Welcome!
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 Tatton Brown Rahman Syndrome and DNMT3A Patient Registry serves to:
- Provide a convenient online platform for patients or their legally authorized representative to report cases of rare diseases;
- Support the design of clinical trials that explore new rare disease treatments;
- Conduct a prospectively-planned natural history study that will result in the most comprehensive understanding of TBRS, HESJAS, and DNMT3A-related disorders, and their progression over time;
- Characterize and describe TBRS, HESJAS, and DNMT3A-related disorders, separately and as a whole, and to better understand the variability and stages of TBRS, HESJAS, and DNMT3A-related disorders;
- Assist researchers studying the pathology of TBRS, HESJAS, and DNMT3A-related disorders;
- 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 TBRS, HESJAS, and/or DNMT3A-related disorders; and
- Identify people with TBRS, HESJAS, and/or DNMT3A-related disorders 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.
What types of data will be collected in the TBRS and DNMT3A Patient Registry?
The TBRS and DNMT3A Patient Registry collects data on the following topics:
- Socio-demographics
- Medical history and diagnostics
- Treatment and disease progression
- Management of care
- Quality of life
- Clinical Research ID (CRID)
Is the data secure?
The TBRS and DNMT3A Patient 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 (data at rest). Communications between the registry platform application server and the database are also encrypted. 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.
Video links:
Janet Woodcock, Former Director CDER FDA on NORD Registry Program