RecruitingPHASE2INTERVENTIONAL

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

This study is called a 'Phase 2a' trial and it's looking at a new medicine called SAT-3247 for boys with Duchenne muscular dystrophy (DMD). DMD is a condition that causes muscles to become weaker over time. This particular part of the study focuses on boys aged between 7 and 9 years old who can still walk. We want to find out the best dose of SAT-3247, make sure it's safe, and see if it helps these boys. Some boys in the study will receive SAT-3247, while others will be given a 'placebo' – a dummy medicine that looks the same but contains no active drug. This helps us understand if any changes are truly due to the new medicine. The treatment will be taken for 12 weeks on weekdays. This study is happening in several countries, including the UK.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Satellos Bioscience, Inc.

Enrolment target

Start

08 Dec 2025

Estimated completion

30 Jun 2027

Duchenne Muscular Dystrophy Duchenne DMD Neuromuscular Diseases Muscular Dystrophies

What is this study about?

Duchenne muscular dystrophy (DMD) is a serious condition that mainly affects boys, causing their muscles to gradually weaken. Researchers are always looking for new treatments to help manage the condition and improve quality of life. This particular study is testing a new medicine called SAT-3247. This is a 'Phase 2a' study, which means it's an early stage of testing a new medicine. At this stage, doctors are mainly focused on two things: finding the right dose of the medicine, and checking that it's safe for people to take. They also start to look for early signs that the medicine might be helpful. For this study, they are specifically looking at boys aged 7 to 9 who can still walk. During the study, some boys will receive the active medicine (SAT-3247) and others will receive a 'placebo' – a dummy medicine that looks identical but has no active ingredients. This is a common and important way to test new medicines, as it helps doctors understand if the real medicine is genuinely making a difference. The study will last for 12 weeks, with boys taking the medicine on weekdays.

Key takeaways

This study is testing a new medicine (SAT-3247) for boys (7-9 years old) with Duchenne muscular dystrophy.
It's a 'Phase 2a' study, focusing on finding the right dose and checking safety.
Some boys will get the real medicine, and some will get a dummy medicine (placebo).
Participation involves taking medicine daily for 12 weeks and attending a few clinic visits.
Close medical monitoring is provided throughout the study.
You can withdraw your son from the study at any time without affecting his ongoing care.

Who may be eligible?

This study is specifically for boys who have Duchenne muscular dystrophy (DMD) and are between 7 and 9 years old. It's important that they can still walk on their own at the time they join the study. To be eligible, boys must have a confirmed diagnosis of DMD through genetic testing. They also need to be on a stable dose of certain steroid medicines (like prednisolone or deflazacort) for at least three months before starting the study, if they are taking them. If they stopped taking these steroids, they must have stopped at least three months ago. Other regular medicines or supplements should also be stable for at least a month. Boys who have previously received certain other DMD treatments, such as gene therapy or exon-skipping drugs, might still be able to join. However, there are specific waiting times and conditions, such as their muscle function test results, that need to be met. It's best to discuss your son's full medical history with the study doctor to see if he qualifies.

Quick self-check

Is my son a boy diagnosed with Duchenne muscular dystrophy?
Is my son between 7 and 9 years old?
Is my son able to walk on his own?
Has his DMD diagnosis been confirmed by a genetic test?
If he takes steroid medication, is he on a stable dose for at least 3 months? (Or stopped them at least 3 months ago?)
Are any other regular medicines or supplements he takes on a stable dose for at least 1 month?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your son takes part in this study, he will be screened within 28 days before starting any medicine. This involves checks to make sure he's suitable for the study. After that, there will be a main 'Baseline' visit where things are measured before treatment begins. For 12 weeks, your son will take either the study medicine (SAT-3247) or a placebo (dummy medicine) once a day, every weekday. It's important to stick to this schedule. During these 12 weeks, there will be a phone call after one week, and then visits to the clinic at week 4, week 8, and finally at week 12. These visits are to check how your son is doing, assess the safety of the medicine, and see if there are any signs it's helping. Throughout the study, doctors and nurses will closely monitor your son's health and any changes. The total duration of active participation, including screening and the 12 weeks of treatment and visits, would be about 3 to 4 months.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks. A potential benefit is that SAT-3247 could help with Duchenne muscular dystrophy, but this is not guaranteed as it's still being tested. Your son will also receive close medical attention and monitoring throughout the study, which can be reassuring. Potential risks could include side effects from the medicine, or the inconvenience of regular visits and tests. The study team will explain all known side effects in detail. It's important to remember that participating is completely voluntary, and you have the right to withdraw your son from the study at any time, for any reason, without it affecting his usual medical care.

Locations (21)

University of California Los Angeles
Los Angeles, United States· Recruiting
Colorado Children's
Aurora, United States· Recruiting
Lurie Children's
Chicago, United States· Recruiting
UMass Memorial Medical Center
Worcester, United States· Recruiting
Washington University
St Louis, United States· Recruiting
Nationwide Children's Hospital
Columbus, United States· Recruiting
Seattle Children's
Seattle, United States· Recruiting
Children's Hospital at Westmead
Westmead, Australia· Not yet recruiting
Royal Children's Hospital Melbourne
Melbourne, Australia· Recruiting
Hôpital De La Citadelle (CHR)
Liège, Belgium· Not yet recruiting
UZ Gent
Ghent, Belgium· Recruiting
Children's Hospital Eastern Ontario
Ottawa, Canada· Recruiting

+9 more sites — see the official record for the full list.

Common questions

What is Duchenne muscular dystrophy (DMD)?

DMD is a genetic condition that causes muscles to get weaker over time, making everyday activities more difficult. It mainly affects boys.

What is a 'placebo'?

A placebo is a 'dummy' medicine that looks like the real thing but doesn't contain any active drug. It helps doctors see if the new medicine is really working better than nothing.

Will my son definitely get the new medicine?

No, there's a chance your son might receive the placebo. This is decided by chance, like flipping a coin, so the study can be fair and accurate.

How long will the study last for my son?

Once enrolled, the main part of the study involves taking the medicine for 12 weeks, with a few visits and a phone call during that time. There's also a screening period before starting.

Can my son stop being in the study if we change our minds?

Yes, absolutely. You can choose to withdraw your son from the study at any time, for any reason, and it won't affect his medical care.

How to find out more

Satellos Medical Information

medicalinfo@satellos.com +1 647-660-1780 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.