NCT06214507 · Rocket Pharmaceuticals Inc.
Danon Disease Natural History Study
What this study is about
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
View original scientific description
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Prospective Cohort:
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
- Patient or parent/legal guardian are capable and willing to provide signed informed consent
- Age ≥ 8 years at enrollment Female Prospective Cohort:
- Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment. Retrospective (only) Cohort:
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
- Patient or parent/legal guardian are capable and willing to provide signed informed consent, as required by local regulations
- Age ≥ 8 years at enrollment
- Prior cardiac transplantation or prior mechanical circulatory support
- At least 30 days of retrospective medical records available prior to cardiac transplantation or mechanical circulatory support Female Retrospective (only) Cohort:
- Prior evidence of left ventricular hypertrophy. Key Exclusion Criteria: All Cohorts:
- Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
- Previous treatment with a gene therapy Prospective Cohort:
- Prior mechanical circulatory support at time of enrollment to this study
- Prior cardiac transplantation at time of enrollment to this study Female patients:
- Age \>51 years at enrollment
Exclusion criteria
- All Cohorts:
- Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
- Previous treatment with a gene therapy Prospective Cohort:
- Prior mechanical circulatory support at time of enrollment to this study
- Prior cardiac transplantation at time of enrollment to this study Female patients:
- Age \>51 years at enrollment
Where
- Phoenix, Arizona
- La Jolla, California
- Aurora, Colorado
- Gainesville, Florida
- Ann Arbor, Michigan
- St Louis, Missouri
- New York, New York
- Houston, Texas
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced May 5, 2026 · Source of record for eligibility and locations