About the registry
The Registry is a self-reported, international, listing registry for dystonia-affected persons and their families who carry any of the dystonia genes.
A listing registry is “Data that is collected in a naturalistic manner such that the management of patients is determined by the caregiver and patient together and NOT by the registry protocol.”
As the Registry is a voluntary program, Tyler’s Hope and partners support and monitor several activities aimed at increasing enrollment and data submission by participants, their families. These activities are managed by the DIPR Registry Associates (RAs) operating from offices in the US and Europe..
The purpose of the Registry is to collect uniform and meaningful clinical data.
About the Registry Sponsors
Tyler's Hope for a Dystonia Cure, is a non-profit, 501(c)(3) organization committed to supporting scientific research that will lead to a better understanding of the causes for prevention and ultimately for a cure for DYT-1 dystonia. Founded by the family of Tyler, a DYT1 child, the organization is also dedicated to raising awareness of DYT1 dystonia. As the founding organization for the Dystonia International Patient Registry (DIPR), Tyler's Hope looks to expedite clinical research into potential new treatments for this form of dystonia. For more information on Tyler's Hope for a Dystonia Cure, contact the organization at www.tylershope.org.
The Dystonia Medical Research Foundation, is dedicated to advancing research for more treatments and ultimately a cure, promoting awareness and education and supporting the needs and well being of affected individuals and families with dystonia. The DMRF was founded in 1976 and has been a part of most of the scientific breakthroughs in dystonia research in the past three decades. The DMRF was pleased to join Tyler's Hope in the important DIPR registry effort. To learn more about dystonia, contact the Dystonia Medical Research Foundation at 1-800-377-3978 or www.dystonia-foundation.org.
Registry Steering Committee
Rick Staab
Tylers Hope Foundation - CoFounder
InterMed Biomedical Services, Inc. – President InterMed Group CEO
Peter West
Technology Consultant
Jan Teller, MA, PhD
Chief Scientific Advisor
Dystonia Medical Research Foundation
Janet Hieshetter
Executive Director
Dystonia Medical Research Foundation
Michael Okun, MD
Movement Disorders Center
University of Florida McKnight Brain Institute
National Medical Director, National Parkinson Foundation
DATA COLLECTION AND SUBMISSION
The purpose of the Registry is to collect uniform and meaningful clinical data on participants with DYT-1 Dystonia and any other form of dystonia which results in a positive genetic test (for example DYT6). Since, at the time of enrollment, participants will be at various stages in their disease course and medical care, the Registry is focused on collecting basic information with updates being made to information such as treatments, drug therapies etc. associated with the management of their disease. Both retrospective and prospective data collection are encouraged. Data collection will focus on contact information, familial DYT-1 (and other dystonia genes) history, medications, previous treatments and others.
Secure Internet technology and other electronic advancements will be utilized to enhance data collection, education, and reporting capabilities. Where secure Internet technology is not available, or if it is not the choice of the patient to use them, data are collected on paper CRFs. The data collected are maintained by Tyler’s Hope Foundation and it’s partners in an electronic database. Paper originals will be scanned and maintained electronically as source documents.
Since a number of these participants are children, a primary Registry contact person will be designated and will be responsible for completing and submitting the standard DIPR registry form and Link.
QUALITY ASSURANCE OF DATA
Data collected through this Registry will first be entered via the DIPR registry website and stored in a database. Staff of the Registry program will review the data for missing datapoints, incomplete information, and discrepancies with previously submitted data. If necessary, issues will be resolved with the individual electronically, by telephone, or FAX. Quarterly this data will be reviewed by a Data Monitoring Committee (DMC) and once deemed usable will be marked in the Registry with the date of DMC review. Only then, will this data be available as Registry data.
All data management and analysis will occur in a validated computing environment. However, Registry data are not verified against source documents, in contrast with clinical trials, where source verification is performed in accordance with Good Clinical Practice (GCP) guidelines.
STATISTICAL METHODS AND DATA REPORTING
Data will be analyzed and reported periodically and upon individual requests from participating physicians or requesting researchers. In addition, annual reports summarizing de-identified data from this registry will be provided to appropriate regulatory authorities and all registry stakeholders. Analyses will be performed using the SAS statistical software system. Data are summarized using descriptive statistics for participants and family members enrolled in the Registry.
CONFIDENTIALITY
Patient Confidentiality
To maintain participant confidentiality, participant contact information is stored in a separate database than the participant's medical information. These are linked by the Registry identification (ID) number and Registry Family identification number. Additionally, all participants are referenced by Registry ID and Registry Family ID. If supplemental laboratory or imaging reports are submitted to the Registry, the participants’s name should be deleted and the participant’s ID number and Family ID number should be added to each attached page.
Physician Confidentiality
All physician information is confidential. Furthermore, physician-specific patient data will remain confidential and will not be released to other physicians without the patient’s prior written approval, in accordance with applicable laws related to the confidentiality of patient information. No physician-to-physician data comparisons will be made.
PATIENT DISCONTINUATION
Participation is voluntary. A participant may decline to participate or withdraw consent at any time and without affecting their medical treatment.
RISKS AND BENEFITS
Benefits
Participants and their family members will benefit by participating in the DIPR Registry in many ways. Their primary benefit will be – access to the DIPR registry website where they will be able to view up to date research – what’s happening on the road to a cure.
Risks
Since participation in this registry does not require any assessments or procedures, the only risk to the participant is disclosure of personal health data. Every effort has been taken to protect the individual’s identity and health information in accordance with the Code of Federal Regulations Part 46 – Protection of Human Subjects.