Valbenazine for Cerebral Palsy

Recruiting at29 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Neurocrine Biosciences

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

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This trial is testing valbenazine, a medication that may help reduce involuntary movements. It targets children and adults with cerebral palsy who have these movements. The drug works by balancing chemicals in the brain to control movement better. Valbenazine has a longer duration of action and improved side effect profile compared to other similar medications.

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.

How is the drug Valbenazine unique for treating cerebral palsy?

Valbenazine is unique because it is a selective VMAT2 inhibitor, originally approved for treating tardive dyskinesia, a condition involving involuntary movements. Its mechanism of modulating dopamine release may offer a novel approach for managing movement disorders like cerebral palsy, although it is not yet a standard treatment for this condition. ^{1 2 3 4 5}

Research Team

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Eligibility Criteria

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.

Trial Timeline

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

Treatment Details

Interventions

• Valbenazine (Vesicular Monoamine Transporter 2 (VMAT2) Inhibitor)

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.

Participant Groups

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.

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

Findings from Research

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]

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 (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 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 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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Valbenazine (Ingrezza): The First FDA-Approved Treatment for Tardive Dyskinesia. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Efficacy of Valbenazine (NBI-98854) in Treating Subjects with Tardive Dyskinesia and Mood Disorder. [2022]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Valbenazine: First Global Approval. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Valbenazine for Tardive Dyskinesia. [2022]

5. Spain pubmed.ncbi.nlm.nih.gov

Valbenazine for the treatment of tardive dyskinesia. [2017]