Diagnosed with Danon Disease?
Research 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
Natural history study to collect the medical history and day-to-day experiences of people with Danon Disease. No treatment in this study.
Interventional study for Danon Disease, with the 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 help’s 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, 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
Steps to Participate in 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 you 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.
On certain cases this step may be
done remotely.
STEP 4
Initial Testing
This will occur at the study site and
includes ECHO, cardiac MRI, heart
biopsy and additional lab assessments.
STEP 5
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
STEP 6
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 geneticist or another specialist, 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 researchers observe and collect information about a condition. Unlike a clinical study, no new medicine is given. People in the study stay on the treatments they already use.
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First, fill out and send in the form with the basic information needed to see if the child or teen meets the study’s requirements. If they do, the support team will contact you and connect you with a study site. Then, the study site coordinator will talk with you in a longer interview to see if the study is a good fit.
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Yes. If the person you care for joins the Natural History study and is able and willing to join the Gene Therapy study, you can apply for a prescreening interview with the LAMPLIGHT Gene Therapy team. The study site will make the final decision about whether they can join the Gene Therapy study.
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The costs of the therapy and visits will be covered by the study sponsor, as is travel to the study sites.
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Participation is completely up to you and your family. You can withdraw permission to access records from the Natural History study, and discontinue the process of screening and testing for the gene therapy without any problem to you.
Ready to Join?
Fill out the form here so we can contact you with more information about joining our LAMPLIGHT Danon disease studies