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.

Privacy Policy

Privacy Policy

Rocket Pharmaceuticals, Inc. (Rocket) and its subsidiaries and affiliates recognize the importance of protecting your privacy. This Privacy Policy (Policy) discloses our information-gathering and dissemination practices for this website at rocketpharma.com.

By using this Website, you agree to be bound by the terms and conditions of this Policy and the Terms of Use. If you do not agree with the terms of this Policy or the Terms of Use, or other terms and conditions on this Website, then do not use this Website. You may also be subject to another privacy policy or other terms and conditions that are posted on Rocket’s website specific to a particular Rocket program or activity (Other Agreement). Based upon your specific relationship(s) with Rocket we may collect, use or disclose Personal Information (defined below) in ways described in more detail in one or more Other Agreements. To the extent the Other Agreement includes additional terms that are not covered by or are inconsistent with this Policy, the terms and conditions of the Other Agreement will supersede this Policy.

Within this Policy you will find covered matters related to the following:

  • Children
  • Information We Collect
  • Personal Information
  • Non-Personal Information
  • Cookies
  • Third-Party Advertisers
  • How We Use Your Personal Information
  • Disclosure of Your Personal Information
  • Choice to Opt-Out and Access to Your Information
  • Links to Third Party Sites
  • Security
  • Changes
  • Governing Law and Jurisdiction
  • Contact Information
  • Special Notice to California Residents
  • Do Not Track

CHILDREN

This Website is not directed at children and we do not knowingly collect Personal Information (as defined below) online from children under the age of 13. If we were to receive Personal Information from a child under the age of 13, we would delete it.

INFORMATION WE COLLECT

Personal Information

We collect and store Personal Information that you provide voluntarily when using this Website. Personal Information is information that can identify you as a specific individual (Personal Information). You are responsible for providing accurate and complete information. For example, you might provide us the following information: information requests, business proposals, employment inquiries, responses and survey information, site registrations or web portal registration information. Personal data does not include anonymized aggregate or statistical information from which such identification cannot be reconstituted.

Other Information

When you visit any page on this Website, we use a program that monitors and evaluates this Website’s traffic. It automatically records every visitor’s host, domain name, pages visited, length of user session, browser type and/or IP address. We use this information to find out how many people visit this Website to determine the areas of greatest interest on this Website, and to make improvements to this Website.

When you visit and interact with this Website, Rocket and third parties with whom Rocket has contracted to provide services to Rocket, may collect non-personal information (for example, a catalog of the site pages you visit, or your IP address).

Cookies

As with many other sites, this Website may use cookies or other technologies to help us deliver content specific to your interests, to process your inquiries or requests, and/or to analyze your visiting patterns. Cookies, by themselves, will not be used by Rocket to disclose your individual identity. This information identifies your browser, but not you, to our servers when you visit this Website. If you want to disable the use of cookies or remove them at any time from your computer, you can disable or delete them using your browser (consult your browser’s help menu to learn how to delete cookies), however you must enable cookies to access certain information on this Website.

Third-Party Advertisers

In addition, we may use third-party advertisers to serve or track advertisements on other websites, to which this Privacy Policy does not apply (those websites are subject to their own terms of use and privacy policies). Please note that the third-party advertisers that we may use also may use cookies, pixel tags and other technologies to, among other things, enable such third-party advertisers to record which ads your browser has loaded and which site pages you may have been viewing when the ads were delivered or accessed. To learn more about the functions of such third-party advertisers, you may visit www.AboutAds.info.

HOW WE USE YOUR PERSONAL INFORMATION

We may use your Personal Information to:

  • Complete any registration or other transactions you request online;
  • Improve this Website;
  • Personalize your experience on this Website, and customize the content and/or format of the pages you visit;
  • Notify you about news from Rocket, its affiliates, and selected third parties;
  • Ask you to participate in brief surveys; and/or
  • Generate aggregate statistical studies and conduct research related to our products and services and the use of this Website.

Based upon your specific relationship(s) with Rocket, we may use your Personal Information in ways described in more detail in one or more Other Agreements.

In some instances, we may combine your Personal Information with other information (such as embedding e-mail addresses in cookies). If we do combine any non-personal information with Personal Information, the combined information will be treated by us as Personal Information in accordance with this Policy.

DISCLOSURE OF YOUR PERSONAL INFORMATION

We disclose your Personal Information to certain third parties with whom we have a direct or indirect business or contract relationship, to provide the products and services you have requested.

When you request other services, we will provide your personal data to third parties with whom we arrange for those services to be provided. We may also disclose information to third parties that provide administrative or processing services for us, for example, printing or mailing. Once these third-party service providers receive your Personal Information from us, their use of that information to provide services to you is subject to the terms of use and privacy policies of those third parties.

We may also disclose Personal Information collected on this Website, in accordance with applicable law, to assert or defend our rights and property, to prevent harm to others, to collect a debt, or in response to legal processes such as subpoenas. We may also provide (or reserve the right to provide) Personal Information to credit reporting agencies and to cooperate with law enforcement authorities and other governmental authorities consistent with applicable laws.

If you have specifically consented to our doing so, we may also share your personal data with third parties in order that they may offer you information about their goods and services.

Based upon your specific relationship(s) with Rocket, we may disclose your Personal Information in ways described in more detail in one or more Other Agreements.

LINKS TO THIRD PARTY SITES

This Website may contain links to other third party websites. Rocket does not control the privacy policies or practices of these third-party sites. You should review those policies before providing any information. Rocket is not responsible for the content or practices of any linked third-party sites, and we provide these links solely for the convenience and information of our visitors.

