Active not recruitingPHASE1INTERVENTIONAL

A Study to Evaluate the Safety and Pharmacokinetics of Single and Multiple Doses of Prosetin in Healthy Volunteers and Participants With ALS

This research is investigating a new medicine called prosetin. It's being tested to see if it's safe and how it works in both healthy people and those with Amyotrophic Lateral Sclerosis (ALS), a condition affecting nerve cells. The study is being done in phases. The first parts looked at safety in healthy volunteers. The ongoing parts are focusing on people with ALS to see the medicine's effects, how it's handled by the body, and if it changes certain markers related to ALS. Some participants will receive prosetin, while others will get a placebo (a dummy medicine with no active ingredients). This helps researchers understand if prosetin is effective and safe.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

ProJenX

Enrolment target

Start

26 Feb 2022

Estimated completion

31 Oct 2026

Amyotrophic Lateral Sclerosis

What is this study about?

This research study is all about a new medicine called prosetin. The main aim is to find out if prosetin is safe for people to take and how it behaves in the body, especially in those living with Amyotrophic Lateral Sclerosis (ALS). ALS is a serious condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and problems with movement.

The study has a few stages. The first two stages have already finished and involved healthy volunteers. This helped the researchers understand the basic safety of prosetin and how it's processed by the body when given in single or multiple doses. Now, the study is focusing on people with ALS to see if prosetin is safe for them and if it has any noticeable effects on their condition or certain markers that can give clues about the disease.

Another important part of this study is comparing prosetin to a 'placebo'. A placebo looks like the real medicine but doesn't contain any active ingredients. This comparison helps researchers work out if any changes people experience are truly due to prosetin, or if they might be due to other factors or just the expectation of feeling better. Some participants will also have the chance to continue taking prosetin for a longer period after the initial study phase if they choose to.

Key takeaways

This study is testing a new medicine called prosetin for ALS.
It aims to find out if prosetin is safe and how it works in the body.
Some participants will receive prosetin, others a dummy medicine (placebo).
Participation involves clinic visits and health assessments.
You must be 18 or older and have a confirmed ALS diagnosis to join.
You can stop participating in the study at any time.

Who may be eligible?

To be considered for the ongoing parts of this study, you would need to be at least 18 years old and have a diagnosis of ALS that meets specific criteria. Your lung function, measured by something called Slow Vital Capacity, needs to be reasonably good. If you're already taking medicines for ALS, like riluzole, you'll need to have been on a steady dose for at least a month before joining and plan to continue that throughout the study.

There are also some reasons why you might not be able to join. For example, if you have other serious neurological conditions (apart from ALS) or significant ongoing health issues that require active treatment. A history of seizures, certain types of cancer in the last five years, or having taken part in other experimental drug trials recently would also mean you couldn't join. The doctors running the study will check all these things carefully to make sure the study is safe for you.

If you complete the main part of the study, you might then be able to join an optional longer-term part, as long as you continue to meet certain health requirements.

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 at least 18 years old?
Do you have a confirmed diagnosis of ALS?
Is your lung function (SVC) good enough (above 50% predicted)?
If taking ALS medicines, have you been on a stable dose for at least one month?
Do you have any other serious health conditions that need ongoing treatment?
Have you taken part in another experimental drug trial in the last 30 days?

Answer every question to see your result.

What does participation involve?

If you join the study, you would be taking either the study medicine (prosetin) or a placebo (a dummy medicine) for a set period. In the main part of the study, this would involve taking the medicine for 14 days. During this time, you would have regular visits to the clinic so the study team can check how you are doing, monitor your health, and see how the medicine is affecting your body. This will include different tests and assessments, such as blood tests and checks on your overall health and well-being.

After completing the main 14-day treatment, if eligible and you choose to, you might have the option to continue for a much longer period of up to 52 weeks in an 'open-label' extension. In this longer period, everyone would know they are receiving the actual study medicine, not a placebo. Throughout both parts, you would have ongoing support and monitoring from the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that you might receive access to a new medicine (prosetin) that is not generally available, which could potentially help with your ALS. However, it's also possible that the medicine may not help you, or you might receive the placebo. As with any medicine, prosetin could have side effects, which the study team will carefully monitor. You will be given full details of known risks before deciding to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Massachusetts General Hospital
Verified postcode
Boston, United States
Worldwide Clinical Trials Early Phase Services
Verified postcode
San Antonio, United States
The Neuro - Montréal Neurological Institute-Hospital
Verified postcode
Montreal, Canada
University Medical Center Utrecht
Verified postcode
Utrecht, Netherlands

Common questions

What is ALS?

ALS stands for Amyotrophic Lateral Sclerosis. It's a condition that affects nerve cells in your brain and spinal cord, which help control your muscles. Over time, it can lead to muscle weakness and problems with movement.

What is 'prosetin'?

Prosetin is a new medicine being tested in this study. Researchers want to see if it's safe and how it works in people, especially those with ALS.

What is a 'placebo'?

A placebo is a 'dummy' medicine that looks like the real thing but doesn't contain any active ingredients. It's used in studies to help researchers understand if the real medicine is truly having an effect.

Will I know if I'm getting prosetin or the placebo?

In the main part of the study, neither you nor your doctor will know if you're getting prosetin or the placebo. This is called 'double-blind' and helps ensure the study results are fair. If you join the longer-term part, everyone knows they are receiving prosetin.

What if I experience side effects?

The study team will be carefully monitoring you for any side effects. It's very important to tell them about anything new or unusual you experience so they can provide help and ensure your safety.