Valbenazine for Huntington's Disease

Recruiting at 32 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Neurocrine Biosciences

Must not be taking: VMAT2 inhibitors

Disqualifiers: Pregnancy, Heart disease, Substance abuse, 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: 156 weeks

154 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing the safety of valbenazine, a medication that helps control involuntary movements in patients with Huntington disease who suffer from chorea. Valbenazine works by balancing brain chemicals to reduce these unwanted movements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you cannot have used a VMAT2 inhibitor within 30 days before starting the trial.

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. However, long-term safety data is still being gathered, and its full safety profile will be clearer with more real-world use. ^{1 2 3 4 5}

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

Valbenazine is unique because it is a highly selective VMAT2 inhibitor, originally approved for tardive dyskinesia, and is now being evaluated for its potential to treat chorea (involuntary movements) in Huntington's disease, offering a new option where few exist. ^{1 3 4 5 6}

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

Valbenazine is a drug that has been 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 also be effective for this condition. ^{1 3 4 5 7}

Who Is on the Research Team?

Chief Medical Officer

Principal Investigator

Chief Medical Officer

Are You a Good Fit for This Trial?

This trial is for individuals with Huntington's Disease and chorea who either completed a prior study (NBI-98854-HD3005) or have not used VMAT2 inhibitors in the last 30 days. Participants must be able to walk, understand English, consent to the study, and agree to use contraception if of childbearing potential.

Inclusion Criteria

Did not participate in Study NBI-98854-HD3005.

I can walk, even if I need help or a device.

I meet at least one of the required conditions for inclusion.

Exclusion Criteria

I have lost more than 550 mL of blood or donated blood in the last 30 days.

I have a history of heart rhythm problems or heart failure.

I haven't taken any experimental drugs (except valbenazine) in the last 30 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive valbenazine for the treatment of chorea associated with Huntington disease

156 weeks

Extended Maintenance

Participants in the US have the option to continue valbenazine administration for up to 104 additional weeks

104 weeks

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 trial is testing Valbenazine's long-term safety and effectiveness in treating chorea associated with Huntington’s Disease. It's an open-label Phase 3 study providing ongoing access to Valbenazine for those who need it.

How Is the Trial Designed?

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

Huntington Study Group

Collaborator

Trials

15

Recruited

9,400+

Published Research Related to This Trial

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]

Citations

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]