Danon Disease Natural History Study

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Rocket Pharmaceuticals Inc.
Study ID
NCT06214507
Status
Recruiting
Apply to this trial

Conditions

  • Danon Disease

Eligibility Criteria

Sex
ALL
Age
8 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • No intervention — OTHER
    No intervention

Study Details

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).

Key Dates

Start date
Dec 20, 2023
Status verified
Apr 2026
Primary completion
Dec 31, 2029
Completion
Mar 31, 2030

Study Design

Enrollment
60 participants (estimated)

Arms

  • Arm: Prospective Cohort
  • Arm: Retrospective Cohort

Primary Outcome Measure

Left Ventricular Mass Index (LVMI) by echocardiogram [ Time Frame: Month 12, 24, 36 ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016
Andrew Papez, MD
[email protected]
(602) 546-0512
Sanjana Khanna
[email protected]
6029334482
Andrew Papez, MD (PRINCIPAL_INVESTIGATOR)
University of California, San DiegoLa JollaCalifornia92037
Kim Hong, MD
[email protected]
(858) 246-2996
Kimberly Hong, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital ColoradoAuroraColorado80045
Matthew Taylor, MD, PhD
[email protected]
(720) 848-1030
Matthew Taylor, MD (PRINCIPAL_INVESTIGATOR)
University of Florida College of MedicineGainesvilleFlorida32610
Mohammed M Ahmed, MD
[email protected]
(352) 273-7770
Mohammed M Ahmed, MD (PRINCIPAL_INVESTIGATOR)
University of MichiganAnn ArborMichigan48109
Supriya Shore
[email protected]
Supriya Shore, MD (PRINCIPAL_INVESTIGATOR)
Josh Meisner, MD (SUB_INVESTIGATOR)
Washington UniversitySt LouisMissouri63110
Charles Canter, MD
[email protected]
314-286-2404
Teresa Roberson
[email protected]
314-302-7209
Charles Canter, MD (PRINCIPAL_INVESTIGATOR)
Columbia University Medical Center/New York PresbyterianNew YorkNew York10032
Farhana Latif
[email protected]
(212) 305-3172
Jessica Idumonyi
[email protected]
Farhana Latif, MD (PRINCIPAL_INVESTIGATOR)
Texas Children's HospitalHoustonTexas77030
Kyle Hope, MD
[email protected]
(832) 822-4280
David Garuba
[email protected]
8328265622
Kyle Hope, MD (PRINCIPAL_INVESTIGATOR)

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Phoenix Children's Hospital· Phoenix, AZUniversity of California, San Diego· La Jolla, CAChildren's Hospital Colorado· Aurora, COUniversity of Florida College of Medicine· Gainesville, FLUniversity of Michigan· Ann Arbor, MIWashington University· St Louis, MO

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