RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart.

An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. and EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months.

Phase 2 Trial Sites

For more details about the RP-A501 for Danon Disease clinical trial, visit clinicaltrials.gov.

Frequently Asked Questions

The Natural History of a Disease is the typical progression of the disease over a person’s lifetime starting from its onset

Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time. This information helps describe the disease course and identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes.

Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge.

Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience.

Engaging in a Natural History Study offers numerous benefits to the community, such as:

  • Raising awareness in medical, academic and pharmaceutical sectors
  • Empowering rare disease patient communities with knowledge and support for advocating better healthcare policies and support services
  • Enhancing patient care with improved insights into disease progression, allowing for more personalized treatment plans and potentially earlier detection of complications
  • Contributing to advancements in healthcare

These studies provide various avenues to help inform and design clinical trials, such as:

  • Highlighting areas of unmet medical need
  • Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective.
  • Describing disease progression, enabling the identification of clinical outcome measures and biomarkers
  • Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments.
Learn more about Rocket’s clinical trial for FA

Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com

View more details about the Phase 2 pivotal trial of RP-A501 and natural history study on clinicaltrials.gov.

To read our Expanded Access statement, click here.

Dilated Cardiomyopathy (DCM) Resources

Patients & Caregivers:

Dilated Cardiomyopathy (DCM) Resources

Dilated cardiomyopathy, DCM, is the most common form of cardiomyopathy or heart failure and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that are unable to pump blood. Mutations in the BAG3 gene are among the more common mutations observed in DCM, otherwise known as BAG3-associated DCM (BAG3-DCM).

The BAG3 protein is expressed predominantly in the heart, where it plays a role in multiple key cellular functions of the heart. Loss of BAG3 leads to an accumulation of misfolded and damaged proteins, which can impair the heart’s ability to contract, leading to impaired cardiac function, heart failure, and even premature death.

Patients living with BAG3-DCM have an urgent unmet medical need, as current medical and interventional therapies (including implantable cardioverter defibrillator [ICD], pacemakers, and heart transplant) do not consistently prevent disease progression, are associated with significant morbidity including inappropriate ICD shocks and device, procedure, and transplant-related complications, and do not address the underlying pathophysiology or genetic mutation.

Learn more about Rocket’s clinical trial for BAG3-DCM

For additional resources and more information, check out:

If your group would like to be included in our list of patient resources, please reach out to us at [email protected].
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