Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients with Spinal and Bulbar Muscular Atrophy (SBMA)

This study is looking into a new medicine called NIDO-361 for men who have a condition called Spinal and Bulbar Muscular Atrophy (SBMA). SBMA is a rare genetic condition that causes muscles to become weak and waste away over time. The main goal of the study is to find out if NIDO-361 can help increase muscle size in the thighs and overall body, and if it's safe to use. Half the participants will receive the actual medicine, while the other half will get a placebo (a tablet with no active medicine). This helps researchers understand the true effects of NIDO-361. They will also be checking for any side effects and how participants feel overall.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Nido Biosciences Inc.

Enrolment target

Start

31 Jan 2024

Spinal and Bulbar Muscular Atrophy

What is this study about?

This research study is about a condition called Spinal and Bulbar Muscular Atrophy, or SBMA. SBMA is a rare genetic condition that mainly affects men. It causes muscles to gradually weaken and waste away, particularly in the arms, legs, and the muscles used for speaking and swallowing.

The study aims to test a new investigational medicine, NIDO-361. Researchers want to understand if this medicine can help people with SBMA. Specifically, they are looking to see if NIDO-361 can increase muscle size, especially in the thighs, and generally improve overall muscle health. They also want to make sure the medicine is safe and check for any side effects it might cause.

To get clear results, the study is designed so that some participants will receive the active medicine (NIDO-361), while others will receive a placebo – a tablet that looks the same but contains no active medicine. Neither the participants nor the doctors will know who is getting which tablet until the end of the study. This setup helps researchers confidently determine if any changes or improvements are actually due to NIDO-361 and not to other factors.

Key takeaways

This study is testing a new medicine (NIDO-361) for men with SBMA.
It aims to see if the medicine can increase muscle size and is safe to use.
Half of the participants will receive the active medicine; the other half will receive a dummy pill (placebo).
Participation involves regular clinic visits for various tests and health checks.
You have the right to withdraw from the study at any point.

Who may be eligible?

This study is specifically looking for men who have Spinal and Bulbar Muscular Atrophy (SBMA). You must be at least 18 years old to take part; there is no upper age limit currently stated.

Because this study is testing a new medicine, there will be other specific health requirements and checks to make sure it's safe for you to participate and that the study can get accurate results. These will likely involve looking at your overall health and any other medical conditions you might have.

If you're a man aged 18 or over with SBMA and are interested, your doctor will be able to check the full list of requirements to see if this study could be right 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 a man?
Have you been diagnosed with Spinal and Bulbar Muscular Atrophy (SBMA)?
Are you 18 years old or older?
Are you able to attend regular clinic appointments and complete study assessments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be asked to take a tablet, either the experimental medicine NIDO-361 or a placebo (a dummy pill), for a set period. You wouldn't know which one you're taking, and neither would your study doctor. Throughout the study, you would have regular visits to the clinic.

During these visits, doctors and nurses would perform various assessments. These could include physical examinations, blood tests, and scans of your muscles, such as whole-body MRI scans, to measure muscle size. They would also ask you to do certain tasks to check your walking, grip strength, and general physical activity levels. You might also fill out questionnaires about how you're feeling and how your symptoms are changing.

Your safety would be monitored very closely, with careful checks for any side effects or changes in your health. The total duration of your involvement in the study, including follow-up, would be fully explained by the study team.

Potential risks and benefits

Participating in research studies like this can offer several potential benefits. While there is no guarantee, you might experience improvements in your muscle health or overall well-being. You would also receive close medical attention and monitoring from a dedicated research team. However, it's also important to understand there are potential risks. The investigational medicine, NIDO-361, could cause side effects, some of which might be mild, moderate, or even severe. The study procedures themselves, like blood tests or scans, carry their own small risks. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

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

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Unverified
Italy
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Unverified
Denmark

Common questions

What is SBMA?

SBMA stands for Spinal and Bulbar Muscular Atrophy. It's a rare genetic condition that causes muscles to get weaker and waste away, mainly in men.

What is NIDO-361?

NIDO-361 is an experimental medicine being tested in this study to see if it can help improve muscle health in men with SBMA.

Why will some people get a 'dummy pill'?

Giving some people a dummy pill (placebo) helps researchers clearly see if the medicine NIDO-361 is truly causing any changes, rather than other factors.

What kind of tests will I have?

You might have muscle scans (like MRI), blood tests, physical tests to check your walking and grip strength, and fill out questionnaires.

Can I leave the study if I change my mind?

Yes, you can leave the study at any time, for any reason, without it affecting your medical care.