RecruitingPHASE3INTERVENTIONAL

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

This study is investigating a new medication called Votoplam for people who have early symptoms of Huntington's disease. The main goals are to find out how safe Votoplam is, if people taking it tolerate it well, and if it can slow down the speed at which Huntington's disease gets worse. To do this, some participants will receive Votoplam, while others will get a placebo (a dummy medicine with no active ingredients). This is a 'blinded' study, meaning neither the participants nor their doctors will know who is receiving which treatment until the study is over. The study involves taking the medicine for up to 3 years and includes regular check-ups.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

770

Start

24 Mar 2026

Estimated completion

30 Apr 2030

Huntington Disease

What is this study about?

Huntington's disease is a condition that affects the brain and can lead to problems with movement, thinking, and mental health. This study is testing a new medicine called Votoplam to see if it can help people who are in the early stages of the disease. Researchers want to understand if Votoplam can make a real difference in slowing down how quickly the disease progresses.

To make sure the results are fair and accurate, the study uses a method called 'blinded' treatment. This means that some participants will receive Votoplam, and others will receive a 'placebo' – which looks exactly like the study medicine but doesn't contain any active ingredients. Neither you nor your study doctor will know which one you are taking until the study has finished. This helps to ensure that any improvements or side effects observed can be directly linked to the actual medicine.

The information gathered from this study will be crucial in deciding if Votoplam could become a new treatment option for people living with Huntington's disease. It's an important step in ongoing research to find better ways to manage this condition.

Key takeaways

This study is testing a new medicine called Votoplam for early Huntington's disease.
It aims to see if Votoplam is safe, well-tolerated, and can slow disease progression.
Participants will receive either Votoplam or a placebo for up to 3 years.
The study includes a screening phase, a treatment phase, and a safety follow-up.
Eligibility is for adults aged 21-70 with genetically confirmed early-symptomatic Huntington's.
Your decision to join is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you need to be an adult between the ages of 21 and 70. You must have a confirmed diagnosis of Huntington's disease through genetic testing, and it should be clear that you are in the earlier stages where you are experiencing symptoms, but are still able to walk. The genetic test also needs to show a specific number (40 or more) in your CAG repeat length.

There are also some reasons why you wouldn't be able to join. For example, if you've had certain brain surgeries for Huntington's, or if you have specific heart problems or certain infections like active hepatitis or HIV, you wouldn't be eligible. Women who could become pregnant are only able to join if they meet certain criteria, such as having had specific surgeries that prevent pregnancy, or if they are past menopause.

Quick self-check

Are you between 21 and 70 years old?
Have you been diagnosed with Huntington's disease through genetic testing?
Do you have a CAG repeat length of 40 or more?
Are you still able to walk?
Are you free from certain heart conditions or active infections like hepatitis or HIV?
If you are a woman of childbearing potential, have you had specific surgeries to prevent pregnancy or are you post-menopausal?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, your first step will be a 'Screening Period' which can last up to 42 days. During this time, doctors will do tests and checks to make sure the study is right for you. If you qualify, you'll then enter the 'Double-blind Treatment Period', where you'll be given either Votoplam or a dummy medicine. You could be on this treatment for up to 3 years, although not everyone will complete the full 3 years.

Throughout the treatment period, you'll have regular visits to the clinic so doctors can monitor your health and see how you're responding to the treatment. After you finish the treatment, there will be one final 'Safety Follow-up' visit or phone call about a month later. This is to check on your health one last time, especially if you're not continuing into a separate, longer-term study.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and potential risks. A potential benefit of this study is that Votoplam might slow down the progression of Huntington's disease, and you would be contributing valuable information to help others in the future. However, there's no guarantee you'll receive the active drug or that it will help you. Potential risks could include side effects from the medication, which would be monitored closely by the study team. You always have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (13)

CenExcel Rocky Mtn Clin Research
Englewood, United States· Recruiting
Georgetown University
Washington D.C., United States· Recruiting
Albany Medical College
Albany, United States· Recruiting
UBMD Neurology
Buffalo, United States· Recruiting
University of Pennsylvania
Philadelphia, United States· Recruiting
TX Movement Disorder Spec PLLC
Georgetown, United States· Recruiting
U of TX Health Science Ct
Houston, United States· Recruiting
University of Washington Medical Center
Seattle, United States· Recruiting
North York General Hospital
North York, Canada· Recruiting
Centre de recherche du CHUM
Montreal, Canada· Recruiting
CUSM Montreal Neurological Institute
Montreal, Canada· Recruiting
Novartis Investigative Site
London, United Kingdom· Recruiting

+1 more sites — see the official record for the full list.

Common questions

What does 'blinded' mean in a study?

It means neither you nor your study doctor will know if you are receiving the real medicine or a dummy medicine (placebo) until the study is over.

How long will I be in the study?

The treatment part of the study could last up to 3 years, but not everyone will stay in for the full period. There's also a screening period before treatment and a follow-up check after.

What is a 'placebo'?

A placebo is a dummy medicine that looks just like the real drug but contains no active ingredients. It helps researchers compare the effects of the active medicine fairly.

Can I leave the study if I change my mind?

Yes, you are free to leave the study at any time, for any reason. Your decision will not affect your usual medical care.

What is Huntington's disease?

Huntington's disease is a condition that affects the brain, causing parts of it to slowly stop working over time. This can lead to problems with movement, thinking, and emotions.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com 1-888-669-6682 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.