A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
This clinical study is investigating a new treatment called GNT0004 for Duchenne Muscular Dystrophy (DMD). DMD is a muscle-wasting condition where the body doesn't make enough of a vital protein called dystrophin. GNT0004 is a 'gene therapy' designed to deliver a modified, working version of the dystrophin gene to a patient's muscles. The goal is to help their muscles produce a shortened, but functional, dystrophin protein. This could potentially slow down the progression of the disease. The study is for boys aged 6-10 and is looking at how safe the treatment is and how well it works compared to a placebo (a dummy treatment). Participants receive the treatment as a one-time drip into a vein, and they will be followed for several years.
At a glance
What is this study about?
Duchenne Muscular Dystrophy (DMD) is a serious condition that causes muscles to become weaker over time. It happens because the body can't make a protein called dystrophin, which is vital for healthy muscle function. Without enough dystrophin, muscle cells get damaged easily, leading to a gradual loss of muscle strength, affecting walking, breathing, and heart function.
This study is testing a new treatment called GNT0004. This is a type of 'gene therapy,' which means it aims to fix the underlying problem by delivering a new, working gene. Think of it like giving your muscles a tiny instruction manual to make a crucial protein. GNT0004 uses a harmless virus (like a delivery truck) to carry a special, shortened version of the dystrophin gene directly into the muscle cells, including the heart. The hope is that once inside, these cells can start making a functional 'mini-dystrophin' protein, which could protect the muscles from damage and slow down the disease.
The researchers want to see how safe GNT0004 is and whether it can effectively help boys with DMD. They also want to understand how long any positive effects might last. This treatment is given as a single infusion (a drip into a vein) to help the new gene reach muscles all over the body. It's a significant step in finding new ways to manage and treat DMD.
Key takeaways
- This study tests a new gene therapy, GNT0004, for Duchenne Muscular Dystrophy (DMD).
- It aims to replace a missing protein in muscles to slow disease progression.
- The study involves boys aged 6-10 who can still walk.
- Participation includes a single treatment (or placebo) and up to 5 years of follow-up.
- Researchers are assessing both the safety and effectiveness of GNT0004.
- All participants eventually receive the active treatment.
Who may be eligible?
This study is specifically for boys aged between 6 and 10 years old who have Duchenne Muscular Dystrophy (DMD). To be considered, they must be able to walk on their own and have a confirmed diagnosis of DMD through genetic testing.
There are several other criteria to ensure the treatment is as safe as possible and to help researchers accurately measure its effects. For example, boys must have received standard steroid treatment for at least six months before joining the study. They will also need to meet certain physical ability scores in walking tests and have a healthy heart, as assessed by doctors.
Boys would not be able to join if they have certain specific genetic changes in their dystrophin gene, or if they have certain existing health conditions like severe heart problems or need help with breathing. They also can't have certain antibodies in their blood that might stop the treatment from working.
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.
- Is the person a boy aged between 6 and 10 years old?
- Does he have a confirmed diagnosis of Duchenne Muscular Dystrophy?
- Can he walk on his own without assistance?
- Has he been taking standard steroid medication for at least 6 months?
- Does he have healthy heart function, as confirmed by a doctor?
- Does he meet specific criteria for muscle function tests?
What does participation involve?
Taking part in this study involves several stages over a long period. Firstly, you will undergo screening tests to see if you are a good fit for the study. If eligible, you will then be randomly assigned to receive either the GNT0004 treatment or a placebo (a dummy treatment that looks like GNT0004 but contains no active medicine).
The GNT0004 or placebo is given as a single, one-time drip into a vein. For the first 18 months, you won't know if you received the active treatment or the placebo (this is called being 'blinded'). After these 18 months, there's a crossover: those who received the placebo will then get the GNT0004, and those who received GNT0004 will receive the placebo. Following this, everyone who received GNT0004 will be followed for a total of 5 years from when they first got the treatment. This long follow-up helps researchers check for any long-term side effects and see how long the treatment's benefits last. During the study, you'll have regular visits, assessments, and tests (like blood tests and walking tests) to monitor your health and the treatment's effects. The total duration of participation could be up to 5 years.
Potential risks and benefits
Locations (14)
- Hôpital Universitaire des Enfants Reine FabiolaVerified postcodeBrussels, Belgium· Recruiting
- UZ LeuvenVerified postcodeLeuven, Belgium· Recruiting
- Hôpital de la CitadelleVerified postcodeLiège, Belgium· Not yet recruiting
- University Hospital of BordeauxVerified postcodeBordeaux, France· Recruiting
- Brest University Hospital CentreVerified postcodeBrest, France· Recruiting
- Hôpital Femme Mère EnfantVerified postcodeBron, France· Recruiting
- CHU LilleVerified postcodeLille, France· Recruiting
- Centre Hospitalier Universitaire De MontpellierVerified postcodeMontpellier, France· Not yet recruiting
- Institut I-Motion, Hôpital Armand TrousseauVerified postcodeParis, France· Recruiting
- Hôpitaux Universitaires de StrasbourgVerified postcodeStrasbourg, France· Recruiting
- Hospital Sant Joan de Déu BarcelonaVerified postcodeEsplugues de Llobregat, Spain· Recruiting
- Hospital Universitario y Politécnico La FeVerified postcodeValencia, Spain· Not yet recruiting
Common questions
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD) is a genetic condition that causes muscles to become weaker over time, leading to difficulty with movement, breathing, and heart function.
What is GNT0004?
GNT0004 is an experimental 'gene therapy' that aims to deliver a working version of the dystrophin gene into muscle cells to help them produce a vital protein that strengthens muscles.
Is GNT0004 a cure for DMD?
GNT0004 is a new treatment being studied to see if it can slow down or delay the progression of DMD. It's not yet known if it can cure the condition.
How is GNT0004 given?
GNT0004 is given as a single dose through a drip into a vein (intravenous infusion), which helps it travel to muscles throughout the body.
Who can join this study?
This study is for boys aged 6 to 10 years old with Duchenne Muscular Dystrophy who are still able to walk and meet other specific health criteria.
How to find out more
Medical Lead
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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