Welcome to the Myotonic Dystrophy Family Registry, a tool we've created to help researchers and the DM community learn more about the scope and impact of this disease.
The purpose of the Myotonic Dystrophy Family Registry (which we call “The Registry”) is to:
- Assist researchers seeking participants for clinical trials and research studies.
- Speed up research in myotonic dystrophy (DM) by collecting information that scientists can use.
- Communicate with the DM community about upcoming research studies and clinical trials.
- Understand how DM can affect the quality of life and circumstances of people and families living with it.
- Help medical professionals improve how they treat individuals affected by DM.
- Learn how and why certain treatments work and don’t work.
Collecting the information you provide when you register and complete the survey will enable researchers, medical professionals, MDF, legislators, insurers and others to learn more about the scope and impact of this genetic disorder on individuals and families. Having this information is crucial to the process of developing new, improved treatments for myotonic dystrophy, and advocating on behalf of people living with DM to insurers, government agencies, school districts, and other institutions.
The Registry will also provide you with information regarding clinical trials for which you or your affected family member(s) may be eligible. And you can learn about DM, its impacts and the broader DM community in the same way researchers can, any time you want, by simply logging into and searching the Registry’s anonymous (de-identified) data.
The Registry is owned by the Myotonic Dystrophy Foundation (MDF), a U.S.-based charity created to support people living with DM and help find treatments and a cure for myotonic dystrophy through outreach, education, advocacy, and research.
The Registry platform is provided by Patient Crossroads (PC), a leading international provider for registries in the rare disease arena. Patient Crossroads currently provides the registry platform for over 30 rare disease organizations around the world. The National Institutes of Health (NIH) recently selected Patient Crossroads through a competitive process as the lead registry vendor to establish a Global Rare Diseases Registry and Data Repository (GRDR) to collect patient-entered clinical information for clinical research, and Patient Crossroads is a full partner in a sister project recently launched in Europe called RD-CONNECT that is working to develop an integrated platform connecting databases, registries, biobanks, and other information for rare disease research.
The Myotonic Dystrophy Family Registry is hosted in a SAS 70 Type II HIPAA compliant infrastructure with dedicated firewalls and advanced intrusion detection capability to secure your information. All registry transmissions are encoded for added protection. The highly-trusted and tested Patient Crossroads registry platform ensures that individuals sharing information through this registry can be confident in the privacy of their de-identified data (after you enter your information, your name and other identifying information is removed before it is shared with others, as described below) and the knowledge that PC does not sell or own any of the data you provide.
For the purpose of this Consent the “affected individual” will refer to the person diagnosed with myotonic dystrophy. “You” will refer to the person entering the information. The person entering the data may be the affected individual or a family member or guardian of the affected individual (the person legally responsible for the care and maintenance of the affected individual).
What is a registry?
A registry is a place to store detailed information about affected individuals with a specific disease or syndrome. In this case, The Registry is for people living with myotonic dystrophy. Establishing The Registry addresses two critical needs. First, scientists studying myotonic dystrophy need accurate, firsthand information to understand how myotonic dystrophy affects people. Second, scientists who are ready to start studies and test new treatments, will need to access The Registry to identify people that may be eligible to participate in studies. If an affected individual looks like a good match for a research study or trial, an interested scientist will contact The Registry. The Registry Coordinator or an agent of The Registry (such as a genetic counselor) will then contact the affected individual (or their family). Scientists cannot contact affected individuals (or their families) directly through The Registry, because they only have access to anonymous (de-identified) information. Your information is completely anonymous to anyone viewing The Registry except for the Registry Coordinator, MDF and other Registry staff.
Whose data are you collecting in The Registry?
Individuals who have a diagnosis of myotonic dystrophy, including those who have passed away.
What if I register in more than one registry/database?
You are encouraged to register with any DM or rare disease registries that will benefit from your information and which are appropriate for your participation. The Myotonic Dystrophy Family Registry works collaboratively with other DM registries to coordinate activities and improve access to critical research information. Your personal identifiable information submitted to The Registry will always remain protected.
Who can consent to join this Registry?
Affected individuals over the age of 18 who understand this consent (and thus do not have a legal custodian) are eligible to join The Registry on their own. Otherwise, the legal guardian, parent, or custodian of the individual with a diagnosis of myotonic dystrophy must consent for the affected individual to join. When the affected individual becomes 18 (and if they are able), consent will be obtained directly from the affected individual for continued participation.
