Natural History Study for Danon Disease

Recruiting at 15 trial locations

Overseen By Clinical Information

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Rocket Pharmaceuticals Inc.

Must not be taking: Investigational agents

Disqualifiers: Gene therapy, Age, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand the progression of Danon disease and its impact on the heart and overall quality of life. Researchers focus on tracking changes in heart structure, symptoms, and major health events such as heart failure hospitalization or the need for a heart transplant. The study examines both current and past medical records. It is best suited for males over 8 years old and females under 30 diagnosed with a genetic variant linked to Danon disease. As an unphased trial, this study offers a unique opportunity to contribute to the understanding of Danon disease and potentially improve future treatments.

What prior data suggests that this observational study is safe?

The study aims to better understand Danon disease by observing its effects over time. It does not test any new treatments or drugs, so no new safety information is available. The focus is on collecting data about symptoms, heart health, and quality of life in individuals with Danon disease. This data helps researchers learn more about the condition itself. Participants will not receive any new medications or therapies as part of this study. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the Danon Disease Natural History Study because it aims to uncover valuable insights into the progression of Danon Disease, a rare genetic disorder. Unlike current treatments, which primarily focus on managing symptoms, this study seeks to establish a comprehensive understanding of how the disease develops over time. By analyzing both prospective and retrospective data, researchers hope to identify new patterns and potential therapeutic targets. This could pave the way for the development of more effective and targeted treatments in the future.

Are You a Good Fit for This Trial?

Inclusion Criteria

You had a heart transplant or mechanical heart support in the past 2 years.

You have a specific genetic change in the LAMP2 gene confirmed by a certified genetic testing lab.

We need to see your medical records from at least 30 days before you had a heart transplant or mechanical circulatory support.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Retrospective Data Collection

Collection of historical data to describe the natural history of Danon disease

Varies

Prospective Data Collection

Ongoing collection of data to assess changes in cardiac structure, biomarkers, symptoms, and quality of life

24 months

Regular visits every 6 months

Follow-up

Participants are monitored for safety and effectiveness after data collection

6 months

What Are the Treatments Tested in This Trial?

How Is the Trial Designed?

Group I: Retrospective Cohort Experimental Treatment 1 Intervention

Group II: Prospective Cohort Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Rocket Pharmaceuticals Inc.

Lead Sponsor

Trials

17

Recruited

430+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06214507

NCT06214507 | Danon Disease Natural History Study

This study will collect data about the clinical course of Danon disease including signs and symptoms, key clinical events, and the impact of the disease on ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT03766386

NCT03766386 | The Natural History of Danon Disease

This study aims to determine the natural history of DD through the collection and analysis of retrospective and prospective data. ... data for this study ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12542076/

Danon disease in male patients: a prospective natural ...

The multisystemic longitudinal data captured in this natural history study provide a more comprehensive characterization of the clinical ...

4.

rocketpharma.com

rocketpharma.com/our-science/natural-history-studies/danon-disease/

Natural History Danon Disease

Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart. An open-label, global Phase 2 ...

5.

jacc.org

jacc.org/doi/10.1016/j.jacc.2023.08.014

International Consensus on Differential Diagnosis and ...

Herein, the authors review the differential diagnosis of Danon disease, diagnostic criteria, natural history, management recommendations, and recent advances in ...