RecruitingPHASE2INTERVENTIONAL

Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old

This research study is about a new medicine called Givinostat for young boys with Duchenne Muscular Dystrophy (DMD). DMD is a condition that causes muscles to weaken over time. The study wants to understand how Givinostat moves through the body (this is called pharmacokinetics) and if it is safe for boys aged between 2 and 6 years old. Researchers will give the medicine to two groups of boys in this age range and closely monitor them. This study is called a 'Phase 2' trial, which means it builds on earlier research and aims to gather more information about the medicine's effects and safety before it can be considered for wider use. There will be careful checks throughout the study, including a main treatment period and an extension period.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Italfarmaco

Enrolment target

Start

02 Jan 2025

Estimated completion

01 Dec 2029

Duchenne Muscular Dystrophy

What is this study about?

Duchenne Muscular Dystrophy (DMD) is a serious genetic condition that mainly affects boys, causing their muscles to become weaker over time. This can lead to problems with walking, breathing, and heart function. Researchers are always looking for new ways to help manage DMD and improve the lives of those affected. This particular study is focused on a new medication called Givinostat.

The main goal of this study is to learn more about Givinostat in younger boys with DMD, specifically those aged 2 to 6 years. The study will look at two key things: firstly, how the medicine is absorbed, distributed, used, and removed by the body (this is often called 'pharmacokinetics'); and secondly, how safe the medicine is for these younger boys. By understanding these aspects, doctors and researchers can decide if Givinostat could be a helpful treatment option in the future.

This is a 'Phase 2' study, which means it's a step in a larger research process. It's not the very first time Givinostat has been studied, but it aims to get more detailed information about its effects and safety. The study will be 'open-label,' which means both the patients (and their parents) and the doctors will know who is receiving the Givinostat medication. It will involve two groups of boys, split by age, to help tailor the dosing and understanding.

Key takeaways

The study is for boys with Duchenne Muscular Dystrophy (DMD).
Eligible boys must be between 2 and 6 years old.
The medicine being tested is called Givinostat.
The study explores how the medicine works and if it is safe in young boys.
Participation could last up to 38 months.
You can withdraw your child at any time.

Who may be eligible?

This study is looking for male children with a confirmed genetic diagnosis of Duchenne Muscular Dystrophy. They must be between 2 and 6 years old when the study starts.

Boys who are already taking steroid medication for DMD can join, but their dose needs to have been stable for at least 3 months. If a boy is not currently taking steroids, they must not start them during the first 48 weeks of the study. It's important that children haven't taken part in other drug studies or some specific DMD treatments recently, for example, within 3 to 12 months, depending on the type of treatment.

Parents or legal guardians will need to give their written permission for their child to join. Older children, depending on local rules, may also need to agree in writing to take part.

Quick self-check

Is your child a male with DMD?
Is your child between 2 and 6 years old?
Does your child have a genetic diagnosis of DMD?
If on steroids, has the dose been stable for at least 3 months, or not planning to start them during the first 48 weeks?
Has your child avoided other study drugs or specific DMD treatments recently (e.g., in the last 3-12 months)?

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, your child's involvement will begin with about 4 weeks of screening tests to make sure they are suitable for the study. After that, there will be a main treatment period where your child will receive the Givinostat medication for about 48 weeks (just under a year). Following this, there's an optional extension period of about 96 weeks (nearly two years), where treatment with Givinostat would continue.

After the treatment periods, there will be a follow-up period of about 4 weeks to check on your child's health. The total time your child could be involved in the study, from screening to the end of follow-up, could be up to 151 weeks, which is about 37 to 38 months.

Potential risks and benefits

Taking part in any medical study carries potential benefits and risks. The potential benefits could include receiving a new medication that might help manage Duchenne Muscular Dystrophy, and closely monitoring by a team of medical experts. However, there may be risks and side effects from the medication, which will be carefully explained to you. It's important to remember that Givinostat is an investigational drug, so we don't yet know its full effects. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (9)

Queen Fabiola Children's University Hospital HUDERF
Brussels, Belgium· Recruiting
Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca' Granda - NeuroMuscolar Omnicenter
Milan, Italy· Recruiting
Ospedale Pediatrico Bambino Gesù
Roma, Italy· Recruiting
Policlinico Universitario Agostino Gemelli - Università Cattolica del Sacro Cuore
Roma, Italy· Recruiting
Leids Universitair Medisch Centrum (LUMC)
Leiden, Netherlands· Recruiting
Leeds Teaching Hospital NHS Trust
Leeds, United Kingdom· Not yet recruiting
Great Ormond Street Hospital - GOSH
London, United Kingdom· Not yet recruiting
Newcastle upon Tyne Hospitals NHS Foundation Trust - Newcastle University
Newcastle upon Tyne, United Kingdom· Recruiting
Oxford University Hospitals NHS Foundation Trust
Oxford, United Kingdom· Recruiting

Common questions

What is Duchenne Muscular Dystrophy (DMD)?

DMD is a genetic condition that causes muscles to become weak and waste away over time, mainly affecting boys.

What is Givinostat?

Givinostat is a new medicine being studied to see its effects and safety in boys with DMD.

Will my child get a placebo (a dummy medicine)?

No, this study is 'open-label,' meaning all participants will receive Givinostat, and everyone involved will know they are getting the active medicine.

How long will the study last?

Overall, participation could last up to 37-38 months, including screening, treatment, and follow-up periods.

Can my child leave the study early?

Yes, you can withdraw your child from the study at any point without it affecting their regular medical care.

How to find out more

Italfarmaco Patient Advocacy

patientadvocacy@italfarmacogroup.com +39 02 64431 Official trial record

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