What are the steps to filling out The Registry with an affected individual’s information?
Read “Understanding Your Participation” above and then decide whether you want to participate in The Registry. If you do, complete this Registry Consent. The next step is to provide basic information about you, to create a login username and password. You will then be directed to the registration page where you will include your personal information, as well as information about your family member who has myotonic dystrophy, if you are participating on behalf of someone else. After completing the registration page, you can answer the registry survey.
If more than one family member has myotonic dystrophy, you can create a single-family account. Register the first individual with myotonic dystrophy and answer the survey. You can then add other family members with myotonic dystrophy to the same account.
Where will the data go?
All of the information you provide will be maintained in a safe, secure database conforming to strict government privacy protection policies. Any information that could identify you and your family members will not be shared without your approval.
Will I be contacted about the information I provide in the Registry?
Yes. The Registry Coordinator may contact you to gather any missing or incomplete information in your profile. Having complete, accurate and current profiles is critical to maintaining the value and purpose of The Registry. The Registry Coordinator or an agent of The Registry (such as a genetic counselor) will also contact you if a researcher would like to communicate with you or an affected family member in your account regarding possible participation in a research study or clinical trial.
Will I be expected to provide The Registry with additional information in the future?
Yes. The Registry is most valuable for scientific research when it is kept up-to-date. Therefore, you will be asked to update your profile and information once a year. We will send out notifications annually to remind you. We may also ask you to fax or upload your test results [such as genetic test results], and any other relevant reports, if requested by a researcher. You should also update The Registry whenever there is a change in condition (gain or loss of skill), change in medication, or new symptom.
Who will have access to the medical and other records on The Registry?
You own the data you provide to The Registry. The Myotonic Dystrophy Family Registry is the guardian of the information you provide. Unless you instruct us otherwise, MDF, as owner of the registry, will have access to the information you provide.
The goal of The Registry is to share detailed medical and other information with scientists, other researchers, clinicians, pharmaceutical companies, and the DM community, while protecting your privacy and the privacy of all other participants. We do this by hiding the name, address and other “identifying” information from any individuals who register to view this information. We call this “de-identified” or “de-coded” data (information from which all personal identification has been removed).
A subset of anonymous, de-identified information collected from each profile may be shared with certain other databases, including the Global Rare Diseases Patient Registry and Data Repository (GRDR) created by the National Institutes of Health (NIH). We will combine our de-identified data with the GRDR and other databases in order to develop global knowledge of myotonic dystrophy that may lead to new research studies, clinical trials, and clinical treatments.
The Registry has to follow rules to protect information about you. Federal and state laws also protect your privacy. This part of the Registry Consent tells you what information about you may be collected in this study. It also tells you who might see or use your information.
Generally, only the Registry Coordinator will know that you have given information and will see your information. However, there are a few exceptions that are listed later in this section of the Registry Consent.
Are there risks to registering?
There is minimal risk in taking part in The Registry. The Registry includes questions that can be sensitive and you may feel uncomfortable answering. You do not have to share any information you do not want to. Another unlikely risk is potential breaches in the computer system. In the event the there is a breach in The Registry’s computer system all participants will be notified.
I want to be involved in a clinical trial. If I register, is this guaranteed?
Although one of the main goals of The Registry is to make it easier for affected individuals to participate in research, there is no guarantee that that you or your family member will be eligible for a trial.
(Please note that even if the coordinators and staff of a clinical trial believe that you might be eligible for the trial, based on the data about you stored in The Registry, it is still possible you will not meet the trial inclusion criteria (requirements to be eligible) after all. Please also be aware that if we inform you about the existence of a trial, this does not imply that we endorse it. In order to participate in any trial, you will need to fill out a separate informed consent form.)
I don’t want to be involved in a clinical trial. Should I still register?
Absolutely. We encourage you to register, even if you don’t want to take part in a trial. Your information will still be useful to researchers who are trying to learn more about people living with myotonic dystrophy.
Can I withdraw if I change my mind?
If I have given data or information to doctors, researchers, clinics or hospitals in the past, is it OK to give my data to The Registry now?
Yes. We will be taking precautions to make sure information used is not redundant with data that may have previously been collected.
What are my options if I do not want to be in the Registry?
You do not have to join The Registry. Participation is voluntary.
Will it cost me anything to be in this Registry?
No. Participation in The Registry is free.
Who should I contact if I have any questions?