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Retrospective only for Danon Disease

N/A
Recruiting
Research Sponsored by Rocket Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6, 12, 18 and 24 months
Awards & highlights

Study Summary

The goal of this international observational study is to learn about the natural history of Danon disease in male patients >8 years of age. The key objectives include assessing change over time in cardiac structure (left ventricular mass and wall thickness), cardiac biomarkers, symptoms, and quality of life and the incidence of clinical events such as HF hospitalization, cardiac transplantation, and death.

Eligible Conditions
  • Danon Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6, 12, 18 and 24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6, 12, 18 and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Left Ventricular Mass Index (LVMI) by echocardiogram
Secondary outcome measures
Cardiac biomarkers (NT-proBNP, BNP)
Cardiac biomarkers (hsTnI)
Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization
+3 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Retrospective onlyExperimental Treatment1 Intervention
Group II: ProspectiveExperimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
14 Previous Clinical Trials
334 Total Patients Enrolled
4 Trials studying Danon Disease
278 Patients Enrolled for Danon Disease

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Danon Disease Natural History Study
2023. "Danon Disease Natural History Study". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06214507.
All > Paid Studies San Diego > Colorado Springs
~31 spots leftby Aug 2026
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