AuthorisedHuman Pharmacology (Phase I)- First administration to humansInterventional

A Study to Assess the Safety of RG6496 in Huntington’s Disease Gene Expansion Carriers with the Selected Genetic Variant (POINT-HD)

This research study, called POINT-HD, is investigating a new medicine, RG6496, for individuals who have the genetic change that causes Huntington's disease. Crucially, this is the first time this experimental medicine will be given to people. The main purpose of this initial stage, often called Phase I, is to carefully observe and understand how safe the medicine is in humans. Researchers will be looking to see if there are any unwanted side effects and how the body reacts to the new drug. This type of study is a vital first step in developing new treatments for serious conditions like Huntington's disease, helping scientists decide if it’s safe enough to study further.

At a glance

Status

Authorised

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

F. Hoffmann-La Roche AG

Enrolment target

Start

11 May 2026

Huntington’s Disease Gene Expansion Carriers

What is this study about?

This research is looking into a new medicine called RG6496. It’s for people who carry the genetic fault that can lead to Huntington’s disease. Huntington's is a condition that affects the brain and can cause uncontrolled movements, problems with thinking, and changes in mood.

This particular study is a very early step in testing the new medicine. It's the first time RG6496 will be given to people, so the main aim is to check its safety and how the body handles it. This is really important because before any medicine can be tested to see if it works, we need to be as sure as possible that it won't cause serious harm.

Finding new ways to treat or even prevent conditions like Huntington's disease is a big challenge. Studies like this one are essential for gathering the initial information needed to see if a potential new treatment is promising enough to move on to larger studies in the future.

Key takeaways

This study is testing a new medicine (RG6496) for Huntington's disease.
It's the very first time this medicine is being given to people.
The main aim is to check how safe the medicine is.
Only adults who carry the Huntington's disease gene can take part.
Participation involves regular clinic visits and close monitoring.
You can stop participating at any time without issue.

Who may be eligible?

To be able to take part in this study, you would need to be an adult, aged 18 or older. There is no upper age limit for joining.

Crucially, you would need to be someone who carries the specific gene change that causes Huntington's disease. The researchers are looking for people who have certain genetic characteristics related to this condition.

Both men and women are welcome to take part in this study, provided they meet all the other requirements. The research team will review your medical history and test results to see if you're a good fit for this particular trial.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been told you carry the gene for Huntington's disease?
Are you willing and able to attend regular clinic appointments?
Are you comfortable with receiving an experimental medicine?
Do you want to contribute to medical research?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive the study medicine, RG6496. Since this is the first time it’s being given to humans, you would be closely monitored by the study doctors and nurses. This would involve regular visits to the clinic for health checks, blood tests, and other assessments to make sure you are safe and to see how your body is reacting to the medicine. The total duration of your involvement in the study would be explained in detail by the research team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit could be contributing to the development of a new medicine that might help people with Huntington's disease in the future. However, because this is a first-time-in-human study, the direct benefit to you specifically isn't guaranteed and is unlikely at this early stage. Potential risks include experiencing side effects from the new medicine, which could be mild or, less commonly, more serious. All risks will be fully explained before you agree to take part. Remember, you can decide to stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy
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Unverified
Poland
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Unverified
Denmark
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Unverified
Germany
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Unverified
Portugal
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Unverified
Spain

Common questions

What is Huntington's disease?

Huntington's disease is a serious condition that affects the brain, leading to problems with movement, thinking, and mental health over time.

What is a 'gene expansion carrier'?

This means someone who has the specific genetic change (a 'gene expansion') that is known to cause Huntington's disease.

What does 'Phase I study' mean?

It means this is the very first time the medicine is being tested in people. The main goal is to check for safety and how the body handles it, not yet to see if it works.

Will I definitely get better if I join this study?

No, this study is primarily about checking the safety of a new medicine, not about treating your symptoms at this stage. Any direct benefit to you is not expected.

Can I leave the study if I change my mind?

Yes, absolutely. You can withdraw from the study at any point, and your decision will not affect your ongoing medical care.