A phase 2b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis (ALS)
This research study is looking at a new medicine called Usnoflast for adults living with Amyotrophic Lateral Sclerosis (ALS), sometimes called Motor Neurone Disease. The main goal is to find out if Usnoflast can help slow down the progression of ALS and if it is safe to use. Half of the participants will receive the active medicine, Usnoflast, while the other half will receive a 'dummy' medicine called a placebo. The placebo looks exactly like Usnoflast but contains no active drug. This helps researchers fairly compare the effects of the new medicine. The study will last for 36 weeks, and throughout this time, researchers will carefully monitor how the disease changes and keep track of any side effects.
At a glance
What is this study about?
This study is a clinical trial exploring a new medicine called Usnoflast for people 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 aim of this research is to see if Usnoflast can help slow down the progression of ALS and improve the quality of life for those affected. Researchers will also be checking how safe the medicine is.
To get reliable results, this study is 'randomised' and 'double-blind'. This means that participants will be randomly assigned to receive either the active medicine (Usnoflast) or a placebo, which looks identical but contains no medicine. Neither you nor your study doctor will know which you are receiving, which helps ensure the study results are as fair and unbiased as possible. This is a common and important way to test new medicines.
During the study, several different measures will be used to understand how Usnoflast affects ALS. These include tracking changes in your daily abilities, breathing, and levels of certain substances in your blood and spinal fluid. Your overall health and any side effects will also be closely monitored. This information helps researchers understand how the medicine works and if it could be a helpful treatment in the future.
Key takeaways
- This study is testing a new medicine called Usnoflast for adults with ALS.
- It aims to see if Usnoflast can slow down ALS progression and if it's safe.
- Participants will receive either Usnoflast or a look-alike placebo.
- The study involves regular clinic visits for assessments over 36 weeks.
- You have the right to withdraw from the study at any time.
- This is a Phase 2b study, meaning it's still in the early stages of testing.
Who may be eligible?
This study is looking for adults who have been diagnosed with Amyotrophic Lateral Sclerosis (ALS). Both men and women are welcome to participate.
To be considered, you must be at least 18 years old. There isn't an upper age limit mentioned, so older adults with ALS are also encouraged to see if they might qualify.
Beyond these basic requirements, there will be other medical checks and criteria that your doctor will discuss with you to ensure the study is safe and suitable for your specific health situation.
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 regular clinic visits for about 36 weeks?
- Are you willing to take study medicine (either active or placebo) daily?
What does participation involve?
If you decide to take part in this study, you would receive either the new medicine, Usnoflast, or a placebo (a dummy medicine) in capsule form. The capsules for both Usnoflast and the placebo will look exactly the same – same size, shape, and colour – so you won't know which you are receiving. This treatment period will last for 36 weeks, which is about 8 to 9 months.
Throughout these 36 weeks, you will have regular visits to the study clinic. During these visits, the research team will perform various health checks and assessments. These might include measurements of your breathing, tests to assess your daily abilities (like walking, eating, and speaking), blood tests, and possibly some spinal fluid tests. These tests are to see how the medicine is affecting your ALS and to monitor your general health and any side effects.
After the 36-week treatment period, there will be follow-up assessments to ensure your well-being. All your care and study-related medicines will be provided by the research team during your participation.
Potential risks and benefits
Locations (9)
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Common questions
What is ALS?
ALS stands for Amyotrophic Lateral Sclerosis. It's a condition that affects nerve cells, leading to muscle weakness and difficulty with movement, speaking, and breathing.
What is Usnoflast?
Usnoflast is the name of the new medicine being tested in this study to see if it can help people with ALS.
What is a placebo?
A placebo is a 'dummy' medicine that looks exactly like the real medicine but contains no active drug. It helps researchers compare the effects of the new medicine fairly.
How long will the study last?
The main treatment part of the study will last for 36 weeks, which is about 8 to 9 months, including follow-up checks.
Will I know if I'm getting the real medicine?
No, this is a 'double-blind' study, meaning neither you nor your doctors will know if you're receiving Usnoflast or the placebo until after the study is complete, or in an emergency.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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