Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease

This medical study is looking into a new treatment for early Huntington's disease called AMT-130. It's happening across different hospitals in Europe and is the second phase of testing this medication. Researchers want to find out if AMT-130 is safe and if it helps to improve symptoms or slow down the disease. Some participants will receive either a low or high dose of AMT-130 directly into a specific part of their brain. In a specific group of patients (Cohort 3), some will also be given other medications to help their body accept the treatment. The process will be carefully watched by an expert group to ensure everyone's safety. This study builds on earlier research from the US.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

UniQure Biopharma B.V.

Enrolment target

Start

07 Oct 2021

Estimated completion

07 Oct 2029

Huntington Disease

What is this study about?

This study is exploring a new treatment called AMT-130 for adults who are in the early stages of Huntington's disease. Huntington's disease is a complex condition that affects the brain over time, leading to difficulties with movement, thinking, and mental health. The aim of this research is to see if AMT-130 is safe for people to use and if it can help improve or at least stabilise the symptoms of the disease. This is important because there are currently no treatments that can stop Huntington's disease from progressing.

The treatment, AMT-130, involves a special delivery directly into a part of the brain. The study is divided into different groups, or 'cohorts'. In one group (Cohort 3), some patients will receive a low dose of AMT-130, while others will receive a higher dose. To help the body respond well to the treatment, participants in this group will also be given a course of medicines that help to calm the immune system. This part of the study is designed to learn more about how different doses work and if these 'helper' medicines are useful.

This European study is a continuation of a similar study already happening in the US. By bringing the research to Europe, more people can participate, and scientists can gather more information to better understand the treatment's effects. All participants will be closely monitored for 5 years with various check-ups, brain scans, and tests. An independent safety committee will regularly review all the information to make sure the study is being conducted safely.

Key takeaways

This study is testing a new treatment (AMT-130) for early Huntington's disease.
It aims to check both safety and if the treatment helps with symptoms.
The treatment involves a procedure to deliver medication directly to the brain.
Some participants will also take medications to help their body accept the treatment.
The study involves regular check-ups and monitoring for 5 years.
Participation is only for adults aged 25-65 with early Huntington's disease.

Who may be eligible?

To be considered for this study, you need to be an adult between 25 and 65 years old. You must have been diagnosed with early Huntington's disease, confirmed by specific genetic testing showing a certain number of 'repeats' (40 or more CAG repeats) in your HTT gene. Your medical records must show you're in the early stages of the disease, which is determined by a specific score from your doctor, or because you have mild thinking problems linked to Huntington's.

Your brain scans (MRI) would also need to show that certain parts of your brain are a specific size. Importantly, any other medications you take for your Huntington's symptoms must have been stable for at least 3 months before the study starts, meaning no changes in dose or new medicines. You must also be able and willing to attend all study appointments and follow the study rules. If you are female and could become pregnant, you cannot be pregnant or breastfeeding, and all participants must agree to use effective birth control methods.

Quick self-check

Are you between 25 and 65 years old?
Do you have an official diagnosis of early Huntington's disease?
Has your diagnosis been confirmed by genetic testing (40 or more CAG repeats)?
Have any other Huntington's medications you take been stable for at least 3 months?
Are you able and willing to attend all study appointments for 5 years?
If you could become pregnant, are you using effective birth control and not currently pregnant or breastfeeding?

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 have regular visits over a period of 5 years. These visits will involve various checks, such as blood tests, brain scans (like MRIs), and symptom assessments. For those receiving AMT-130, the treatment involves a procedure where the medication is delivered into your brain. Some participants in Cohort 3 will also take special medications (dexamethasone, sirolimus, and rituximab) before and after the brain procedure to reduce the immune system's response to the treatment.

Some participants will be randomly assigned to receive either a low or high dose of the treatment. For a specific group, neither you nor the doctors will know which dose you're receiving until 3 years into the study. The study involves ongoing monitoring to ensure your safety and to track how the treatment might be affecting your condition, over a total duration of 5 years.

Potential risks and benefits

Participating in research studies like this can have potential benefits. You might gain access to a new treatment that isn't widely available, which could potentially help with your Huntington's disease symptoms. However, there are also potential risks involved. The main treatment involves a surgical procedure on the brain, which carries risks like infection or bleeding, as with any surgery. The medications you receive, including AMT-130 and the immune-suppressing drugs, could have side effects. You will be closely monitored for any problems. It's very important to remember that you are free to leave the study at any time, for any reason, without it affecting your medical care.

Locations (4)

Instytut Psychiatrii i Neurologii
Warsaw, Poland
Interventional Neuro Center
Warsaw, Poland
Cardiff University
Cardiff, United Kingdom
National Hospital for Neurology & Neurosurgery
London, United Kingdom

Common questions

What is AMT-130?

AMT-130 is a new experimental treatment for Huntington's disease that targets the faulty gene to reduce harmful proteins in the brain.

What does 'early manifest' Huntington's disease mean?

This means you have already started to show symptoms of Huntington's disease, but they are still in the early, milder stages.

Will I know if I'm getting the treatment or an inactive substance?

For some parts of the study, you might be randomly assigned to either a low or high dose of the treatment. In one group, neither you nor your doctors will know which dose you receive until 3 years into the study.

Why do some people receive other medications with AMT-130?

These extra medications are called 'immunosuppressants' and are given to help your body accept the new treatment and reduce any strong immune reactions.

How long will I be involved in the study?

If you join the study, you will be followed closely for a total of 5 years.

How to find out more

Official trial record

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