RecruitingOBSERVATIONAL

Danon Disease Natural History Study

This international study is called the Danon Disease Natural History Study. It's designed to help doctors and researchers understand more about Danon disease, a rare genetic condition that usually causes heart problems. The study will collect information from adult males (aged 8 and over) and females (aged 8 to 50) who have Danon disease. Researchers will look at symptoms, how the disease changes over time, important health events, and how it affects daily life, all while people receive their usual medical care. No new treatments are being tested in this study. The information gathered will be used to build a clearer picture of Danon disease, which could help in developing future treatments.

At a glance

Status

Recruiting

Sponsor

Rocket Pharmaceuticals Inc.

Enrolment target

Start

20 Dec 2023

Estimated completion

01 Mar 2030

Danon Disease

What is this study about?

Imagine doctors are trying to understand a very rare puzzle, and that puzzle is Danon disease. This study, called the Danon Disease Natural History Study, is like carefully gathering all the pieces of that puzzle to see the full picture. It's an international effort, meaning doctors from different countries are working together. The main goal is to simply observe and learn about Danon disease as it naturally unfolds in people's lives.

Danon disease is a genetic condition, which means it's caused by a change in a person's genes. It often leads to serious heart problems. Because it's so rare, there isn't a lot of information about how it progresses over many years. This study aims to fill that gap by collecting a lot of information from people who have Danon disease – looking at their symptoms, how their condition changes, any major health events they experience, and how the disease affects their daily life. Importantly, people in this study will continue to receive their standard medical care; no new experimental treatments are being given.

By gathering this detailed information, researchers hope to get a much better understanding of Danon disease. This knowledge is incredibly valuable because it can help in several ways: it can improve how doctors diagnose and manage the condition, and it can also lay the groundwork for developing new and more effective treatments in the future. Some of this information might even be used to compare with people taking part in trials of new medicines.

Key takeaways

This study is about understanding Danon disease, not testing new treatments.
It collects health information from people with Danon disease from different countries.
Participation helps researchers learn how the disease changes over time.
Male patients aged 8+ and female patients aged 8-50 with confirmed Danon disease are eligible.
Your privacy and medical care will remain a top priority throughout the study.

Who may be eligible?

To join this study, specific requirements must be met to make sure all the information gathered is useful for research. Generally, if you have been diagnosed with Danon disease through a genetic test showing a change in the LAMP2 gene, and you are aged 8 or older, you might be eligible.

For females, if you are between 8 and 50 years old and have shown signs of a thickened heart muscle (left ventricular hypertrophy), you could be considered. If you've previously had a heart transplant or needed a heart pump, and have at least 30 days of medical records from before that procedure, you might also be able to join by providing past health information.

However, you wouldn't be able to join if you are already taking part in another study that tests new medicines or if you have previously received gene therapy. Also, if you are a female over 50 years old, you wouldn't be able to participate. This is to ensure the study focuses on a specific group of people to get the most accurate results.

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.

Do you have a confirmed genetic diagnosis of Danon disease (LAMP2 gene change)?
Are you male and aged 8 or older?
Are you female and aged between 8 and 50?
If you are a female, do you have evidence of a thickened heart muscle?
Are you NOT currently in another study testing a new drug or treatment?
Have you NOT had gene therapy before?

Answer every question to see your result.

What does participation involve?

Taking part in this study means sharing your existing and future health information with the researchers. There are no new medications or treatments involved; you will continue to receive your usual medical care from your own doctors. Researchers will collect information from your medical records from before you joined the study, and they will continue to collect information about your health as you go through your regular doctor's appointments and treatments. The duration of your involvement isn't fixed, as it depends on how much information is needed over time to understand the disease's natural course.

Potential risks and benefits

The main benefit of taking part is helping doctors and researchers learn more about Danon disease, which could lead to better diagnoses, care, and future treatments for others. There are no direct medical benefits for you, as no new treatments are given. The risks are generally low, mainly involving the release of your medical information to the study team, though this is managed carefully to protect your privacy. You have the right to withdraw from the study at any time without affecting your medical care.

Locations (13)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Phoenix Children's Hospital
Verified postcode
Phoenix, United States· Recruiting
University of California, San Diego
Verified postcode
La Jolla, United States· Recruiting
Children's Hospital Colorado
Verified postcode
Aurora, United States· Recruiting
University of Florida College of Medicine
Verified postcode
Gainesville, United States· Recruiting
University of Michigan
Verified postcode
Ann Arbor, United States· Recruiting
Washington University
Verified postcode
St Louis, United States· Recruiting
Columbia University Medical Center/New York Presbyterian
Verified postcode
New York, United States· Recruiting
Texas Children's Hospital
Verified postcode
Houston, United States· Recruiting
German Heart Center Munich
Verified postcode
München, Germany· Recruiting
Sheba Medical Center
Verified postcode
Ramat Gan, Israel· Recruiting
Azienda Ospedaliera Universitaria Meyer IRCCS
Verified postcode
Florence, Italy· Recruiting
Hospital Universitario Puerta de Hierro Majadahonda
Verified postcode
Majadahonda, Spain· Recruiting

Common questions

What is Danon disease?

Danon disease is a rare genetic condition that primarily affects the heart, causing it to become thick and weak. It can also affect other parts of the body.

Will I receive new medicine in this study?

No, this is an 'observational' study, meaning doctors will just be watching and learning about Danon disease. You will continue to receive your usual medical care from your own doctors.

Who can take part in this study?

The study is looking for males aged 8 and over, and females aged 8 to 50, who have a confirmed diagnosis of Danon disease. There are specific medical criteria, so your doctor can check if you are eligible.

How long will I be in the study?

The study doesn't have a fixed end date for individual participants. Information will be collected over time to understand the disease's long-term effects.

Will my privacy be protected?

Yes, all your personal and medical information will be handled with strict confidentiality and in line with data protection rules.

How to find out more

Clinical Information

clinicaltrials@rocketpharma.com 646-627-0033 Official trial record

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