Valbenazine for Huntington's Disease

Recruiting at 2 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Neurocrine Biosciences

Must be taking: Valbenazine

Disqualifiers: Pregnancy, Breastfeeding, Swallowing difficulty, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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: Long-term

7 Participants Needed

Trial Summary

What is the purpose of this trial?

This study will evaluate long-term safety and tolerability of valbenazine in participants with chorea associated with Huntington Disease (HD) who participated in Study NBI-98854-HD3006 (NCT04400331) in Canada.

Do I need to stop my current medications to join the trial?

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.

What data supports the effectiveness of the drug Valbenazine for Huntington's Disease?

Valbenazine is approved for treating tardive dyskinesia, a movement disorder, and is being studied for its potential to help with chorea (involuntary movements) in Huntington's disease. It is similar to tetrabenazine, which is already used for Huntington's disease, suggesting it might be effective for this condition too. ^{1 2 3 4 5}

Is Valbenazine safe for humans?

Valbenazine, also known as Ingrezza, is approved in the USA for treating tardive dyskinesia and has shown a better short-term side effect profile compared to similar drugs. It is being studied for Huntington's disease, but more long-term data is needed to fully understand its safety. ^{1 2 3 5 6}

How is the drug valbenazine unique for treating Huntington's disease?

Valbenazine is unique because it is a highly selective VMAT2 inhibitor, which helps manage chorea (involuntary movements) in Huntington's disease by modulating dopamine release, and it offers the convenience of once-a-day dosing with a better short-term side effect profile compared to similar treatments like tetrabenazine. ^{1 2 3 5 7}

Research Team

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Eligibility Criteria

This trial is for individuals in Canada with Huntington's Disease who experience chorea (involuntary movements) and were part of a previous study, NBI-98854-HD3006. Specific eligibility details are not provided, but typically participants must meet certain health standards to be included.

Inclusion Criteria

Participants of childbearing potential must agree to use contraception consistently while participating in the study until 30 days after the last dose of the study treatment

Participants who participated in Study NBI-98854-HD3006 and completed dosing through the Week 156 visit

Participants who were actively participating, receiving study treatment, and completed an early termination visit in Study NBI-98854-HD3006 at the time of the study closure at sites in Canada

Exclusion Criteria

Participants who are currently pregnant or breastfeeding

I have trouble swallowing.

I have a significant health issue found through tests or exams.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive valbenazine orally once daily

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Valbenazine

Trial Overview The focus of this study is on the long-term safety and tolerability of a drug called Valbenazine for patients with chorea due to Huntington's Disease. It extends research from an earlier trial.

Participant Groups

Group I: Valbenazine Experimental Treatment 1 Intervention

Capsule, administered orally once daily.

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

Findings from Research

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]

A meta-analysis of randomized controlled trials found that tetrabenazine and deutetrabenazine have similar efficacy and safety profiles in treating Huntington's disease, with no significant differences in motor scores or adverse events.

However, deutetrabenazine showed a significant advantage in reducing depression and somnolence compared to tetrabenazine, suggesting it may be a preferable option for some patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tetrabenazine Versus Deutetrabenazine for Huntington's Disease: Twins or Distant Cousins? Rodrigues, FB., Duarte, GS., Costa, J., et al.[2022]

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]

In a retrospective study of 58 Huntington disease patients treated with deutetrabenazine over an average of 476 days, there was a significant reduction in chorea scores, indicating its effectiveness in managing HD-associated chorea.

The treatment was generally well-tolerated, with a low adverse event rate of 32.8%, and the most common side effects included sedation, insomnia, and diarrhea, supporting its safety profile compared to tetrabenazine.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-World Experience With Deutetrabenazine for Huntington Disease Chorea. Curtis, K., Sung, V.[2023]

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

Valbenazine: First Global Approval. [2018]

2. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of valbenazine for the treatment of chorea associated with Huntington's disease (KINECT-HD): a phase 3, randomised, double-blind, placebo-controlled trial. [2023]

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

Valbenazine for Tardive Dyskinesia. [2022]

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

Tetrabenazine Versus Deutetrabenazine for Huntington's Disease: Twins or Distant Cousins? [2022]

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

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

6. England pubmed.ncbi.nlm.nih.gov

Real-World Experience With Deutetrabenazine for Huntington Disease Chorea. [2023]

7. Spain pubmed.ncbi.nlm.nih.gov

Valbenazine for the treatment of tardive dyskinesia. [2017]