Valbenazine for Huntington's Disease

Not currently recruiting at 33 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests valbenazine to determine its safety and tolerability over an extended period for treating chorea, the uncontrolled movements often seen in individuals with Huntington's disease. Participants must have either been in a previous related study or have a confirmed diagnosis of Huntington's disease with chorea and be able to walk. Those unable to swallow pills or with certain heart problems might not qualify. The trial aims to improve chorea symptom management while ensuring the treatment's safety for ongoing use. As a Phase 3 trial, it represents the final step before FDA approval, offering patients an opportunity to contribute to the potential availability of a new treatment.

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

Research has shown that valbenazine is generally safe for people with Huntington's disease. One study demonstrated that valbenazine reduced chorea symptoms, the jerky, involuntary movements common in this condition. The treatment proved as safe as a placebo, causing no more side effects than a sugar pill.

Further studies over three years confirmed that valbenazine's side effects aligned with doctors' expectations. The most common side effects were mild and did not prevent most people from continuing the treatment.

Overall, the evidence suggests that valbenazine is safe for treating chorea in Huntington's disease. ^{1 2 3 4 5}

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

Valbenazine is unique because it offers a new approach to treating Huntington's Disease by specifically targeting and inhibiting VMAT2, a protein involved in dopamine regulation. Unlike other treatments that primarily focus on managing symptoms, such as tetrabenazine and deutetrabenazine, Valbenazine's mechanism may provide a more direct way to manage the involuntary movements associated with the disease. Researchers are excited about Valbenazine because it is administered once daily in a capsule form, which could improve patient compliance and quality of life compared to more complex treatment regimens.

What evidence suggests that valbenazine might be an effective treatment for chorea in Huntington's disease?

Research has shown that valbenazine, the treatment under study in this trial, helps reduce involuntary movements, known as chorea, in people with Huntington's disease. One study found that patients taking valbenazine experienced noticeable improvements in their symptoms compared to those taking a placebo, which contains no active ingredients. The treatment was generally well tolerated, with few bothersome side effects reported. Another study examined valbenazine over an extended period and confirmed its benefits up to Week 156. These findings suggest that valbenazine is a promising option for managing chorea in Huntington's disease. ^{3 6 7 8 9}

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 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]

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]

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. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-presents-new-three-year-data

Release Details - Neurocrine Biosciences Efficacy was evaluated through Week 156 using changes from baseline in the Unified Huntington's Disease Rating Scale (UHDRS®) Total Maximal ...

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

Efficacy in adults with Huntington's disease chorea Find clinical trial results of INGREZZA to reduce chorea symptoms in adult patients with Huntington's disease. See Prescribing Info, including Boxed ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1474442223001278

Articles Safety and efficacy of valbenazine for the treatment ... In individuals with Huntington's disease, valbenazine resulted in improvement in chorea compared with placebo and was well tolerated.

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. 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.

6. 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 ... INGREZZA was generally well tolerated over the long term, and TEAEs observed in the study were consistent with its established safety profile ...

7. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-presents-new-post-hoc-data-analyses

Neurocrine Biosciences Presents New Post-Hoc Data ... In the 12-week Phase 3 KINECT-HD clinical trial, INGREZZA demonstrated a significant reduction in chorea severity, as measured by the Unified Huntington's ...

8. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT04102579?recrs=a&cond=Neurodegenerative+Diseases&phase=2&prcd_s=04/01/2021&prcd_e=11/01/2021&draw=1&rank=2

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

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

HD Chorea Safety | INGREZZA® (valbenazine) capsules View the safety profile including adverse reactions for INGREZZA in adults with Huntington's disease chorea. See Prescribing Info, including Boxed Warning.