For Patients

LGMD2A/Calpainopathy Registry

Welcome!

The LGMD2A/Calpainopathy Registry is an online registry for people with Calpainopathy. It is sponsored by Coalition to Cure Calpain 3 and hosted by the National Organization for Rare Disorders (NORD®) on their IAMRARE® platform. This registry will collect information from participants (or their authorized representatives) who are affected by Calpainopathy.

What is a Patient Registry?

A patient registry is a collection of standardized information about a group of patients who share a condition. The information may be used for a variety of purposes such as conducting natural history studies and supporting disease-specific clinical trial recruitment. The LGMD2A/Calpainopathy Registry serves to:

  • Support the design of clinical trials that explore new Calpainopathy treatments;
  • Describe the people who have Calpainopathy and how Calpainopathy may be different for different people;
  • Understand how Calpainopathy changes over a person’s lifetime;
  • Learn how Calpainopathy is treated and how people respond to those treatments;
  • Help to develop best practices, management guidelines, and recommendations so that clinicians can know how to give the best care to improve the quality of life and outcomes of people with Calpainopathy; and
  • Identify people with Calpainopathy who might be willing to take part in other research studies or clinical trials. You will be able to choose whether you want to hear about these other studies. If you choose to, you will be contacted by registry staff on behalf of outside researchers to inform you about these studies.

What types of data will be collected in the LGMD2A/Calpainopathy Registry?  

The LGMD2A/Calpainopathy Registry collects data on the following topics:

  • Socio-demographics
  • Medical history and diagnostics
  • Treatment and disease progression
  • Management of care
  • Quality of life

Is the data secure?

The LGMD2A/Calpainopathy Registry follows strict government guidelines to assure patient information is protected. The platform is served over HTTPS, which means that the data is encrypted when being sent from the user’s browser to the NORD servers. The data is also kept encrypted in the NORD database. Communications between the registry platform application server and the database are also encrypted. NORD stores Sponsor and Participant Registry Data on NORD encrypted servers and/or encrypted servers of third-party vendors hosted in Canada. Regular back-up at commercially acceptable intervals is provided. These servers meet industry standards and are compliant with US and international regulations, including GDPR.  As with any information one provides electronically, there is a very rare chance that privacy could be compromised. However, the registry and the security measures minimize the chance of this occurring.

What is a Registry?

Janet Woodcock, Former Director CDER FDA on NORD Registry Program