Condition
Danon Disease clinical trials and research
As of November 12, 2026, Find a Trial lists 4 Danon Disease clinical trials that are recruiting, at sites in 8 countries (most in United States (3), Germany (2), Italy (2)). By phase: Phase 2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 4Clinical trials
- 8Countries
Newest Danon Disease clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Using advanced heart MRI to improve diagnosis and risk prediction for rare cardiomyopathies | — | Beijing | Recruiting | Jan 13, 2026 |
| Natural history study of rare glycogen storage diseases | — | Durham, North Carolina | Recruiting | Jan 27, 2025 |
| Observing the natural history of Danon disease | — | Phoenix, Arizona and 15 other locations | Recruiting | Jan 19, 2024 |
| RP-A501 gene therapy for males with Danon disease | Phase 2 | La Jolla, California and 5 other locations | Recruiting | Oct 23, 2023 |
All recruiting trials (4)
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Using advanced heart MRI to improve diagnosis and risk prediction for rare cardiomyopathies
This project tests whether advanced cardiac MRI techniques can help diagnose and predict future risk for people with rare heart muscle diseases like Danon, Fabry, cardiac amyloidosis, Noonan syndrome, and cardiac sarcoidosis.
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Natural history study of rare glycogen storage diseases
This study is collecting health information from people with rare glycogen storage diseases to better understand their conditions and help improve future treatments.
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Observing the natural history of Danon disease
This study looks at how Danon disease affects boys over 8 and girls under 30 over time to see changes in their heart health, symptoms, and quality of life.
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RP-A501 gene therapy for males with Danon disease
This gives a single IV dose of RP-A501 gene therapy to males aged 8 and older with Danon disease to see if it improves heart structure and symptoms.
Related topics
Quick answers
How many Danon Disease clinical trials are recruiting right now?
4, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 8 countries. The most sites are in United States (3 trials), Germany (2 trials), Italy (2 trials), United Kingdom (2 trials), China (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.