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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05206513




Registration number
NCT05206513
Ethics application status
Date submitted
10/01/2022
Date registered
25/01/2022

Titles & IDs
Public title
Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
Scientific 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
Secondary ID [1] 0 0
EUCT
Secondary ID [2] 0 0
NBI-98854-DCP3018
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dyskinesia 0 0
Cerebral Palsy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Parkinson's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - Valbenazine

Placebo comparator: Placebo - 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.

Experimental: Valbenazine - 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.


Treatment: Drugs: Placebo
Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube

Treatment: Drugs: Valbenazine
Capsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Baseline, Week 12 and Week 14
Secondary outcome [1] 0 0
Change in the Clinical Global Impression of Severity (CGI-S) Score from Baseline to Week 14
Timepoint [1] 0 0
Baseline, Week 14
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Baseline, Week 12 and Week 14
Secondary outcome [3] 0 0
Change in the Total Maximal Dystonia (TMD) Score of the UHDRS from Baseline to the Average of the Week 12 and Week 14 Assessments
Timepoint [3] 0 0
Baseline, Week 12 and Week 14
Secondary outcome [4] 0 0
Patient Global Impression of Improvement (PGI-I) Score at Week 14
Timepoint [4] 0 0
Week 14
Secondary outcome [5] 0 0
Caregiver Global Impression of Improvement (CaGI-I) Score at Week 14
Timepoint [5] 0 0
Week 14
Secondary outcome [6] 0 0
Clinical Global Impression of Improvement (CGI-I) Score at Week 14
Timepoint [6] 0 0
Week 14
Secondary outcome [7] 0 0
Goal Attainment Score at Week 14 Using the Goal Attainment Scale (GAS)
Timepoint [7] 0 0
Week 14
Secondary outcome [8] 0 0
Change in Pain Assessment from Baseline to Week 14 Using the Faces Pain Scale-Revised (FPS-R)
Timepoint [8] 0 0
Baseline, Week 14
Secondary outcome [9] 0 0
Change in the UHDRS Total Motor Score (TMS) from Baseline to the Average of the Week 12 and Week 14 Assessments
Timepoint [9] 0 0
Baseline, Week 12 and Week 14

Eligibility
Key inclusion criteria
Key

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
Minimum age
6 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/04/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2026
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Neurocrine Clinical Site - Clayton
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oklahoma
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
Argentina
State/province [14] 0 0
Buenos Aires
Country [15] 0 0
Argentina
State/province [15] 0 0
Ciudad Autónoma de Buenos Aires
Country [16] 0 0
Brazil
State/province [16] 0 0
Brasília
Country [17] 0 0
Brazil
State/province [17] 0 0
Porto Alegre
Country [18] 0 0
Italy
State/province [18] 0 0
Calambrone
Country [19] 0 0
Poland
State/province [19] 0 0
Gdansk
Country [20] 0 0
Poland
State/province [20] 0 0
Oswiecim
Country [21] 0 0
Portugal
State/province [21] 0 0
Lisboa
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Madrid
Country [24] 0 0
Spain
State/province [24] 0 0
San Sebastián

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Neurocrine Biosciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Development Lead
Address 0 0
Neurocrine Biosciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Neurocrine Medical Information Call Center
Address 0 0
Country 0 0
Phone 0 0
877-641-3461
Fax 0 0
Email 0 0
medinfo@neurocrine.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05206513