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.

RARE DISEASE DAY 2021

Rocket Rare Disease Logo
Rare Disease Day Logo
Rare Disease Day takes place every year on the last day of February—with February 29th as the rarest date on the calendar—to underscore the nature of rare diseases and what patients and families face.

ROCKET RARE DISEASE MONTH 2021: Expert Panel Series

Abbey Meyers: A Parent’s Quest to Make a Difference in Rare Diseases

February 5, 2021 11 a.m. – 12:30 p.m. EST
Join us for an intimate discussion with Abbey Meyers, catalyst for the Orphan Drug Act of 1983 and National Organization for Rare Disorders (NORD) founder. Abbey’s indelible story as a parent-turned-advocate shows how a parent’s quest to change her child’s life would ultimately improve respect, understanding and care for rare disease patients around the world in the decades since.

Revealing the Power of Genetic Testing

February 12, 2021 11 a.m. – 12:30 p.m. EST

Early detection of rare diseases through genetic testing may lead to the best possible outcomes for patients and those around them. However, making this decision isn’t always easy. Learn the ABCs of genetic testing from patient advocates Seth Rotberg and Ron Gladue, and genetic counselor Amy Jonasson. Topics will include their personal journeys and practical advice for patients and families including types, access, and timing of genetic tests, and benefits of testing.

Beyond ClinicalTrials.gov – The Clinician’s Perspective in a Gene Therapy Study

February 19, 2021 11 a.m. – 12:30 p.m. EST
Gene therapy clinical trials are on the rise and aim to fill of the unmet need for many rare diseases that are void of approved treatment. Deciding to volunteer for a gene therapy trial can be daunting. Join study physicians Claire Booth, Julian Sevilla Navarro and Ami Shah as they share insight on how to navigate this decision by discussing some of the common challenges and considerations for gene therapy clinical trials.

How to Go from Caring for 1 to Caring for a Community

February 26, 2021 11 a.m. – 12:30 p.m. EST

In rare disease, the magnitude of ‘being your own best advocate’ is astronomical. Given that the sheer rarity of some diseases means a Google search turns up a ‘zero’ result, taking the initiative to unearth even the most basic information and resources to get the best possible care can become an obsession. As successful advocates explain, fostering this obsession into something that benefits a patient community can be immensely rewarding. Join our panel to learn how they have built supportive communities and the lessons learned along the way.

For any questions please reach out to [email protected]

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