Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A study to learn how vamorolone affects the body, how safe it is and how it moves into, through and out of the body in healthy male participants

This study is exploring a new medicine named vamorolone. It's a very early-stage study, called a Phase 1 trial, and it involves healthy male volunteers. The main goals are to find out how vamorolone behaves in the human body, whether it's safe to use, and how it's absorbed, distributed, and removed by the body. This is an important first step for medicines that could eventually help people with serious conditions like Duchenne muscular dystrophy and Becker muscular dystrophy. By studying healthy individuals first, researchers can gather crucial information before potentially moving on to studies directly involving patients who have these muscle-wasting conditions.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Santhera Pharmaceuticals (Schweiz) AG, Santhera Pharmaceuticals (Schweiz) AG

Enrolment target

Start

06 May 2024

Phase I study in healthy male volunteers. Intended indication: Duchenne muscular dystrophy and Becker muscular dystrophy

What is this study about?

This study is about a new medicine called vamorolone. It's an early-stage study, which means it's one of the very first times this medicine is being tested in people. The main purpose is to understand how the medicine works in the body of healthy men. Researchers want to see how much of the medicine gets into the blood, how it travels around the body, and how the body gets rid of it. They also want to check for any side effects and learn about the safest dose.

Think of it like testing a new car engine for the first time. You wouldn't put it straight into a racing car; you'd test it in a controlled environment to see how it runs, how much fuel it uses, and if there are any unexpected problems. Similarly, with a new medicine, an early study in healthy volunteers helps scientists gather important information about how it performs before trying it in people who are unwell.

The valuable information gathered from this study, especially about how safe the medicine is and how the body handles it, is a crucial step. This knowledge will help researchers decide if vamorolone could be a good treatment for muscle-wasting conditions like Duchenne muscular dystrophy and Becker muscular dystrophy in the future. It's an important part of making sure any future treatments are as safe and effective as possible for patients.

Key takeaways

It's a very early-stage study of a new medicine called vamorolone.
Only healthy men aged 18 and over can join.
The main goal is to check how safe the medicine is and how it works in the body.
Findings could help develop future treatments for muscle conditions.
Participation involves taking the medicine and having regular health checks.
You can stop taking part at any time without a reason.

Who may be eligible?

This study is looking for volunteers who are men aged 18 years or older. This means you need to be an adult male to take part. There is no upper age limit mentioned, so older men can also be considered.

The study is specifically designed for healthy individuals. This means that to be eligible, you should generally be in good health and not have any significant ongoing medical conditions that might affect how the medicine works or put you at higher risk. The study team will check your health carefully to make sure it's safe for you to participate.

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?
Are you 18 years old or older?
Are you generally in good health?
Are you able to attend clinic visits regularly?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be given the study medicine, vamorolone. You would likely have several visits to the study clinic over a period of time. During these visits, the study team would carry out different assessments. This might include blood tests to see how much of the medicine is in your body and how your body is reacting to it, as well as general health checks like measuring your blood pressure and heart rate. You would also be asked about any feelings or changes you notice after taking the medicine. The total length of your involvement in the study, including all visits and follow-ups, would be explained in detail by the study team.

Potential risks and benefits

Taking part in a study like this might not directly benefit your health immediately, as it's designed to gather general information about a new medicine. However, you would be helping researchers learn more about a potential new treatment for serious muscle conditions, which could benefit others in the future. As with any medicine, there's always a chance of side effects, which could be mild or, in rare cases, more serious. The study team will explain all known or potential risks to you before you decide to join. Remember, taking part is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your 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
Germany

Common questions

What is Duchenne and Becker muscular dystrophy?

These are serious conditions that cause muscles to become weak and waste away over time. This study is hoping to find ways to help people living with these conditions in the future.

Why are only healthy men being asked to join?

This is a very early-stage study (Phase 1). Testing new medicines in healthy volunteers first helps researchers understand how the medicine works and if it's safe, without the complications of existing illnesses. This makes it easier to spot how the medicine affects the body.

Will I get paid for taking part?

The study information doesn't specify payment, but it's common for early-stage studies involving healthy volunteers to offer some compensation for your time and travel. This is something the study team will discuss with you.

What kind of tests will I have?

You can expect to have general health checks, blood tests and possibly urine tests. These help the doctors understand how the medicine is affecting your body and to check for any side effects.

Is this medicine available for patients now?

No, vamorolone is an experimental medicine, which means it's still being tested. It's not yet available as a treatment for conditions like Duchenne or Becker muscular dystrophy.