SECURITY

Rocket has established safeguards to help prevent unauthorized access to or misuse of your Personal Information, but cannot guarantee that your Personal Information will never be disclosed in a manner inconsistent with this Policy (for example, as a result of unauthorized acts by third parties that violate applicable law or relevant Web policies).

CHANGES

Rocket reserves the right to change this Policy to respond to new laws, regulations, technology, or for other business reasons. Please check this Website from time to time to review any such changes. Using this Website following the posting of changes to this Policy shall constitute your acceptance of the revised Policy.

GOVERNING LAW AND JURISDICTION

You agree that any claim, action or proceeding arising out of this Policy, or your use of this Website, shall be governed by and construed in accordance with the laws of the United States and the State of New York, without regard to principles of conflict of laws. Any claim, action or proceeding related to this Policy will be resolved exclusively in the state or federal courts located in the State of New York and you consent to the jurisdiction of those courts.

CONTACT INFORMATION

If you have any questions or concerns about this Website or security policies, you may contact us by e-mail at [email protected].

DO NOT TRACK

Although our Website currently does not have a mechanism to recognize the various web browser Do Not Track signals, we do offer you choices to manage your preferences as described in the sections above. To learn more about browser tracking signals and Do Not Track please visit http://www.allaboutdnt.org.

See also: Terms of Use

This Privacy Policy is effective as of: December 4, 2020

Cookie Policy

This Cookies Policy forms part of our general Privacy Policy.

Read full Privacy Policy.

Information We Automatically Collect When You Visit Our Site

We monitor traffic on our site and measure which pages are visited in order to improve the Site and to provide information tailored to the needs and interests of our visitors. During your visit to our Site, we automatically collect certain information as described below:

Cookies:

When you visit our Site, a cookie (a small text file) is stored on your computer’s hard drive. We use cookies to record non-personal information about visitors to our Site in order to recognize you as a return visitor the next time you visit, so we can enhance and customize your experience on our Site. Cookies record the type of browser, operating system, and Internet service provider and allow us to tabulate the total number of visitors to our site. We do not use these cookies to run programs or deliver software to your computer. You may disable the cookie feature on your computer or set it to alert you when cookies are being sent by using the preferences on your browser. If you choose to disable cookies, however, you may be unable to access some parts of our Site and certain features (e.g., customized delivery of information) available on our Site.

Log files:

We also collect certain technical information from your computer each time you request a page during a visit to our Site and store it in log files. This information may include your Internet Protocol (IP) address, your computers operating system, browser type, the addresses of pages you visit before and after visiting our Site, the date and time of your visit, and which pages you view during your visit to the Site.

Clear Gifs:

The Site also uses a software technology called clear gifs (also known as web beacons or web bugs), which are tiny graphics that we use to record which pages are viewed by a Site visitor. In contrast to cookies, which are stored on a computer’s hard drive, clear gifs are embedded invisibly on web pages as they are displayed and are about the size of the period at the end of this sentence. Clear gifs do not contain any personally identifiable information, and we do not use them to identify individual visitors.

Third-Party Cookies

In addition to our own cookies, we work with various reputable companies and vendors to help us analyze website traffic and to optimize our Website. The following companies help us achieve this through use of cookies:

Google Analytics

Google Analytics is a service provided by Google, Inc. (“Google”). Google Analytics uses cookies to help us analyze how users use our website and our mobile site. The information generated by these cookies (including your truncated IP address) is transmitted to and stored by Google on servers in the United States. Google will use this information for the purpose of evaluating your, and other users’, use of our website and mobile site, compiling reports for us on website activity and providing other services relating to website activity and Internet usage. Please note that Google only receives your truncated IP address. This is sufficient for Google to identify (approximately) the country from which you are visiting our sites or accessing our players, but is not sufficient to identify you, or your computer or mobile device, individually.

Google cookies are those beginning “__ut”. You can find more information here, including a link to Google’s privacy policy. To opt out of this analysis by Google, please see the Opting Out section below.

Google AdWords and Google Remarketing

We may use the online advertising program Google AdWords and the Google Remarketing technology, both operated by the company Google Inc. (“Google”).

For measuring conversion with Google AdWords, a conversion tracking cookie is placed when a user clicks on an advertisement delivered by Google (“Ads”). Conversion tracking cookies expire after 30 days and are not used for personal identification. Google uses a different cookie for each Google AdWords customer and there is no consolidation of the cookie data with other data. You can find more information about Google AdWords here.

Google cookies are those beginning “__ut”.

To opt out of personalized ad serving by Google, please see the Opting Out section below.

Opting Out

We think it’s important that you are able to control the collection of personal data and have therefore made sure that you can opt out of any personal data collection using cookies or other technologies as set out below. (Please note that you cannot opt out of our use of Flash cookies or HTML5 local storage as these are necessary for the technical operation of the platform. However, we do not use these technologies to collect any personal data.)

Cookies:

You can use the settings within your browser to control the cookies that are set on your computer or mobile device. However, please be aware that cookies are important to many aspects for the Platform – if you set your browser to reject cookies, you may not be able to enjoy all of the features of the Platform. To find out how to see what cookies have been set and how to reject and delete cookies, please visit http://www.aboutcookies.org.

To opt-out of analysis by Google Analytics on our website and other websites, please visit http://tools.google.com/dlpage/gaoptout.

To opt-out of personalized ad delivery on the Google content network, please visit Google’s Ads Preferences Manager or if you wish to opt-out permanently even when all cookies are deleted from your browser you can install their plugin here.

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