Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A Randomized, Placebo-Controlled, Double-Blind, Single Dose, Phase 1 Study to Explore the PK/PD Relationship of QRL-101 in People Living With ALS

This is an early-stage research study, called a Phase 1 study, for a new medicine named QRL-101. It's designed for people living with Amyotrophic Lateral Sclerosis (ALS), sometimes known as Motor Neuron Disease. The main goal is to understand how QRL-101 behaves in the body (pharmacokinetics) and what effects it has (pharmacodynamics) after a single dose. Participants will be given either the active medicine or a 'dummy' medicine (placebo) without knowing which one they receive. The researchers and participants are kept unaware of who gets what, which helps ensure the study results are as fair and unbiased as possible. This study is an important first step in testing new potential treatments for ALS.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Quralis Corp.

Enrolment target

Start

14 Oct 2024

Amyotrophic lateral sclerosis (ALS)

What is this study about?

This study is an early investigation into a new potential treatment for Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neuron Disease. ALS is a condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and wasting. The new medicine being tested is called QRL-101.

Because this is a Phase 1 study, it's focused on understanding the basics of how QRL-101 works. Researchers will be looking at how the medicine is absorbed, how it moves through the body, and how quickly it's removed. They also want to see if it has any noticeable effects. This helps them work out a safe and effective dose for future studies, and to better understand if it could be a helpful treatment for ALS.

The study is designed to be 'randomized' and 'double-blind.' This means that some participants will receive the actual new medicine, while others will receive a 'placebo' – a substance that looks exactly like the medicine but contains no active drug. Neither the participants nor the doctors and nurses involved will know who is receiving which, to ensure that everyone's experience is measured as fairly as possible. This is a common and important step in developing new medicines.

Key takeaways

This is an early-stage (Phase 1) study for a new ALS medicine called QRL-101.
It aims to understand how the medicine works in the body and its effects.
Participants will receive either the study medicine or a dummy medicine (placebo).
Neither you nor the doctors will know which treatment you are receiving.
It's open to adults aged 18 and over with an ALS diagnosis.
Your involvement helps researchers learn about potential new ALS treatments.

Who may be eligible?

This study is looking for adults who have been diagnosed with Amyotrophic Lateral Sclerosis (ALS). You must be at least 18 years old to take part; there is no upper age limit.

Both men and women are welcome to participate in this study. The researchers want to include a wide range of people with ALS to understand how QRL-101 might affect different individuals.

Whilst the exact details of who can and cannot join are not fully available here, a doctor involved in the study will review your medical history and current health to determine if this study is suitable and safe for you.

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 18 years old or older?
Have you been diagnosed with Amyotrophic Lateral Sclerosis (ALS)?
Are you able to attend study visits as required?
Are you comfortable with the possibility of receiving a placebo (dummy medicine)?
Are you willing to have blood tests and other health checks during the study?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive a single dose of either the study medicine (QRL-101) or a placebo. You won't know which one you receive, and neither will the study team. Over a period of time after receiving the dose, you would have several visits to the clinic. During these visits, the study team would check your health, take blood and urine samples, and carry out other tests to see how the medicine is affecting your body and to monitor for any side effects. The total duration of your involvement in the study would be explained to you in detail by the study team.

Potential risks and benefits

Participating in a study like this may not directly benefit you, as the main goal is to learn more about the medicine. However, you would be contributing valuable information that could help others with ALS in the future. As with any medicine, there's a chance of side effects, which the study team will monitor closely and explain in full. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands

Common questions

What is ALS?

ALS stands for Amyotrophic Lateral Sclerosis, also known as Motor Neuron Disease. It's a condition that affects nerve cells that control muscles, leading to muscle weakness over time.

What does 'Phase 1' mean?

Phase 1 is the first stage of testing a new medicine in people. It mainly focuses on understanding if the medicine is safe, how it works in the body, and finding a suitable dose.

What does 'placebo' mean?

A placebo is an inactive substance that looks exactly like the real medicine. Some people in the study will get the actual medicine, and some will get the placebo, but no one will know which, to ensure fair results.

Will I get the actual medicine or a dummy medicine?

Neither you nor the study doctors will know if you receive the active medicine (QRL-101) or the dummy medicine (placebo). This is a standard way to make sure the study results are reliable.

Why is this study important for people with ALS?

This study is an early but crucial step in finding new treatments for ALS. By understanding how QRL-101 works, researchers can decide if it's worth exploring further in larger studies.