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Natural History of Danon Disease

NCT05548855 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 59

Last updated 2023-11-21

No results posted yet for this study

Summary

This is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients with Danon disease (DD). This study is composed of 2 parts:

* Feasibility study: to identify participating sites, assess site and team capabilities, confirm the site and investigator qualification for taking part in the study,
* Retrospective chart review: Data will be collected retrospectively by means of a chart review of living and/or deceased DD patients with a confirmed lysosome associated membrane protein-2 gene (LAMP2) mutation, and,
* For living patients (who have not undergone heart transplantation or placement of a cardiac assist device), availability of at least 6-month cardiology follow-up data,
* For living patients who underwent heart transplantation or placement of a cardiac assist device, and for deceased patients, at least 1 MRI or echo assessment prior to heart transplantation/cardiac assist device placement or death.

Conditions

  • Danon Disease

Interventions

PROCEDURE

Heart Transplant

Patients who have undergone a heart transplant as standard of care (SOC) procedure

DEVICE

Cardiac Assistive Device

Patients who have undergone a placement of a cardiac assistive device

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-09-26
Primary Completion
2023-10-31
Completion
2023-10-31

Countries

  • Czechia
  • France
  • Ireland
  • Italy
  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05548855 on ClinicalTrials.gov