Active not recruitingPHASE2INTERVENTIONAL

An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)

This research study is an extension of an earlier study and aims to understand the long-term safety and how a medicine called Votoplam works in people living with Huntington's disease. If you took part in the first study (study PTC518-CNS-002-HD) and finished the treatment period, you might be able to join this one. All participants in this extended study will receive active Votoplam. If you took Votoplam in the first study, you'll continue with the same dose. If you received a dummy medicine (placebo) before, you'll now be given Votoplam at a specific dose. Doctors will monitor how your body responds to the medicine over an additional 54 months (about 4 and a half years). This helps researchers learn more about Votoplam's effects over a longer period.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

144

Start

25 Aug 2023

Estimated completion

16 Jul 2029

Huntington Disease

What is this study about?

Huntington's disease is a condition that affects the brain and can lead to changes in movement, thinking, and mental health. There isn't a cure yet, so researchers are always looking for new treatments to help manage the symptoms and slow down the disease's progress.

This study is an important next step in understanding a medicine called Votoplam. If you've already been part of a previous study testing Votoplam (called Study PTC518-CNS-002-HD and identified as NCT05358717), and you completed the treatment part of that study, this new study offers a chance to continue receiving Votoplam. The main aim is to see how safe Votoplam is over a much longer period and how it affects the body and the disease.

By continuing to observe participants for an extra 54 months, researchers hope to gather valuable information about the long-term effects of Votoplam. This kind of extended study is crucial for understanding how a new medicine works over time and helps scientists determine if it could be a helpful treatment for people with Huntington's disease in the future.

Key takeaways

This study is an extension of a previous Votoplam trial for Huntington's disease.
It aims to understand the long-term safety and effects of Votoplam.
Only participants who completed the previous study can join.
All participants in this study will receive active Votoplam.
The study involves regular check-ups over about 4.5 years.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you must have already completed the full treatment period of the previous Votoplam study (known as Study PTC518-CNS-002-HD). This means you would have finished all the medicines and check-ups required for that earlier study.

If you did not complete the treatment period in the previous study, unfortunately, you would not be able to join this particular extension study. This study is specifically designed for those who have already participated in and completed the initial research.

Please remember that even if you meet these main criteria, there might be other specific reasons why you could or could not join. Your study doctor can give you all the details and help you understand if this study is right for you.

Quick self-check

Did I complete the treatment part of the previous Votoplam study (PTC518-CNS-002-HD)?
Am I willing to have regular hospital visits and check-ups for about 4.5 years?
Am I comfortable with receiving Votoplam?
Am I at least 25 years old?
Do I have Huntington's Disease?

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

What does participation involve?

If you join this study, you will continue to receive the study medicine, Votoplam, over an extra 54 months. If you received Votoplam in the previous study, you'll continue with the same dose. If you received a dummy medicine (placebo) before, you will now be given Votoplam at a specific dose, which will be decided for you. You won't know which exact dose of Votoplam you are on, and neither will your study team, so that the results can be as fair as possible.

Over these 54 months, you will have regular visits with the study team. During these visits, doctors will perform different assessments to check your health, how you are responding to the medicine, and to see if the medicine is affecting your body in any way. These assessments might include physical exams, blood tests, and other specific tests related to Huntington's disease.

The main aim is to carefully track your progress and look at the long-term safety and effects of Votoplam over approximately four and a half years.

Potential risks and benefits

Taking part in this study could offer several potential benefits, such as continued access to Votoplam, a medicine being investigated for Huntington's disease. You will also receive close medical attention and monitoring from a dedicated research team. However, as with any medicine, there are potential risks and side effects that Votoplam might cause, which you will be fully informed about. Some tests you have during the study might also have minor risks, such as discomfort from blood draws. It's important to remember that your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (24)

Novartis Investigative Site
Clayton, Australia
Novartis Investigative Site
Westmead, Australia
Novartis Investigative Site
Innsbruck, Austria
Novartis Investigative Site
Ottawa, Canada
Novartis Investigative Site
Angers, France
Novartis Investigative Site
Marseille, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Berlin, Germany
Novartis Investigative Site
Bochum, Germany
Novartis Investigative Site
Münster, Germany
Novartis Investigative Site
Ulm, Germany
Novartis Investigative Site
Bologna, Italy

+12 more sites — see the official record for the full list.

Common questions

What is the main goal of this study?

The main goal is to understand how safe a medicine called Votoplam is and how it affects people with Huntington's disease over a long period (about 4.5 years).

Who can join this study?

Only people who have already completed the treatment part of a previous Votoplam study (Study PTC518-CNS-002-HD) can join this extension study.

Will I know if I'm getting the actual medicine?

Yes, everyone in this extension study will receive active Votoplam. However, you and your study team usually won't know which specific dose you are receiving to keep the study fair.

How long will I be in this study?

You'll be part of this study for an additional 54 months, which is about four and a half years.

Can I leave the study if I change my mind?

Yes, you are completely free to leave the study at any time, for any reason, and it will not affect your regular medical care.