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

Primary Purpose

Dyskinesia, Cerebral Palsy

Status
Recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Placebo
Valbenazine
Sponsored by
Neurocrine Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dyskinesia focused on measuring Cerebral Palsy, Vesicular monoamine transporter 2 (VMAT2) inhibitor, Dyskinesia, Neurocrine, Valbenazine, NBI-98854, Dystonia, Chorea, Athetosis, Athetoid cerebral palsy, Choreoathetosis

Eligibility Criteria

6 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  1. Medically confirmed diagnosis of dyskinesia (dystonia and/or choreoathetosis) due to Cerebral palsy (CP). The dyskinetic movements must cause disability and be of at least moderate severity.
  2. Medical conditions are stable and expected to remain stable throughout the study.

Key Exclusion Criteria:

Subjects 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 solids, or requirement to take medications by gastro-enteral 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 ECG abnormalities.

Sites / Locations

  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting
  • Neurocrine Clinical SiteRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

Valbenazine

Arm Description

Placebo dosed for 14 weeks followed by open-label valbenazine treatment for 32 weeks

Valbenazine dosed for 14 weeks followed by open-label valbenazine treatment for 32 weeks

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change in the Clinical Global Impression of Severity (CGI-S) score from baseline to Week 14
Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I score from baseline to 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
Patient Global Impression of Improvement (PGI-I) score at Week 14
Caregiver Global Impression of Improvement (CaGI-I) score at Week 14
Clinical Global Impression of Improvement (CGI-I) score at Week 14
Goal attainment score at Week 14 using the Goal Attainment Scale (GAS)
Change in pain assessment from baseline to Week 14 using the Faces Pain Scale-Revised (FPS-R)
Change in the UHDRS Total Motor Score (TMS) from baseline to the average of the Week 12 and Week 14 assessments

Full Information

First Posted
January 10, 2022
Last Updated
August 17, 2023
Sponsor
Neurocrine Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT05206513
Brief Title
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

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 15, 2022 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
August 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The primary objective of this study is to evaluate the efficacy of valbenazine versus placebo on improving chorea in participants aged 6 to 70 years who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dyskinesia, Cerebral Palsy
Keywords
Cerebral Palsy, Vesicular monoamine transporter 2 (VMAT2) inhibitor, Dyskinesia, Neurocrine, Valbenazine, NBI-98854, Dystonia, Chorea, Athetosis, Athetoid cerebral palsy, Choreoathetosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo dosed for 14 weeks followed by open-label valbenazine treatment for 32 weeks
Arm Title
Valbenazine
Arm Type
Experimental
Arm Description
Valbenazine dosed for 14 weeks followed by open-label valbenazine treatment for 32 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Capsule, administered once daily orally or via gastrostomy tube
Intervention Type
Drug
Intervention Name(s)
Valbenazine
Other Intervention Name(s)
NBI-98854
Intervention Description
Capsule, administered once daily orally or via gastrostomy tube
Primary Outcome Measure Information:
Title
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
Time Frame
Baseline, Week 12 and Week 14
Secondary Outcome Measure Information:
Title
Change in the Clinical Global Impression of Severity (CGI-S) score from baseline to Week 14
Time Frame
Baseline, Week 14
Title
Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I score from baseline to Week 14
Time Frame
Baseline, Week 14
Title
Change in the Total Maximal Dystonia (TMD) score of the UHDRS from baseline to the average of the Week 12 and Week 14 assessments
Time Frame
Baseline, Week 12 and Week 14
Title
Patient Global Impression of Improvement (PGI-I) score at Week 14
Time Frame
Week 14
Title
Caregiver Global Impression of Improvement (CaGI-I) score at Week 14
Time Frame
Week 14
Title
Clinical Global Impression of Improvement (CGI-I) score at Week 14
Time Frame
Week 14
Title
Goal attainment score at Week 14 using the Goal Attainment Scale (GAS)
Time Frame
Week 14
Title
Change in pain assessment from baseline to Week 14 using the Faces Pain Scale-Revised (FPS-R)
Time Frame
Baseline, Week 14
Title
Change in the UHDRS Total Motor Score (TMS) from baseline to the average of the Week 12 and Week 14 assessments
Time Frame
Baseline, Week 12 and Week 14

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: 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. Key Exclusion Criteria: 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 solids, unless medications can be administered via a gastrostomy 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Neurocrine Medical Information Call Center
Phone
877-641-3461
Email
medinfo@neurocrine.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Development Lead
Organizational Affiliation
Neurocrine Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
Neurocrine Clinical Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Stamford
State/Province
Connecticut
ZIP/Postal Code
06905
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Farmington Hills
State/Province
Michigan
ZIP/Postal Code
48334
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-8857
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
http://kinectdcpstudy.com
Description
Study website - Kinect-DCP Study

Learn more about this trial

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

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