Active not recruitingPHASE1INTERVENTIONAL

A Study Evaluating the Safety and Tolerability of QRL-201 in ALS

This study is investigating a new treatment, QRL-201, for people diagnosed with Amyotrophic Lateral Sclerosis (ALS), sometimes known as motor neuron disease. The main goal is to find out if QRL-201 is safe and how well people tolerate different amounts of the medicine when it's given directly into the fluid around the spinal cord. This is an early-stage study (Phase 1), which means it's the first time this specific medicine is being tested in humans for ALS. Participants will receive either QRL-201 or a placebo (a dummy treatment). Scientists will carefully monitor participants to learn about the medicine's effects and how the body handles it. This research is crucial for understanding if QRL-201 could be a potential treatment option in the future.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

QurAlis Corporation

Enrolment target

Start

16 Dec 2022

Estimated completion

30 Nov 2027

Amyotrophic Lateral Sclerosis

What is this study about?

This research study is focused on a new medicine called QRL-201 for people living with Amyotrophic Lateral Sclerosis (ALS). ALS is a condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and wasting. The purpose of this study, which is in a very early stage (Phase 1), is to closely examine the safety of QRL-201 and how well people tolerate it at different dose levels. It's the first time this medicine is being given to people with ALS, so researchers will be paying close attention to any side effects.

Participants in the study will receive the medicine, QRL-201, or a placebo (an inactive substance that looks just like the study medicine). The medicine will be given directly into the fluid that surrounds the spinal cord, a method designed to deliver the treatment effectively. Doctors and researchers will then carefully observe how the body reacts to the medicine and how it moves through the body.

The findings from this study are very important because they will help scientists understand if QRL-201 is safe enough to be tested in larger groups of people in later stages of research. This step is essential in the long process of developing new treatments that could potentially help people with ALS in the future. It's a foundational step to see if the treatment has the potential to move forward.

Key takeaways

This is an early-stage study (Phase 1) for a new ALS medicine called QRL-201.
The main aim is to check if QRL-201 is safe and how well people tolerate it.
The medicine is given directly into the fluid around the spinal cord.
Participants will receive either QRL-201 or a placebo.
The study involves regular clinic visits and health monitoring.

Who may be eligible?

To be considered for this study, you would need to be between 18 and 80 years old and have a diagnosis of ALS. Your ALS symptoms should have started within the last two years. You'd also need to meet certain health criteria, such as having good lung function and showing specific signs of ALS that doctors look for. It's important that you are not pregnant or breastfeeding, and if you can get pregnant, you must be willing to use effective contraception.

There are also some reasons why you might not be able to join. For example, if you have certain genetic changes related to ALS (like in the SOD1 or FUS genes), or if you're already taking part in another clinical trial with an experimental treatment. People who have had stem cell or gene therapy in the past, or have certain other medical conditions or abnormal test results, would also not be able to participate. You'll also need to be able to comfortably have a lumbar puncture (a procedure where a small amount of fluid is taken from your spine) as part of the study.

If you are currently taking approved medicines for ALS, you would typically need to be on a stable dose of those medicines before joining the study.

Quick self-check

Are you between 18 and 80 years old?
Were you diagnosed with ALS less than 2 years ago?
Are you not pregnant or breastfeeding?
Are you able to have a lumbar puncture?
Are you willing to use effective contraception if you can get pregnant?
Are you not currently in another gene or stem cell therapy trial?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would receive the study medicine, QRL-201, or a placebo, which looks identical but contains no active medicine. The medicine will be given directly into the fluid around your spinal cord through a procedure called a lumbar puncture. You'll have this done multiple times. The study involves several visits to the clinic where doctors will perform various assessments, including physical examinations, blood tests, and possibly other tests to monitor your health and how your body is reacting to the treatment.

Initially, the study will involve small groups of participants receiving increasing doses of the medicine to establish a safe dose. Following this, a larger group will participate, with most receiving QRL-201 and some receiving the placebo. After this initial 'double-blind' period, where neither you nor your doctor would know if you're getting the active medicine or placebo, there's an option for an 'open-label' extended dosing period where everyone would know they are receiving QRL-201.

Throughout the study, you would be closely monitored by the study team. They will track your health, any side effects you experience, and how the medicine is working in your body. The full duration of your participation, including follow-up visits, would be explained in detail by the study team.

Potential risks and benefits

Participating in a clinical trial like this may offer potential benefits, such as contributing valuable information to medical science and potentially gaining access to a new investigational treatment for ALS that is not yet widely available. However, there are also potential risks involved. These might include side effects from the study medicine, the discomfort of procedures like lumbar punctures, or the time commitment required for clinic visits. It's important to remember that because this is an early-stage study, the full effects and risks of QRL-201 are not yet completely known. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (15)

Universitaire Ziekenhuizen Leuven (UZ Leuven)
Leuven, Belgium
University of Calgary
Calgary, Canada
University of Alberta
Edmonton, Canada
Sunnybrook Health Science Centre
Toronto, Canada
CHUM - Hopital Notre-Dame
Montreal, Canada
Montreal Neurological Institute-Hospital
Montreal, Canada
Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE)
Bonn, Germany
Charité Research Organisation
Berlin, Germany
University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology
Lübeck, Germany
Universitätsklinikum Ulm
Ulm, Germany
St James's Hospital
Dublin, Ireland
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands

+3 more sites — see the official record for the full list.

Common questions

What is ALS?

ALS stands for Amyotrophic Lateral Sclerosis, a condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness.

What is a Phase 1 study?

A Phase 1 study is an early stage of clinical research where a new medicine is tested in a small group of people for the first time to check its safety and how the body handles it.

What is a placebo?

A placebo is an inactive substance that looks exactly like the study medicine but doesn't contain any active ingredients. It's used for comparison.

How is the study medicine given?

The study medicine, QRL-201, is given directly into the fluid surrounding the spinal cord through a procedure called a lumbar puncture.

Can I continue my usual ALS medication?

Yes, if you're on approved ALS medications, you would generally need to be on a consistent, stable dose.