A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
This research study is looking into a new treatment called ALN-HTT02 for adults with Huntington's disease. It's a very early-stage study, meaning it's one of the first times this medicine is being tested in people. The main goals are to check if the medicine is safe, how well people can tolerate it, and how it behaves in the body after single or repeated doses. Some people in the study will receive the new medicine, while others will get a placebo (a treatment that looks the same but contains no active medicine). Participants will be adults aged 25 to 70 with a specific stage of Huntington's disease. This type of study helps scientists understand if a new medicine has the potential to help patients in the future.
At a glance
What is this study about?
This study is a very important first step in understanding a new potential treatment for Huntington's disease called ALN-HTT02. Think of it like a journey: this is the initial exploration phase to make sure the path is safe before we can fully map it out. Researchers want to carefully check a few key things: Is ALN-HTT02 safe for people to take? How well do people tolerate it (meaning, what side effects might there be and how severe are they)? And how does the medicine move through and act in the body? This is known as a Phase 1 study, which is typically the first time a new medicine is given to humans.
In this study, some participants will receive the active medicine, ALN-HTT02, while others will receive something called a placebo. A placebo looks exactly like the real medicine but doesn't contain any active ingredients. This is a standard and ethical way to compare the effects of the new medicine against what happens when no active treatment is given. It helps researchers accurately understand if any changes observed are truly due to the new medicine or if they might happen anyway.
The overall aim is to gather crucial information about ALN-HTT02. If it proves to be safe and well-tolerated, and if researchers learn enough about how it works, it may then move on to larger studies to see if it can effectively improve symptoms or slow the progression of Huntington's disease.
Key takeaways
- This is an early-stage study testing a new medicine for Huntington's disease.
- It aims to check the safety and how the medicine works in the body.
- Some participants will receive the new medicine, others a dummy treatment (placebo).
- Suitable for adults aged 25-70 with specific stages of Huntington's disease.
- Your health will be closely monitored throughout the study.
- You can stop participating at any time if you wish.
Who may be eligible?
To be considered for this study, you would need to be an adult between 25 and 70 years old. You must also have been diagnosed with Huntington's disease that is at a specific stage, either stage 2 or early stage 3, according to a system doctors use to classify the disease.
There are also some reasons why you might not be able to join the study. For example, if you have other serious brain conditions apart from Huntington's disease, or if your immune system is weakened due to an illness or other treatments. The study also has certain requirements regarding your liver and kidney function, so if those organs aren't working within specific limits, you may not be eligible.
Finally, if you've recently taken part in another clinical trial where you received an experimental medicine (within the last year), you might not be able to join this one. These rules are in place to help keep you safe and ensure the study results are as clear and accurate as possible.
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 70 years old?
- Do you have Huntington's disease, specifically stage 2 or early stage 3?
- Do you have any other serious brain conditions?
- Is your immune system generally healthy?
- Have you participated in another experimental drug trial in the last year?
What does participation involve?
If you decide to take part in this study, you would likely need to visit the study clinic regularly. These visits will involve various check-ups, which could include physical exams, blood and urine tests, and possibly other tests related to your Huntington's disease symptoms. You would receive either the new medicine, ALN-HTT02, or a placebo, which you would take as instructed by the study team. The total time you'd spend in the study, including all visits and follow-up, would be fully explained before you agree to participate.
Potential risks and benefits
Locations (19)
- Clinical Trial SiteVerified postcodeEdmonton, Canada· Recruiting
- Clinical Trial SiteVerified postcodeMontreal, Canada· Recruiting
- Clinical Trial SiteVerified postcodeOttawa, Canada· Recruiting
- Clinical Trial SiteVerified postcodeVancouver, Canada· Recruiting
- Clinical Trial SiteVerified postcodeBerlin, Germany· Recruiting
- Clinical Trial SiteVerified postcodeBochum, Germany· Recruiting
- Clinical Trial SiteVerified postcodeBonn, Germany· Recruiting
- Clinical Trial SiteVerified postcodeDresden, Germany· Recruiting
- Clinical Trial SiteVerified postcodeMünster, Germany· Recruiting
- Clinical Trial SiteVerified postcodeTaufkirchen, Germany· Recruiting
- Clinical Trial SiteVerified postcodeUlm, Germany· Recruiting
- Clinical Trial SiteVerified postcodeBirmingham, United Kingdom· Recruiting
Common questions
What is a Phase 1 study?
A Phase 1 study is the first step in testing a new medicine in people. It mainly checks if the medicine is safe and how the body handles it.
What is a placebo?
A placebo is a substance that looks exactly like the study medicine but doesn't contain any active ingredients. It helps researchers compare the real medicine's effects.
Will I know if I'm getting the real medicine or the placebo?
Often, in studies like this, neither you nor your study doctor will know who is receiving the active medicine and who is receiving the placebo. This helps keep the study fair.
What is Huntington's Disease Integrated Staging System (HD-ISS)?
It's a way doctors classify the stage of Huntington's disease. For this study, you need to be at stage 2 or early stage 3.
Can I stop participating in the study if I change my mind?
Yes, you have the right to leave the study at any time, for any reason, without it affecting your usual medical care.
How to find out more
Alnylam Clinical Trial Information Line
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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