Valbenazine for Huntington's Disease

Enrolling by invitation 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called valbenazine to determine its safety and effectiveness for people with chorea, a movement disorder associated with Huntington's Disease. The focus is on the drug's long-term use for those who participated in a previous study in Canada. To join, participants must have been in the earlier study and completed certain visits, but should not have difficulty swallowing or be pregnant. As a Phase 3 trial, this is the final step before FDA approval, providing an opportunity to contribute to the potential availability of a new treatment.

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.

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

Research shows that valbenazine is generally safe and well-tolerated for treating involuntary movements (chorea) in people with Huntington's disease. Earlier studies confirmed that the safety of this treatment aligned with previous knowledge. Tested in a controlled setting, any side effects matched expectations. Valbenazine also has approval for treating tardive dyskinesia, further supporting its safety. Overall, evidence suggests that valbenazine is safe for most people, with manageable side effects consistent with past findings. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Huntington's Disease?

Valbenazine is unique for treating Huntington's Disease because it specifically targets and inhibits VMAT2 (vesicular monoamine transporter 2), which regulates dopamine levels in the brain. Most treatments for Huntington's focus on managing symptoms like movement disorders and psychiatric issues but don't directly address dopamine dysregulation. Researchers are excited because Valbenazine's mechanism could lead to more precise control over the symptoms, potentially improving quality of life for patients. Plus, its once-daily oral administration makes it a convenient option compared to other therapies that might require more frequent dosing or invasive procedures.

What evidence suggests that valbenazine might be an effective treatment for Huntington's Disease?

Research has shown that valbenazine, which participants in this trial will receive, can greatly reduce movement problems, known as chorea, in people with Huntington's disease. One study found that people taking valbenazine showed noticeable improvements in these movements compared to those who took a placebo, a pill with no active medicine. The medication was generally well tolerated, with most people not experiencing serious side effects. Another study indicated that valbenazine might also help improve emotional well-being and mental stability in patients with Huntington's disease. Overall, these findings suggest that valbenazine could effectively manage movement issues in Huntington's disease. ^{1 6 7 8 9}

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 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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

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

Published Research Related to This Trial

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 (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 (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 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 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]

Citations

1. prnewswire.com prnewswire.com/news-releases/neurocrine-biosciences-presents-new-three-year-data-demonstrating-sustained-clinical-benefits-and-established-safety-profile-of-ingrezza-valbenazine-capsules-for-huntingtons-disease-chorea-302575129.html

Neurocrine Biosciences Presents New Three-Year Data ... Once-daily INGREZZA treatment was generally well tolerated and showed early and sustained improvements in chorea severity ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37210099/

a phase 3, randomised, double-blind, placebo-controlled trial In individuals with Huntington's disease, valbenazine resulted in improvement in chorea compared with placebo and was well tolerated.

3. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-presents-new-kinectr-hd-data-showing

Neurocrine Biosciences Presents New KINECT®-HD Data ... Effects of Valbenazine on Emotional Health and Psychiatric Stability in Adults with Huntington's Disease. This separate post hoc analysis of ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04102579

Study Details | NCT04102579 | Efficacy, Safety, and ... This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of valbenazine to treat chorea in ...

5. ingrezzahcp.com ingrezzahcp.com/efficacy/huntingtons-disease-chorea

HD Chorea Efficacy | INGREZZA® (valbenazine) capsules Find clinical trial results of INGREZZA to reduce chorea symptoms in adult patients with Huntington's disease. See Prescribing Info, including Boxed ...

6. ingrezzahcp.com ingrezzahcp.com/safety/huntingtons-disease-chorea

HD Chorea Safety | INGREZZA® (valbenazine) capsules Safety profile in Huntington's disease chorea. In KINECT-HD, INGREZZA was generally well tolerated. Adverse reactions in the placebo-controlled study of 12 ...

7. accp1.onlinelibrary.wiley.com accp1.onlinelibrary.wiley.com/doi/full/10.1002/jcph.70092

Population Pharmacokinetic and Exposure‐Efficacy Analyses ... Valbenazine improved chorea in individuals with Huntington's disease compared to placebo in a phase 3, placebo-controlled study (KINECT-HD).

8. thelancet.com thelancet.com/journals/laneur/article/PIIS1474-4422(23)00127-8/abstract

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

9. neurologylive.com neurologylive.com/view/valbenazine-linked-to-better-physical-emotional-well-being-tardive-dyskinesia

Valbenazine Linked to Better Physical and Emotional Well ... Valbenazine, originally approved in 2017 for patients with TD, had safety findings that were consistent with known safety and tolerability ...