New York, NYNCT06214507Now EnrollingIRB Ready

Danon Disease Clinical Trial in New York, NY

Access cutting-edge danon disease treatment through this clinical trial at a research site in New York. Study-provided care at no cost to qualified participants.

Sponsored by Rocket Pharmaceuticals Inc.

Quick Self-Assessment

See if you qualify for this New York location

Preparing your pre-screening questions…

Expert Care in New York

Access danon disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related danon disease treatment provided free

Apply for This New York Location

Check if you qualify for this danon disease clinical trial in New York, NY

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to New York

    Convenient for NY residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit New York site if eligible
  4. 4Begin participation

About This Danon Disease Study in New York

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).

Sponsor: Rocket Pharmaceuticals Inc.

Who Can Participate

Inclusion Criteria

Prospective Cohort:
Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
Patient or parent/legal guardian are capable and willing to provide signed informed consent
Age ≥ 8 years at enrollment Female Prospective Cohort:
Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment. Retrospective (only) Cohort:
Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
Patient or parent/legal guardian are capable and willing to provide signed informed consent, as required by local regulations
Age ≥ 8 years at enrollment
Prior cardiac transplantation or prior mechanical circulatory support
At least 30 days of retrospective medical records available prior to cardiac transplantation or mechanical circulatory support Female Retrospective (only) Cohort:
Prior evidence of left ventricular hypertrophy. Key Exclusion Criteria: 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

Exclusion Criteria

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

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in New York?

Yes, this clinical trial (NCT06214507) has an active research site in New York, NY that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Danon Disease Treatment Options in New York, NY

If you're searching for danon disease treatment options in New York, NY, this clinical trial (NCT06214507) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our New York research site is actively enrolling participants for this clinical trial. You'll receive care from experienced danon disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all danon disease clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · New York, NY