinical Site | Miami | Florida | 33155 | United States |
| Neurocrine Clinical Site | Orlando | Florida | 32806 | United States |
| Neurocrine Clinical Site | Chicago | Illinois | 60611 | United States |
| Neurocrine Clinical Site | Baltimore | Maryland | 21205 | United States |
| Neurocrine Clinical Site | Boston | Massachusetts | 02115 | United States |
| Neurocrine Clinical Site | Farmington Hills | Michigan | 48334 | United States |
| Neurocrine Clinical Site | Saint Paul | Minnesota | 55101 | United States |
| Neurocrine Clinical Site | New York | New York | 10003 | United States |
| Neurocrine Clinical Site | Cincinnati | Ohio | 45229 | United States |
| Neurocrine Clinical Site | Columbus | Ohio | 43205 | United States |
| Neurocrine Clinical Site | Oklahoma City | Oklahoma | 73120 | United States |
| Neurocrine Clinical Site | Portland | Oregon | 97239 | United States |
| Neurocrine Clinical Site | Dallas | Texas | 75390-8857 | United States |
| Neurocrine Clinical Site | Fort Worth | Texas | 76104 | United States |
| Neurocrine Clinical Site | Houston | Texas | 77030 | United States |
| Neurocrine Clinical Site | Buenos Aires | C1060AAF | Argentina |
| Neurocrine Clinical Site | Buenos Aires | C1425EFD | Argentina |
| Neurocrine Clinical Site | Brussels | 1200 | Belgium |
| Neurocrine Clinical Site | Brasília | 70684-831 | Brazil |
| Neurocrine Clinical Site | Curitiba | 80250-060 | Brazil |
| Neurocrine Clinical Site | Porto Alegre | 90035-001 | Brazil |
| Neurocrine Clinical Site | Porto Alegre | 90050-170 | Brazil |
| Neurocrine Clinical Site | Ẕerifin | 7030000 | Israel |
| Neurocrine Clinical Site | Calambrone | 56128 | Italy |
| Neurocrine Clinical Site | Milan | 3. 20162 | Italy |
| Neurocrine Clinical Site | Aguascalientes | Mexico |
| Neurocrine Clinical Site | Gdansk | 80-389 | Poland |
| Neurocrine Clinical Site | Krakow | 30-539 | Poland |
| Neurocrine Clinical Site | Oświęcim | 32-600 | Poland |
| Neurocrine Clinical Site | Lisbon | 1169-045 | Portugal |
| Neurocrine Clinical Site | Lisbon | 1649-035 | Portugal |
| Neurocrine Clinical Site | Porto | 4050-651 | Portugal |
| Neurocrine Clinical Site | Barcelona | 08038 | Spain |
| Neurocrine Clinical Site | Madrid | 28006 | Spain |
| ID | Term |
|---|---|
| D020820 | Dyskinesias |
| D002547 | Cerebral Palsy |
| D004421 | Dystonia |
| D002819 | Chorea |
| D001264 | Athetosis |
| ID | Term |
|---|---|
| D009069 | Movement Disorders |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
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| ID | Term |
|---|---|
| C000603978 | valbenazine |
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