Study on Valbenazine for Treating Dyskinesia in Children and Adults with Cerebral Palsy

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What is this study about?

This clinical trial is focused on studying a condition known as dyskinesia due to cerebral palsy. Dyskinesia refers to involuntary movements that can occur in people with cerebral palsy, a group of disorders that affect movement and muscle tone. The trial will test a treatment called Valbenazine, which is being evaluated for its ability to improve these involuntary movements. Valbenazine is taken orally in the form of a hard capsule.

The purpose of the study is to assess how effective and safe Valbenazine is in treating dyskinesia in both children and adults with cerebral palsy. Participants in the study will be randomly assigned to receive either Valbenazine or a matching placebo, which looks like the medication but does not contain the active ingredient. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo, to ensure unbiased results.

Throughout the study, participants will take the medication or placebo for a set period, and their progress will be monitored by healthcare professionals. The study aims to observe changes in the severity of involuntary movements and overall improvement in the condition. The trial will also evaluate the safety and tolerability of Valbenazine, ensuring that it is safe for use in treating dyskinesia due to cerebral palsy.

1 joining the study

Upon joining the study, the patient will be assessed to confirm a diagnosis of dyskinesia due to cerebral palsy, characterized by involuntary movements.

The patient’s medical condition must be stable and expected to remain stable throughout the study.

2 medication administration

The patient will receive either valbenazine or a matching placebo. The medication is administered orally in the form of hard capsules.

The dosage and frequency of administration will be determined by the study protocol and communicated to the patient.

3 primary assessment

The primary goal is to evaluate the change in the Total Maximal Chorea (TMC) score from the baseline to the average of the Week 12 and Week 14 assessments.

This assessment helps determine the effectiveness of the treatment in reducing involuntary movements.

4 secondary assessments

Several secondary assessments will be conducted, including changes in the Clinical Global Impression of Severity (CGI-S) score and the Movement Disorders – Childhood Rating Scale (MD-CRS) Part I score.

Other assessments include the Total Maximal Dystonia (TMD) score, Patient Global Impression of Improvement (PGI-I) score, and Caregiver Global Impression of Improvement (CaGI-I) score at Week 14.

Additional evaluations involve the Clinical Global Impression of Improvement (CGI-I) score, Goal Attainment Scale (GAS) score, and changes in pain assessment using the Faces Pain Scale-Revised (FPS-R).

The UHDRS Total Motor Score (TMS) will also be assessed from baseline to the average of the Week 12 and Week 14 assessments.

5 study duration

The study is expected to conclude by January 16, 2026.

The recruitment for the study began on October 2, 2023.

Who Can Join the Study?

The patient must have a medically confirmed diagnosis of DCP, which stands for dyskinesia due to cerebral palsy. This means they have a movement disorder caused by cerebral palsy, with movements that are jerky or dance-like, known as choreiform movements.
The patient’s medical conditions should be stable, meaning they are not expected to change or get worse during the study.
The patient can be of any gender, as both males and females are eligible to participate.
The patient must be within the age range of 4 to 17 years old, as the study includes children and teenagers.

Who Cannot Join the Study?

Patients who do not have dyskinesia due to cerebral palsy. Dyskinesia refers to involuntary movements, and cerebral palsy is a condition that affects movement and muscle tone.
Patients who do not experience choreiform movements. These are jerky, involuntary movements that can affect different parts of the body.
Patients who are not within the specified age range for the study. The study includes certain age groups, so participants must fall within these ranges.
Patients who are not part of the specified clinical trial groups. The study is designed for specific groups, and participants must belong to these groups.
Patients who are not male or female, as the study includes both genders.
Patients who are not considered part of a vulnerable population. This means the study is open to individuals who may have additional needs or considerations.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Unidade Local De Saude De Sao Jose E.P.E.	Lisbon	Portugal
ASST Grande Ospedale Metropolitano Niguarda	Milan	Italy
Specjalistyczne Gabinety Sp. z o.o.	Cracow	Poland
Centro Hospitalar Universitario De Santo Antonio E.P.E.	Porto	Portugal
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Policlinica Gipuzkoa S.A.	Donostia / San Sebastian	Spain
Stowarzyszenie Na Rzecz Osob Niepelnosprawnych Krok Po Kroku	Gdansk	Poland
IRCCS Fondazione Stella Maris	Pisa	Italy
Fsxvibyk Aiboig Cuaonjyjc	Barcelona	Spain
Ccacklyqa Ugkejvuqyleslb Saoqwaqox	Woluwe-Saint-Lambert	Belgium
Hsjopzgr Uieqpvqlakown Dz Lw Plgunqkv	Madrid	Spain
Iqukrrey Zyufplr Dy Boopxctlvvnahdyzi	Oświęcim	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	02.10.2023
Italy	Not recruiting	02.10.2023
Poland	Not recruiting	02.10.2023
Portugal	Not recruiting	02.10.2023
Spain	Not recruiting	02.10.2023

Trial locations

Investigated drugs:

Valbenazine Ditosylate

Valbenazine is a medication being studied for its ability to help reduce involuntary movements, known as dyskinesia, in people with cerebral palsy. This trial is focused on understanding how well valbenazine works in improving these movements and how safe and tolerable it is for both children and adults with this condition.

Investigated diseases:

Dyskinesia due to cerebral palsy (DCP) – This condition involves involuntary, erratic movements that can affect various parts of the body. It is associated with cerebral palsy, a group of disorders that affect movement and muscle tone. In DCP, the movements are often described as choreiform, meaning they are dance-like and unpredictable. These movements can interfere with daily activities and may vary in intensity. The condition is chronic and typically begins in childhood, persisting into adulthood. The severity and progression of symptoms can differ widely among individuals.

Trial ID:

2022-502713-29-00

Protocol code:

NBI-98854-DCP3018

NCT ID:

NCT05206513

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Valbenazine for Treating Dyskinesia in Children and Adults with Cerebral Palsy

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