Diagnosed with Danon Disease?
Clinical studies can shed new light on the cause and progression of the disease and how to treat it.
Join a Clinical Study in Danon Disease
Danon disease is caused by changes in the LAMP2 gene, which prevent cells from clearing waste properly. Over time, waste buildup can harm the heart and other organs, leading to serious and sometimes life-threatening complications.
Because there are no treatments other than heart transplantation that can slow or stop this process, new research is urgently needed to better understand the disease and develop effective therapies.
Joining a Danon disease clinical study provides the chance to help in the development of an effective treatment led by leading Danon disease specialists.
Danon Disease Research Studies
Observational study to collect the medical history and day-to-day experiences of people with Danon disease to provide researchers a greater understanding of this condition. No treatment in this study.
Interventional study for Danon disease, with the gene therapy designed to deliver a functional LAMP2 gene into a patient's cells to replace the damaged gene.
Danon Natural History Study
You have the opportunity to help with the understanding of Danon disease for future patients and families. Because Danon disease is such a rare condition, and there is limited information available, this study helps researchers understand the disease and how it may be treated. Researchers need to learn from the real-life experience of people living with Danon disease, and by joining the LAMPLIGHT Natural History Study, you can provide the information needed.
What to expect during the study:
Yearly lab and echocardiogram (ECHO) and EKG assessments, reporting on key events, and the impact of the disease on quality of life, as it is managed with current treatments, for a total of 3 years.
Patients do not receive any new therapy.
Visits take place at a study site or in some cases can be conducted remotely.
You will continue to see your doctor for regular care.
Note: Costs associated with participation are covered by the study.
Who Can Participate?
Males, age 8 years or older
Females, age 8 to 50 years
Diagnosed with
Danon disease
LAMPLIGHT Danon Disease Gene Therapy Study
The LAMPLIGHT Gene therapy study is an investigational clinical study. The goal of this clinical study is to understand the long-term safety and effectiveness of the gene therapy in improving heart function for patients living with Danon disease.
How does gene therapy work?
Replacing the gene: The gene therapy delivers a working copy of the LAMP2 gene into heart cells, so the cells will be able to clear waste properly.
Single-dose infusion: Treatment is given only once, as a single intravenous (IV) infusion.
Who Can Participate
Males, age 8 years
or older
Diagnosed with
Danon disease
Have not received a heart transplant
Please note: this study is not available to those currently participating in any other investigational clinical studies or who have received any other gene therapy treatment
How to Join the Gene Therapy Study
STEP 1
Support Team Interview
Share the diagnosis, symptoms, and treatment history. If a patient qualifies, he will be directed to a leading Danon disease specialist participating in the clinical study.
STEP 2
Study Site Review
The study site team will review the
patient’s medical history and confirm whether he can move forward in the clinical study process.
STEP 3
Eligibility Screening & Discussion
This will occur at the study site and
includes lab assessments and a more detailed medical history review.
In certain cases, this step may be
done remotely.
What to Expect When Joining
STEP 1
STEP 2
STEP 3
Initial Testing
This will occur at the study site and
includes ECHO, cardiac MRI, heart
biopsy and additional lab assessments.
Treatment & Monitoring
The patient will receive pre-treatment to prepare the immune system. The patient will be hospitalized for gene therapy treatment and for safety monitoring
which will continue as outpatient.
Follow-Up
The patient will return to the clinical
site for ongoing monitoring over
the course of 5 years.
Frequently Asked Questions
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A genetic test, ordered by a healthcare provider, can detect whether there is a damaged or missing LAMP 2 gene.
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A natural history study is also called an “observational study” because patients do not receive any treatment. Researchers observe and collect information, including lab assessments, about a condition. People in the study stay on the treatments they already use and will continue to see their doctor for regular care.
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First, fill out and submit the form with the basic information. The support team will then contact you to review the information and discuss the study’s requirements. If the patient meets the criteria, the support team will connect you with a clinical study site.
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Yes. If the patients joins the Natural History study and is interested in learning more about the Gene Therapy study, they should speak with their Natural History study Principal Investigator to receive a referral to the Gene Therapy study team.
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The costs associated with study visits and treatment, including travel, lodging, and meals are covered by the study.
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Participation is voluntary, and up to you and your family. You can withdraw permission to participate in the Natural History study, and discontinue the process of screening and testing for the gene therapy at any time. This will not impact your regular clinical care.
Ready to Join?
Fill out the form here so we can contact you with more information about joining our LAMPLIGHT Danon disease studies