NCT06214507

Brief 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) and female patients (8 to 50 years of age).

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at P25-P50 for all trials

Timeline
46mo left

Started Dec 2023

Longer than P75 for all trials

Geographic Reach
6 countries

13 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress38%
Dec 2023Mar 2030

First Submitted

Initial submission to the registry

November 17, 2023

Completed
1 month until next milestone

Study Start

First participant enrolled

December 20, 2023

Completed
1 month until next milestone

First Posted

Study publicly available on registry

January 19, 2024

Completed
5.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2029

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2030

Last Updated

May 5, 2026

Status Verified

April 1, 2026

Enrollment Period

6 years

First QC Date

November 17, 2023

Last Update Submit

April 29, 2026

Conditions

Danon Disease

Keywords

Danon DiseaseLAMP2Lysosome-associated membrane protein 2CardiomyopathyX-linked

Outcome Measures

Primary Outcomes (1)

  • Left Ventricular Mass Index (LVMI) by echocardiogram

    Evaluation of change over duration of follow up

    Month 12, 24, 36

Secondary Outcomes (16)

  • Cardiac biomarkers (hsTnI)

    Month 12, 24, 36 (when available)

  • Cardiac biomarkers (NT-proBNP, BNP)

    Month 12, 24, 36 (when available)

  • NYHA class

    Month 12, 24, 36

  • KCCQ-12

    Month 12, 24, 36

  • PedsQL

    Month 12, 24, 36

  • +11 more secondary outcomes

Study Arms (2)

Prospective Cohort

Other: No intervention

Retrospective Cohort

Other: No intervention

Interventions

No interventionOTHER

No intervention

Prospective CohortRetrospective Cohort

Eligibility Criteria

Age8 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Male patients 8 years of age and over, Female patients 8 to 50 years of age (at the point of enrollment) with confirmed Danon disease recruited from a range of applicable care settings.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

University of California, San Diego

La Jolla, California, 92037, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

University of Florida College of Medicine

Gainesville, Florida, 32610, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Washington University

St Louis, Missouri, 63110, United States

RECRUITING

Columbia University Medical Center/New York Presbyterian

New York, New York, 10032, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

German Heart Center Munich

München, Germany

RECRUITING

Sheba Medical Center

Ramat Gan, 5265601, Israel

RECRUITING

Azienda Ospedaliera Universitaria Meyer IRCCS

Florence, 50139, Italy

RECRUITING

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, Madrid, Spain

RECRUITING

University College London - Great Ormond Street Institute of Child Health

London, WC1N 1DZ, United Kingdom

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type IIbCardiomyopathies

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesHeart DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Clinical Information

CONTACT

646-627-0033clinicaltrials@rocketpharma.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 17, 2023

First Posted

January 19, 2024

Study Start

December 20, 2023

Primary Completion (Estimated)

December 1, 2029

Study Completion (Estimated)

March 1, 2030

Last Updated

May 5, 2026

Record last verified: 2026-04

Locations

IT(1)US(8)IL(1)GB(1)DE(1)ES(1)