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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Neurocrine Biosciences Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

1 Mar 2024 → 31 Mar 2026

Decision date (initial)

2023-11-06

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Neurocrine Biosciences, Inc.

External identifiers

EU CT number

2022-502713-29-00

ClinicalTrials.gov

NCT05206513

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Therapy, Others, Efficacy

To evaluate the efficacy of valbenazine versus placebo on improving chorea in pediatric and adult subjects who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.

Secondary objectives 2

1. To evaluate the efficacy of valbenazine versus placebo on improving dyskinesia severity, functioning, pain, and quality of life in pediatric and adult subjects who have DCP with choreiform movements.
2. To evaluate the safety and tolerability of valbenazine administered once daily in pediatric and adult subjects who have DCP with choreiform movements (including incidence of treatment-emergent adverse events [TEAEs] and treatment-emergent serious adverse events [TESAEs]).

Conditions and MedDRA coding

Dyskinesia due to cerebral palsy (DCP)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

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.

Exclusion criteria 6

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 behaviour 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The 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 endpoints 9

1. The change in Clinical Global Impression of Severity (CGI-S) score from baseline to Week 14
2. The change in Movement Disorders - Childhood Rating Scale (MD-CRS) Part I score from baseline to the average of the Week 12 and Week 14 assessments
3. The change in the Total Maximal Dystonia (TMD) score of the UHDRS from baseline to the average of the Week 12 and Week 14 assessment
4. Patient Global Impression of Improvement (PGI-I) score at Week 14 [ Time Frame: Week 14 ]
5. Caregiver Global Impression of Improvement (CaGI-I) score at Week 14 [ Time Frame: Week 14 ]
6. Clinical Global Impression of Improvement (CGI-I) score at Week 14 [ Time Frame: Week 14 ]
7. Goal attainment score at Week 14 using the Goal Attainment Scale (GAS) [ Time Frame: Week 14 ]
8. Change in pain assessment from baseline to Week 14 using the Faces Pain Scale-Revised (FPS-R) [ Time Frame: Baseline, Week 14 ]
9. 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 ]

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 6

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Neurocrine Biosciences Inc.

Sponsor organisation

Neurocrine Biosciences Inc.

Address

6027 Edgewood Bend Court

City

San Diego

Postcode

92130-8235

Country

United States

Scientific contact point

Organisation

Neurocrine Biosciences Inc.

Contact name

Neurocrine Medical Information Call Center

Public contact point

Organisation

Neurocrine Biosciences Inc.

Contact name

Neurocrine Medical Information Call Center

Third parties 9

Locations

5 EU/EEA countries · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 175 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

10 submissions — initial application plus substantial / non-substantial modifications