Valbenazine for Cerebral Palsy

Not currently recruiting at 43 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Neurocrine Biosciences

Disqualifiers: Pregnancy, Dyskinesia from other conditions, Suicidal behavior, Substance abuse, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 14 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called valbenazine to determine its effectiveness for people with cerebral palsy who experience involuntary, dance-like movements known as chorea. Participants will receive either valbenazine or a placebo (a pill with no active drug) for 14 weeks. Afterward, everyone can choose to try valbenazine for an extended period. Suitable candidates for this trial have a confirmed diagnosis of cerebral palsy with chorea and stable health conditions. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to the development of a promising treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that valbenazine is likely to be safe for humans?

Research has shown that valbenazine is generally safe for people. The FDA has already approved it for treating tardive dyskinesia, a movement disorder, indicating a certain level of safety. In other studies, some participants experienced mild side effects like sleepiness and dry mouth, which were not serious for most people. The current trial phase tests valbenazine for movement issues caused by cerebral palsy, suggesting it has shown promising safety results in earlier research. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cerebral palsy?

Unlike the standard treatments for cerebral palsy, which often focus on managing symptoms through physical therapy, muscle relaxants, or surgeries, Valbenazine offers a unique approach. Valbenazine works by specifically targeting and modulating dopamine receptors, which are involved in involuntary movements and muscle control. Researchers are excited because this mechanism could potentially provide more precise symptom control and improve quality of life for individuals with cerebral palsy, offering a new avenue for treatment beyond traditional methods.

What evidence suggests that valbenazine might be an effective treatment for cerebral palsy?

Research shows that valbenazine may help treat movement disorders. In earlier studies, individuals with tardive dyskinesia (TD), a condition causing involuntary movements, experienced significant symptom relief after taking valbenazine. Participants reported better control over their movements. This trial will administer valbenazine to some participants to evaluate its effectiveness in managing dyskinesia caused by cerebral palsy. Although this trial focuses on dyskinesia from cerebral palsy, the positive results seen in TD suggest that valbenazine might also help manage involuntary movements, known as chorea, in cerebral palsy. This hope stems from its success in reducing similar movement issues in other conditions. ^{1 3 4 5 6}

Who Is on the Research Team?

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Are You a Good Fit for This Trial?

This trial is for people aged 6-70 with dyskinesia due to cerebral palsy, specifically those with choreiform movements. Participants should have stable medical conditions and be able to swallow soft solids or take medication via a gastrostomy tube. It's not for pregnant/breastfeeding individuals, those with other causes of dyskinesia, recent suicidal behavior/ideation, substance abuse, or certain heart conditions.

Inclusion Criteria

My health conditions are stable and expected to stay that way during the study.

I have been diagnosed with a movement disorder due to cerebral palsy.

Exclusion Criteria

I cannot swallow soft solids but can take medications through a tube.

I have a history of heart rhythm problems or abnormal ECG results.

You have had thoughts of hurting yourself or have tried to hurt yourself in the past year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either valbenazine or placebo for 14 weeks during the double-blind treatment period

14 weeks

Open-label extension

Participants may opt into continuation of valbenazine treatment long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Valbenazine

Trial Overview The study is testing the effectiveness of Valbenazine compared to a placebo in improving involuntary movement (chorea) in participants with dyskinesia from cerebral palsy. The goal is to see if Valbenazine can help control these movements better than no treatment.

How Is the Trial Designed?

Group I: Valbenazine Experimental Treatment 1 Intervention

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.

Group II: Placebo Placebo Group 2 Interventions

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 is already approved in United States for the following indications:

Approved in United States as Ingrezza for:

• Tardive dyskinesia
• Chorea associated with Huntington's disease

Who Is Running the Clinical Trial?

Neurocrine Biosciences

Lead Sponsor

Trials

78

Recruited

6,600+

Kyle W. Gano

Neurocrine Biosciences

Chief Executive Officer since 2024

PhD in Pharmacology

Dr. Sanjay Keswani

Neurocrine Biosciences

Chief Medical Officer

MD

Published Research Related to This Trial

Valbenazine is the first FDA-approved medication specifically for treating tardive dyskinesia (TD), showing significant improvement in symptoms at a dose of 80 mg/day with a clinically meaningful effect size of 0.90 in short-term trials.

Compared to tetrabenazine, valbenazine offers advantages such as once-a-day dosing and a better short-term side effect profile, although further long-term studies are needed to fully understand its benefits and risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Valbenazine for Tardive Dyskinesia. Freudenreich, O., Remington, G.[2022]

Valbenazine (Ingrezza™) is an approved treatment for tardive dyskinesia (TD) in adults, demonstrating its efficacy as a selective VMAT2 inhibitor for managing this condition.

The drug is also being explored for Tourette syndrome in phase 2 trials, indicating its potential for treating various central nervous system disorders beyond TD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Valbenazine: First Global Approval. Kim, ES.[2018]

Valbenazine (VBZ) significantly improved tardive dyskinesia (TD) symptoms in subjects with mood disorders over a 48-week treatment period, with greater improvements observed at higher doses compared to placebo, indicating its efficacy as a treatment option.

After stopping VBZ treatment, TD symptoms began to return toward baseline levels, suggesting that while VBZ is effective in managing TD, ongoing treatment may be necessary to maintain symptom control.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of Valbenazine (NBI-98854) in Treating Subjects with Tardive Dyskinesia and Mood Disorder. Correll, CU., Josiassen, RC., Liang, GS., et al.[2022]

Valbenazine is a selective VMAT2 inhibitor that effectively modulates dopamine release, showing significant improvement in symptoms of tardive dyskinesia (TD) in clinical trials.

As there are currently no FDA-approved treatments for TD, valbenazine represents a promising new option, with a new drug application submitted for priority review in 2016.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Valbenazine for the treatment of tardive dyskinesia. Barquero, N.[2017]

Valbenazine (Ingrezza) is the first medication approved by the FDA specifically for treating tardive dyskinesia, a condition characterized by involuntary movements often caused by long-term use of antipsychotic medications.

This approval marks a significant advancement in the management of tardive dyskinesia, providing patients with a targeted treatment option to help alleviate their symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Valbenazine (Ingrezza): The First FDA-Approved Treatment for Tardive Dyskinesia. Uhlyar, S., Rey, JA.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05206513

NCT05206513 | Study to Assess the Efficacy, Safety, and ... 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 ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05206513?term=Valbenazine&aggFilters=phase:3,status:not%20rec&rank=1

Study to Assess the Efficacy, Safety, and Tolerability of ... The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure.

3. kennedykrieger.org kennedykrieger.org/research/participate-in-research/cerebral-palsy-studies/valbenazine-for-the-treatment-of-dyskinesia-due-to-cerebral-palsy-neurocrine

Valbenazine for the Treatment of Dyskinesia Due to ... A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia ...

4. ingrezzahcp.com ingrezzahcp.com/efficacy/patient-reported-outcomes

Patient-reported outcomes in tardive dyskinesia Learn about the KINECT-PRO study design and patient reported outcomes with TD Impact Scale. See Prescribing Info, including Boxed Warning.

5. withpower.com withpower.com/trial/phase-3-dyskinesias-2022-aa688

Valbenazine for Cerebral Palsy · Info for Participants Valbenazine (VBZ) significantly improved tardive dyskinesia (TD) symptoms in subjects with mood disorders over a 48-week treatment period, with greater ...

6. nicklauschildrens.org nicklauschildrens.org/medical-services/research-institute/open-clinical-trials/neu99201-(nbi-98854-dcp3018)

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