RecruitingPHASE2, PHASE3INTERVENTIONAL

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

This study is checking the long-term safety and how well a medicine called Givinostat works for boys with Duchenne muscular dystrophy (DMD). It's specifically for those who have already been part of a Givinostat study before. Participants will take the Givinostat liquid medicine twice a day with food. The dose might be adjusted based on their weight and if their blood tests show certain changes, like a drop in blood cell counts. Regular blood tests will be done to make sure the medicine is safe. The study aims to gather more information on Givinostat over a longer period to understand its full effect on DMD.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

Italfarmaco

Enrolment target

206

Start

24 Oct 2017

Estimated completion

01 Dec 2029

Duchenne Muscular Dystrophy

What is this study about?

This study is looking at a medicine called Givinostat, which is being investigated for Duchenne muscular dystrophy (DMD). DMD is a serious condition that causes muscles to become weak over time. Givinostat aims to help by working on certain processes within the body that are linked to DMD.

The main goal of this particular study is to see if Givinostat is safe to take for a long time and to continue observing its effects on the body when used over many months or even years. All the participants in this study have already taken Givinostat in a previous research trial. By continuing to follow these boys, researchers can learn more about the medicine's long-term impact, which is very important for understanding how it might help people with DMD in the future.

The researchers will closely monitor the boys taking part, especially through regular blood tests, to make sure the medicine is not causing any serious side effects. They will also keep an eye on how the boys are doing overall. This long-term information is crucial for deciding if Givinostat could eventually become a treatment option for DMD and for understanding how to use it as safely and effectively as possible.

Key takeaways

This study is for boys with Duchenne muscular dystrophy who have previously taken Givinostat.
It aims to understand the long-term safety and effects of Givinostat.
Participants take Givinostat as a twice-daily oral liquid with food.
Regular blood tests are crucial for monitoring safety, especially blood cell counts.
Dose adjustments may happen based on weight or blood test results.
Participation is voluntary, and you can stop at any time.

Who may be eligible?

To be considered for this study, you must have already taken part in a previous Givinostat study for Duchenne muscular dystrophy. You also need to be at least 6 years old.

It's important that you, and if you're under 16, your parent or guardian, are able to understand and sign the consent forms. You'll also need to agree to use effective birth control methods during and for three months after the study, if that applies to you.

You won't be able to join if you've recently taken other medicines, apart from steroids, that could affect your muscles, or if you're taking another experimental drug. If you have any other serious health problems that the study doctor thinks could make it unsafe for you to participate, you would also not be able to join.

Quick self-check

Have you been in a previous Givinostat study for Duchenne muscular dystrophy?
Are you 6 years old or older?
Are you able to agree to use effective birth control if needed?
Are you currently not taking other experimental drugs?
Are you currently not taking any other muscle-affecting medicine (other than steroids)?

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

What does participation involve?

If you take part in this study, you will continue to receive Givinostat as an oral liquid medicine, twice a day, with food. The amount of medicine you take will be based on what you were taking in the previous study. This dose might be adjusted by the study team, especially if your weight changes, or if certain blood test results show your blood cell counts are too low. You'll have regular check-ups, especially blood tests, which will be weekly for the first month and then every two weeks for the second month, to monitor your safety.

The study medicine would be stopped permanently if you experience severe tummy upsets, have a very serious side effect, or if certain blood test results (like specific blood cell counts) or heart readings fall below safe levels. The study doctor will closely follow you up until any issues are sorted or stable. The total duration of the study is not specified but it is described as a "long-term" study, meaning it could last for an extended period.

Potential risks and benefits

Taking part in this study may offer the potential benefit of continuing to receive Givinostat, which could help manage Duchenne muscular dystrophy in the long term. However, like all medicines, Givinostat can have side effects. Potential risks include developing low blood cell counts (like platelets or white blood cells), which will be closely monitored, or experiencing severe diarrhoea. There's also a tiny risk of heart rhythm changes. You can decide to leave the study at any time, for any reason, without it affecting your medical care.

Locations (39)

University of California - Davis Medical Center - Devis Physical Medicine & Rehabilitation
Sacramento, United States· Completed
Rady Children's Hospital center - UCSD Department of Neuroscience
San Diego, United States· Completed
Connecticut Children's Medical Center, Neurology Division
Hartford, United States· Completed
Child Health Research Institute
Gainesville, United States· Completed
MD Rare Disease Research, LLC
Atlanta, United States· Enrolling by invitation
University of Iowa Children's Hospital
Iowa City, United States· Enrolling by invitation
Washington University School of Medicine in St Louis Department of Neurology 660 S.Euclid Avenue, Campus Box 8111
St Louis, United States· Completed
Shriners Hospitals for Children
Portland, United States· Completed
The Children's Hospital of Philadelphia Colket Translational Research Building
Philadelphia, United States· Completed
Virginia Commonwealth University Childrens Hospital of Richmond at
Richmond, United States· Completed
University Hospitals Leuven, Neuromuscular Reference Centre, Child Neurology
Leuven, Belgium· Enrolling by invitation
Hospital de La Citadelle, Centre de Référence des Maladies Neuromuscolaires (CRMN)
Liège, Belgium· Enrolling by invitation

+27 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 become weak and waste away over time. It mainly affects boys.

What is Givinostat?

Givinostat is a drug being studied to see its effects on Duchenne muscular dystrophy. It's not yet a licensed medicine, but is undergoing research.

Why is this study only for people who were in a previous Givinostat study?

This study is designed to gather more information about the long-term safety and effects of Givinostat, building on what was learned in earlier studies.

How is the medicine given?

Givinostat is given as a liquid medicine that you take by mouth, twice a day, with food.

What happens if I experience side effects?

The study team will closely monitor you with regular check-ups and blood tests. If you have certain severe side effects or concerning blood test results, the medicine would be stopped.

How to find out more

Reference Study ID Number: DSC/14/2357/51

patientadvocacy@italfarmaco.com + 39 02 6443 Official trial record

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