RecruitingPHASE1INTERVENTIONAL

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

This research study is looking at a new medicine called RG6496 for people with Huntington's disease. It's the first time this medicine is being tested in humans, so the main goals are to check its safety, how well people tolerate it, and how the body processes it. This involves giving different single doses of the medicine to see these effects. The study has two main parts: an initial phase where single doses are given, followed by an optional longer-term part for those who complete the first phase. The aim is to find out if RG6496 could be a future treatment option for Huntington's disease.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Hoffmann-La Roche

Enrolment target

Start

19 Nov 2025

Estimated completion

23 May 2029

Huntington's Disease

What is this study about?

This study is about a new investigational medicine called RG6496 for Huntington's disease. Huntington's disease is a complex condition that affects the brain and can lead to problems with movement, thinking, and mood over time. Scientists are always working to find new ways to help manage or treat this condition.

This particular study is a very early-stage trial, often called a 'first-in-human' study. This means it's the first time this specific medicine, RG6496, is being given to people. The main purpose at this stage is not to cure the disease, but to gather important information about safety. Researchers want to make sure the medicine is safe enough to be given to people, to understand what side effects it might have, and to see how the body handles it and reacts to it.

By carefully testing RG6496 in this way, scientists hope to learn if it could be a promising candidate for further research. If it proves safe and well-tolerated, and shows some hopeful signs, it could potentially move on to larger studies in the future. This step-by-step process is crucial for developing new treatments.

Key takeaways

This is an early-stage study for a new Huntington's disease medicine.
The main focus is to check the medicine's safety and how the body handles it.
You might receive the new medicine or a placebo at first.
Participation involves regular hospital visits for health checks and scans.
You can stop participating at any time.
It aims to help scientific understanding of Huntington's disease.

Who may be eligible?

To join this study, you would need to be between 25 and 65 years old. You must have a confirmed genetic test result showing you have the genetic change linked to Huntington's disease, and also have a specific genetic marker (SNP) that the researchers are interested in. You should be able to walk without help and weigh at least 40 kg, with your body mass index (BMI) falling within a certain range.

When you join, doctors will assess your overall health and how Huntington's disease is affecting you. You'll need to be able to read some basic words and speak the language used for the study forms and tests. You also need to be able to have MRI scans, which are detailed pictures of your brain.

There are also some reasons why you might not be able to join. For instance, if you are pregnant or breastfeeding, or if you've recently taken part in other clinical trials with experimental medicines. If you have certain serious health conditions like specific types of cancer, HIV, or hepatitis B, or a history of severe mental health issues that caused harm to yourself or others, you would not be able to participate. Also, if you have severe spinal problems that would make a spinal injection difficult, you might not be eligible.

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 between 25 and 65 years old?
Do you have a confirmed genetic diagnosis for Huntington's disease?
Can you walk without assistance?
Are you able to have MRI scans?
Are you able to understand and sign consent forms in the study's language?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will have two main stages. In the first stage, you would receive either the new medicine (RG6496) or a dummy medicine (placebo) in a single dose. This will involve regular visits for checks, blood tests, and MRI scans afterwards to see how your body reacts and to monitor your health. You won't know if you're getting the active medicine or the placebo during this first part of the study.

After completing the first stage and a follow-up period, there's an optional second stage, called an 'open-label extension'. In this part, everyone who joins would receive the new medicine RG6496, and the doctors would continue to monitor your health and how you're responding. The total duration of your participation, including all visits and follow-ups, would be explained to you in detail by the study team, but it's important to understand this is a longer-term commitment to frequent clinical assessments.

Potential risks and benefits

Participating in a study like this means you are helping advance scientific understanding of Huntington's disease, which could benefit others in the future. While RG6496 is being investigated as a potential treatment, there's no guarantee that you will personally benefit from taking it, especially in this early stage of research. Like all medicines, RG6496 may have side effects, and some may be serious. You will be closely monitored for any unwanted effects throughout the study. It's very important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (6)

Hospital Britanico de Buenos Aires
City only
Ciudad Autonoma Bs As, Argentina· Recruiting
Westmead Hospital
Verified postcode
Westmead, Australia· Recruiting
Monash Health
Verified postcode
Clayton, Australia· Suspended
Royal Melbourne Hospital
Verified postcode
Parkville, Australia· Recruiting
New Zealand Brain Research Institute
Verified postcode
Christchurch, New Zealand· Recruiting
Cambridge Centre for Brain Repair
Verified postcode
Cambridge, United Kingdom· Recruiting

Common questions

What is Huntington's disease?

Huntington's disease is a genetic condition that causes nerve cells in the brain to slowly break down. This can affect movement, thinking, and mental health over time.

What is RG6496?

RG6496 is a new medicine being tested. It's hoped it might help people with Huntington's disease, but it's very early in its development.

What is a 'first-in-human' study?

This means it's the first time RG6496 is being given to people. The main goal is to check how safe it is and how the body reacts to it, rather than to see if it cures the disease.

Will I get the actual medicine or a dummy medicine?

In the first part of the study, you might get the new medicine or a dummy medicine (placebo). In an optional second part, everyone would receive the new medicine.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect your ongoing medical care.

How to find out more

Reference Study ID Number: BP45378 https://forpatients.roche.com/

global-roche-genentech-trials@gene.com 888-662-6728 (U.S. Only) Official trial record

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