RecruitingPHASE3INTERVENTIONAL

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

This research is looking into a new medicine called Givinostat for Duchenne muscular dystrophy (DMD). Specifically, it's for boys aged 9 to under 18 who use a wheelchair and can no longer walk. The main goal is to find out if Givinostat can help slow down the weakening of upper body muscles. Researchers also want to check how safe the medicine is and if people can tolerate it well. For 18 months, 138 participants will be randomly given either Givinostat or a placebo (a dummy pill). This study hopes to add to our understanding of how Givinostat could help boys with DMD.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Italfarmaco

Enrolment target

138

Start

19 Feb 2024

Estimated completion

01 Feb 2028

Duchenne Muscular Dystrophy

What is this study about?

Duchenne muscular dystrophy is a serious and progressive condition that causes muscles to weaken over time. Doctors are always looking for new and better treatments because, while existing medications like steroids can help, they aren't suitable for everyone. This particular study is exploring a new medicine called Givinostat.

Givinostat works by targeting certain processes in the body that are believed to contribute to muscle problems in DMD. The idea is that by blocking these processes, Givinostat might help to protect muscle strength. This study is specifically focused on boys aged 9 to under 18 who are no longer able to walk and use a wheelchair. The researchers want to see if Givinostat can help reduce muscle decline in their upper body.

This is a 'Phase 3' study, which means it's one of the final steps before a new medicine might become widely available. The information gathered from this study will be added to what we already know about Givinostat from previous research, including a study in boys who can still walk. By looking at different groups of patients, scientists can get a complete picture of whether Givinostat is an effective and safe treatment for Duchenne muscular dystrophy.

Key takeaways

This study is for boys with Duchenne muscular dystrophy who use a wheelchair.
It's testing a new medicine called Givinostat to see if it can help muscles and is safe.
Participants will be randomly given either Givinostat or a placebo for 18 months.
Regular check-ups and assessments will be part of the study.
The total time involved could be up to about 21 months.
You can stop participating at any time if you wish.

Who may be eligible?

To join this study, you need to be a boy with Duchenne muscular dystrophy. You must be between 9 and 17 years old (you can't be 18 or older when you first start the screening process). Doctors will confirm your Duchenne diagnosis through genetic testing.

An important part of this study is that you must be in a wheelchair and unable to walk 10 metres, or unable to do so in under 30 seconds without help. Your upper body strength will also be checked using a specific test, and your scores need to fall within a certain range to be eligible.

If you're already taking medicines for heart conditions related to DMD, these need to have been stable for at least one month before starting the study. Also, if you're on steroids, your dose and schedule must have been stable for at least six months. Everyone participating will need to agree to use effective contraception during and for a few months after the study.

Quick self-check

Are you a boy between 9 and 17 years old?
Do you have a confirmed diagnosis of Duchenne muscular dystrophy?
Do you use a wheelchair and are unable to walk?
Are your existing heart and steroid medications (if any) stable?
Are you willing to use effective contraception if needed?

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

What does participation involve?

If you decide to take part, the study will start with a screening period that lasts about 4 weeks. During this time, doctors will do checks to make sure you're suitable for the study. After screening, there will be a 'baseline' visit where you'll be formally enrolled.

Then comes the main treatment period, which lasts for 18 months (about 72 weeks). During this time, you'll be given either the study medicine, Givinostat, or a placebo (an inactive dummy pill). Neither you nor your doctors will know which one you are receiving – this is called a 'double-blind' study. You'll need to attend regular visits during these 18 months for assessments and to collect your medication.

After the 18 months of treatment, there will be a final appointment called the 'end of study visit'. For some participants, there will then be an optional follow-up period of about 4 weeks. The total time you could be involved in the study is up to about 20-21 months.

Potential risks and benefits

Taking part in any medical study has potential benefits and potential risks. You might benefit from Givinostat if it helps slow down muscle decline, but there's also a chance you could receive the placebo and not get the active medicine. Givinostat may have side effects, and the research team will monitor you closely for any changes in your health. You will undergo various tests and assessments, which might involve some discomfort or inconvenience. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (20)

Universitaire Ziekenhuizen Leuven
Leuven, Belgium· Recruiting
British Columbia Children's Hospital
Vancouver, Canada· Recruiting
The University of Western Ontario - Children's Health Research Institute
London, Canada· Recruiting
University of Ottawa - Children's Hospital of Eastern Ontario
Ottawa, Canada· Recruiting
University of Toronto - Holland Bloorview Kids Rehabilitation Hospital
Toronto, Canada· Recruiting
Centre Hospitalier Régional Universitaire de Lille
Lille, France· Recruiting
Centre hospitalier universitaire - Hôpitaux de Marseille
Marseille, France· Recruiting
Hôpital Armand-Trousseau - I-Motion
Paris, France· Recruiting
Charite-Universitaetsmedizin Berlin
Berlin, Germany· Recruiting
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Germany· Recruiting
Associazione La Nostra Famiglia - IRCCS Eugenio Medea - Bosisio Parini
Lecco, Italy· Recruiting
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscular Omnicentre
Milan, Italy· Recruiting

+8 more sites — see the official record for the full list.

Common questions

What is Duchenne muscular dystrophy?

It's a serious genetic condition that causes muscles to weaken and waste away over time.

What does 'non-ambulant' mean in this study?

It means participants use a wheelchair and cannot walk 10 meters, or cannot do it quickly without help.

Will I know if I'm getting the real medicine or the dummy pill?

No, neither you nor your doctor will know during the study. This helps the results be as fair as possible.

How long will I be involved in the study?

The main treatment part lasts 18 months, and total involvement could be up to about 20-21 months, including screening and follow-up.

What happens after the study ends?

At the end, all patients will be offered the chance to join a longer-term safety study (if available and suitable).

How to find out more

Italfarmaco Patient Advocacy

patientadvocacy@italfarmacogroup.com +39 02 6443 1 Official trial record

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