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 2023

Rocket Pharma - Rare Disease Day Logo
show your stripes - zebra

ROCKET PHARMA CELEBRATES RARE DISEASE DAY 2023

STORIES THAT NEED TO BE SHARED: THE HUMAN SIDE OF RARE DISEASE

Our mission is to seek gene therapy cures for people living with rare diseases, who are our North Star. In celebration of Rare Disease Day 2023, Rocket held an event at the Make-A-Wish New Jersey Samuel & Josephine Plumeri Wishing Place in Monroe Township, NJ. 

Resembling a fairy tale castle and dedicated to granting the wishes of children with critical illnesses, the Make-A-Wish venue provided a meaningful backdrop to the event that brought together more than 200 people and featured inspirational stories from the global rare disease community.

Welcoming Remarks

Kinnari Patel, PharmD, MBA, President & Chief Operating Officer, Rocket Pharma

Event Overview: Stories that Need to Be Shared: The Human Side of Rare Diseases

Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma

Ensuring Patient Access to Rare Disease Treatments

Claire Booth, MBBS PhD, Prof. Gene Therapy and Pediatric Immunology, University College of London (UCL), Great Ormond Street Institute of Child Health, Co-founder, AGORA (Access to Gene Therapies for Rare Disease)

Patient & Caregiver Perspective: Arrhythmogenic Cardiomyopathy (ACM)

Moderator: Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma

Speaker Panel: Maddie and Michelle

Raising Awareness of Sudden Arrhythmia Death Syndromes (SADS)

Alice Lara, RN, President and CEO, SADS Foundation

Patient & Caregiver Perspective: Pyruvate Kinase Deficiency (PKD)

Moderator: Gayatri Rao, M.D., J.D., Chief Development Officer, LVV, Rocket Pharma

Speaker Panel: Travis and Jessica

Patient Advocacy Video Resources

“Happy Rare Disease Day” from Shaping Foundations

Thrive with Pyruvate Kinase Deficiency (PKD)

Pyruvate Kinase Deficiency Foundation (PKDF)

Closing Remarks

Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma

For any questions, please reach out to [email protected]

Rocket Rare Disease Day 2022
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