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.

Patients & Caregivers:

Fanconi Anemia Resources

FA Clinical Trials Resources

FAQs about Rocket’s Gene Therapy Approach to FA

FA is a rare DNA repair disorder characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. Approximately two-thirds of FA cases are caused by genetic defects in the FANCA gene, which results in the FA subtype known as FA Complementation Group A (FA-A).

Blood stem cells collected from an FA-A patient are genetically modified to introduce an intact copy of the FANCA gene using a virus that has been changed in the laboratory so that it cannot grow or spread to cause an infection. The genetically modified cells are then returned back into the patient.

FA-A patients who have not developed severe BMF, do not have a human leukocyte antigen (HLA)-matched sibling donor for bone marrow transplant, and are not receiving other experimental therapies. Enrollment for the Phase 1 & 2 clinical trials for FA-A are closed.

Before beginning the study, a patient needed to have several tests to determine whether he or she was eligible to join the trial. These tests can take up to 1 month to complete. After the doctors conducting the study confirm that a patient is eligible for the trial, participation in the study involved:

  • Stem Cell Collection: Stem cells have the potential to make different blood cells in the body. Patients will receive medications that make stem cells in their bone marrow enter the blood, where they will be collected from the patient’s vein (most likely using a large, temporary catheter) during a procedure called apheresis.
  • Infusion of Genetically Modified Stem Cells (the Investigational Gene Therapy): The patient’s stem cells will be genetically modified in a laboratory to introduce the intact copy of the FANCA gene. The patient will then receive an infusion of the genetically modified cells through the intravenous catheter. There is no conditioning chemotherapy before the infusion.
  • Follow-up after Administration of the Investigational Gene Therapy: Patients will need to return for follow-up visits, including blood and bone marrow tests, over the next 3 years. In addition, patients will have long-term follow-up approximately 1-2 times per year for another 12 years.

Financial support, including travel arrangements and housing accommodations for patients and a family member, both for the treatment and follow-up visits, are provided.

  • The Center for Definitive and Curative Medicine at Stanford University, California, USA
  • Hospital Infantil Universitario Niño Jesús, Madrid, Spain
  • University College London, Great Ormond Street Hospital for Children (UCL GOSH), London, UK
  • University of Minnesota, Minnesota, USA

To read our Expanded Access statement, click here.

Learn more about Rocket’s clinical trial for FA

For additional resources, we encourage you to reach out to the groups below.

If your group would like to be included in our list of patient resources, please reach out to us at patients@nullrocketpharma.com.
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