RECRUITING

Danon Disease Natural History Study

Talk to us

Conditions

Danon DiseaseLAMP2Lysosome-associated membrane protein 2CardiomyopathyX-linked

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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).

Official Title

An Observational Study of Genetic Cardiomyopathy, Danon Disease

Quick Facts

Study Start:2023-12-20
Study Completion:2030-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06214507

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:8 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. 1. 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
  2. 2. Previous treatment with a gene therapy
  3. 3. Prior mechanical circulatory support at time of enrollment to this study
  4. 4. Prior cardiac transplantation at time of enrollment to this study
  5. 5. Age \>51 years at enrollment

Contacts and Locations

Study Contact

Clinical Information
CONTACT
646-627-0033
clinicaltrials@rocketpharma.com

Study Locations (Sites)

Phoenix Children's Hospital
Phoenix, Arizona, 85016
United States
University of California, San Diego
La Jolla, California, 92037
United States
Children's Hospital Colorado
Aurora, Colorado, 80045
United States
University of Florida College of Medicine
Gainesville, Florida, 32610
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
Washington University
St Louis, Missouri, 63110
United States
Columbia University Medical Center/New York Presbyterian
New York, New York, 10032
United States
Texas Children's Hospital
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Rocket Pharmaceuticals Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-12-20
Study Completion Date2030-03

Study Record Updates

Study Start Date2023-12-20
Study Completion Date2030-03

Terms related to this study

Keywords Provided by Researchers

  • Danon Disease
  • LAMP2
  • Lysosome-associated membrane protein 2
  • Cardiomyopathy
  • X-linked

Additional Relevant MeSH Terms

  • Danon Disease