154 Participants Needed

Valbenazine for Huntington's Disease

Recruiting at 32 trial locations
NM
Overseen ByNeurocrine Medical Information Call Center
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Neurocrine Biosciences
No Placebo GroupAll 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 DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

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.

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.12345

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.12356

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.12357

Research Team

CM

Chief Medical Officer

Principal Investigator

Chief Medical Officer

Eligibility Criteria

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.
See 1 more

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.
See 10 more

Timeline

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

Treatment Details

Interventions

  • Valbenazine
Trial OverviewThe 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.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: ValbenazineExperimental Treatment1 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

Find a Clinic Near You

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+

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

References

Valbenazine: First Global Approval. [2018]
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]
Valbenazine for Tardive Dyskinesia. [2022]
Tetrabenazine Versus Deutetrabenazine for Huntington's Disease: Twins or Distant Cousins? [2022]
Valbenazine (Ingrezza): The First FDA-Approved Treatment for Tardive Dyskinesia. [2020]
Real-World Experience With Deutetrabenazine for Huntington Disease Chorea. [2023]
Valbenazine for the treatment of tardive dyskinesia. [2017]