Philadelphia, PANCT06092034Now EnrollingIRB Ready

Danon Disease Clinical Trial in Philadelphia, PA

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

Sponsored by Rocket Pharmaceuticals Inc.

Quick Self-Assessment

See if you qualify for this Philadelphia location

Preparing your pre-screening questions…

Expert Care in Philadelphia

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 Philadelphia Location

Check if you qualify for this danon disease clinical trial in Philadelphia, PA

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

Why Participate?

  • No-Cost Study Care

  • Local to Philadelphia

    Convenient for PA 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 Philadelphia site if eligible
  4. 4Begin participation

About This Danon Disease Study in Philadelphia

This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.

Sponsor: Rocket Pharmaceuticals Inc.

Who Can Participate

Inclusion Criteria

Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
Age ≥8 years.
Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
Abnormal thickening of Left ventricular wall,
Left ventricular ejection fraction (LVEF) ≥ 50%.
New York Heart Association (NYHA) Class II to III.
High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)
Ability to comply with study procedures including investigational therapy and follow-up evaluations. Key

Exclusion Criteria

Anti-AAV9 neutralizing antibody titer \>1:40.
Severe heart failure or requirement for advanced therapies.
History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
Prior cardiac or other organ (lung, liver, other) transplantation.

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 Philadelphia?

Yes, this clinical trial (NCT06092034) has an active research site in Philadelphia, PA 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 Philadelphia, PA

If you're searching for danon disease treatment options in Philadelphia, PA, this clinical trial (NCT06092034) 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 Philadelphia 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 · Philadelphia, PA