RecruitingPHASE1INTERVENTIONAL

First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)

This study is looking into a new experimental drug, ALN-SNCA, for people in the early stages of Parkinson's disease. Parkinson's is a condition that affects movement due to the loss of nerve cells in the brain and a build-up of a protein called alpha-synuclein. The main goal is to check if the new drug is safe to use and what side effects it might have, so it can be considered for further testing. Researchers also want to find out if the drug can reduce the level of alpha-synuclein protein in the fluid around the brain and spinal cord. They will also explore how the drug moves through the body and if it might help slow down the progression of Parkinson's symptoms.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Regeneron Pharmaceuticals

Enrolment target

Start

05 Dec 2025

Estimated completion

07 Jun 2029

Parkinsons Disease

What is this study about?

This study is investigating a new medication called ALN-SNCA, which is currently in the early stages of research. It's designed for people who have Parkinson's disease, specifically those in the early stages. Parkinson's is a condition that slowly affects the brain, leading to problems with movement. It's thought to be linked to a harmful protein called alpha-synuclein building up in the brain.

The main aim of this study is to see if ALN-SNCA is safe for people to take and how well their bodies tolerate it. This is a very important first step to decide if the drug is promising enough to continue testing in larger studies. Researchers will also be keeping an eye out for any side effects people might experience.

Beyond safety, the study also aims to understand how the drug works. This includes looking at whether it can lower the levels of that specific alpha-synuclein protein in the fluid around the brain and spine. They'll also track how much of the drug is in the blood, urine, and this spinal fluid at different times. Ultimately, they hope to learn if this new drug could potentially help to slow down the progression of Parkinson's symptoms, offering a new way to manage the condition in the future.

Key takeaways

This study is testing a new drug (ALN-SNCA) for early Parkinson's disease.
The main goals are to check the drug's safety and how well people tolerate it.
Researchers also want to see if it can reduce a specific protein linked to Parkinson's.
You would either get the new drug or a dummy drug (placebo).
Participation involves regular clinic visits, blood tests, and possibly MRI scans and spinal taps.
The study spans ages 50 to 80, for those diagnosed with Parkinson's within the past four years.

Who may be eligible?

To be considered for this study, you would need to be between 50 and 80 years old. You must have received a diagnosis of Parkinson's disease within the last four years. Your doctor will need to confirm your Parkinson's diagnosis based on specific criteria, including having slow movements plus at least one other key sign like shaking at rest or stiffness.

Regarding your current medications, there are two main paths: either you are not currently taking any standard Parkinson's treatments and are not expected to need them for about six months after starting the study drug, OR you have been on a stable dose of common Parkinson's medications (like levodopa) for at least three months and are not expected to need dose changes for about six months. Your body mass index (BMI) also needs to be 35 or less.

There are also some reasons why you wouldn't be able to join. For example, if your doctor suspects your Parkinson's symptoms are actually due to another condition, or if you have serious heart, breathing, liver, kidney, digestive, hormone, blood, mental health, or nerve problems (other than Parkinson's) that could make the study unsafe for you or affect the results. Also, if you have a medical history that makes certain study procedures, like a spinal tap or MRI scan, unsafe, or if you have a history of bleeding problems or an allergy to certain numbing medications, you would not be able to participate.

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.

I am aged between 50 and 80 years old.
I was diagnosed with Parkinson's disease within the last 4 years.
My doctor has confirmed my Parkinson's diagnosis based on specific criteria.
I am either not on Parkinson's medication OR have been on a stable dose for at least 3 months and won't need changes for 6 months.
I do not have any other major health conditions that might make participating unsafe or affect the study results.
I am able to undergo procedures like an MRI scan and potentially a spinal tap.

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive either the experimental drug ALN-SNCA or a placebo (a dummy drug with no active ingredients). This is usually given as an injection. Throughout the study, you will have regular visits to the clinic where you'll undergo assessments such as physical examinations, blood and urine tests, and potentially MRI scans of your brain. Some participants will also have lumbar punctures (spinal taps) to collect fluid from around the brain and spine. These procedures help the research team check your health, see how the drug is affecting your body, and monitor for any side effects. The total duration of your participation in the study, including all treatments and follow-up appointments, will be clearly explained by the study team.

Potential risks and benefits

Participating in this study might offer potential benefits, such as contributing to medical knowledge that could help future Parkinson's patients, and you would receive close medical monitoring. However, as with any experimental drug, there are potential risks, including side effects from the drug itself or from the study procedures like injections or spinal taps. Some side effects might be mild, while others could be more serious or currently unknown. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Montreal Neurological Institute and Hospital
Verified postcode
Montreal, Canada· Recruiting
Center for Human Drug Research
Verified postcode
Leiden, Netherlands· Recruiting

Common questions

What is Parkinson's disease?

Parkinson's disease is a condition that affects the brain, leading to problems with movement like tremors, stiffness, and slow motion.

What is the ALN-SNCA drug?

ALN-SNCA is a new medication being tested to see if it can help with early Parkinson's disease by targeting a specific protein in the brain.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks like the study drug but doesn't contain any active medicine. It helps researchers compare the real drug's effects.

How long does the study last?

The full duration of your involvement, including all treatments and check-ups, will be explained by the study team.

Will I know if I'm getting the real drug or the placebo?

Usually, in studies like this, neither you nor the study team will know whether you are receiving the real drug or the placebo until the study ends. This helps ensure unbiased results.

How to find out more

Clinical Trials Administrator

clinicaltrials@regeneron.com 844-734-6643 Official trial record

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