A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy
This research study is investigating a new medication, NMD670, for adults who have Type 3 spinal muscular atrophy, a condition that affects muscle strength and movement. The main aim is to find out if NMD670 can help people with SMA walk further and improve their overall muscle strength. It will also check how safe the medicine is and if people can tolerate it well, meaning they don't have too many side effects. Participants will be randomly given either NMD670 or a placebo (a dummy pill with no active medicine) for a short period. After a break, they will then switch over to try the other treatment. This helps researchers compare the effects of the new medicine fairly. All participants will be adults, aged 18 or over.
At a glance
What is this study about?
This research is looking at a new medicine called NMD670 for people living with Type 3 spinal muscular atrophy (SMA). SMA is a genetic condition that causes muscles to become weaker over time, which can affect daily activities like walking. Because NMD670 is a new treatment, this study is considered 'Phase 2'. This means researchers are performing initial tests to see if the medicine works, how safe it is, and what the right dose might be, before doing much larger studies.
The main goal of this study is to see if NMD670 can help people with Type 3 SMA walk further. Participants will take either the new medicine or a dummy pill (placebo). A placebo looks just like the real medicine but contains no active drug. This helps researchers work out if any improvements are truly due to NMD670 or something else. Participants will then switch to the other treatment. This helps ensure everyone gets a chance to try the new medicine and makes the study results more reliable.
The study will also look at whether NMD670 helps improve muscle strength in different parts of the body. Beyond walking distance and muscle strength, researchers will carefully monitor how participants feel, checking for any side effects and looking at other signs of health through medical tests. By exploring how NMD670 affects people, this study aims to gather important information that could potentially lead to new and better treatments for Type 3 SMA in the future.
Key takeaways
- This study is testing a new medicine called NMD670 for adults with Type 3 SMA.
- The main goal is to see if NMD670 can help people walk further and improve muscle strength.
- You will take NMD670 or a dummy pill (placebo), then switch treatments.
- Researchers will check your walking, strength, and overall health.
- All participants must be adults, aged 18 or over.
- You can stop participating at any time.
Who may be eligible?
To be considered for this study, you must be an adult aged 18 or over. This study is open to both men and women.
Since this is an early-stage study, researchers are usually very specific about who can take part to ensure safety and clear results. This means that while many people with Type 3 SMA might benefit from a new treatment, not everyone will be able to join this particular study.
Further specific criteria, such as your general health, other medications you might be taking, and how your SMA currently affects you, will be discussed by the study team. These details help make sure the study is right 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?
- Do you have a diagnosis of Type 3 spinal muscular atrophy?
- Are you able to walk (even if with some difficulty or needing aids)?
- Are you able to attend regular clinic visits for assessments?
What does participation involve?
If you decide to take part, you will be in the study for a short period. You will take study medication, which will be either NMD670 or a placebo (a dummy pill), for 21 days. After a break, you will then switch to the other treatment for another 21 days. Neither you nor your study doctor will know which treatment you are taking at any given time.
Throughout the study, you will have several visits to the study clinic. These visits will involve various assessments to see how you are doing. Key assessments include walking tests, like seeing how far you can walk in six minutes, and checks on your muscle strength using special equipment. You will also have regular health checks, such as physical examinations, blood tests, and measurements of your heart activity (ECG). Researchers will also ask about any side effects you might experience. The total duration of your participation in the study, including follow-up, will be relatively short.
Potential risks and benefits
Locations (6)
- —UnverifiedNetherlands
- —UnverifiedSpain
- —UnverifiedBelgium
- —UnverifiedDenmark
- —UnverifiedGermany
- —UnverifiedItaly
Common questions
What is Type 3 spinal muscular atrophy?
It's a genetic condition that causes muscles to become weaker over time, affecting movement and strength.
What is NMD670?
NMD670 is a new medicine being tested to see if it can help improve walking and muscle strength in people with Type 3 SMA.
What does 'placebo' mean?
A placebo is a dummy pill that looks like the actual medicine but contains no active drug. It helps researchers compare the effects of the new medicine fairly.
What is a 'crossover study'?
It means you will try one treatment (either the new medicine or the dummy pill) for a period, and then switch to the other treatment later on, so you try both.
How long will I be in the study?
The study involves a short period of taking medication for about 21 days with each treatment, plus a break and follow-up checks.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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