The goal of the CureDRPLA Global Patient Registry is to collect information on all DRPLA patients in one registry. By collecting the information on all DRPLA patients, the CureDRPLA Global Patient Registry will be a powerful resource for research and will enhance opportunities for treatment development.
This registry is only for people diagnosed with Dentatorubral Pallidoluysian Atrophy.
Participation in this registry will require your consent to share your data without divulging your identity. Participation in the CureDRPLA Global Patient Registry is voluntary. The registry is maintained by CureDRPLA through a Governance Board. Please direct any questions to the registry coordinators at firstname.lastname@example.org
Please review our User Guide to help you get started.
Frequently Asked Questions
What is a patient registry and why is a patient registry needed?
Simply put, a patient registry is a database or collection of information about people affected by a particular condition. The more information that is collected about an illness, the closer researchers and pharmaceutical companies can get to helping communities find treatments that work. When a clinical trial is being planned, it is very important that patients suitable for that trial can be found and contacted quickly. The best way of ensuring this can happen is to make sure that patients’ details are all collected together in a registry that contains all the information that researchers will need.
What is the purpose of the CureDRPLA Global Patient Registry?
The objective of the CureDRPLA Global Patient Registry is to establish a longitudinal database of patient-reported data on individuals affected with DRPLA. The CureDRPLA Global Patient Registry will address patient needs by:
- Characterizing the spectrum of symptoms and heterogeneity of disease presentation of DRPLA.
- Establishing the rate of disease progression in the context of patient self-reporting of symptoms.
- Providing data to the DRPLA research community on patient experience with the disease and priorities for treatment.
- Connecting DRPLA patients with opportunities to participate in other research projects.
How is the CureDRPLA Global Patient Registry different than the DRPLA Natural History and Biomarkers study?
The differences between the two projects are:
- Management of the databases – CureDRPLA and Ataxia UK are the Lead Organisations in the CureDRPLA Global Patient Registry. The DRPLA Natural History and Biomarkers study has principal investigators in different countries (UK, USA, and Japan) under the guidance of CureDRPLA and Ataxia UK.
- Person entering data – The CureDRPLA Global Patient Registry is designed to house patient-entered data; the DRPLA Natural History and Biomarkers Study is designed to house clinician-entered data.
- Type of data collected – The CureDRPLA Global Patient Registry data reflects the patients’ perspectives of living with DRPLA; the DRPLA Natural History and Biomarkers Study data is based on clinical assessments conducted by medical professionals (e.g. measurements of movement and thinking abilities, MRI, and specimen collections – including blood and spinal fluid).
The CureDRPLA Global Patient Registry is available to all persons affected with DRPLA and parents of children affected with DRPLA. This Registry has a more global reach than the DRPLA Natural History and Biomarkers Study because data entered into the Registry is not dependent on visiting a study site.
Is the registry available internationally?
Yes. Participation is available to all DRPLA patients and families regardless of country of residence. Initially, the registry will be available in English, French, Italian, Japanese, Korean and Portuguese.
Does my doctor need to be involved or give permission?
No permission is needed from your doctor.
How often should I update my information in the registry? How can I update my data if it changes
You can update your information on the survey forms every 12 months. You will receive an email notification to remind you to review and update the survey forms.
Where will my data be kept and will it be confidential?
Your data you be stored securely in REDCap Cloud, an electronic data capture system that is 21 CFR Part 11 validated, ISO 27001 certified, HIPPA, CDISC, and GDPR compliant.
With your permission, the registry will share information on participants with patient and advocacy groups, scientists, industry and other qualified researchers in an anonymized and aggregated form that will prevent identification of individual participants.
Do I have to participate in a registry and can I withdraw if I change my mind?
Your participation in the CureDRPLA Global Patient Registry is completely voluntary. You can withdraw your name and personal information from the CureDRPLA Global Patient Registry at any time.
Was the CureDRPLA Global Patient Registry reviewed for ethical approval?
This registry was created with input from CureDRPLA and Ataxia UK representatives, DRPLA patients and caregivers, neurologists, scientists, and industry representatives.
This survey has been reviewed and approved by WCG IRB, an international ethics review board company (https://www.wcgirb.com/).
Who should I contact if I have any questions?
If you have any questions please do not hesitate to contact the Registry Coordinator at email@example.com.
Click here for CureDRPLA Global Patient Registry
Learn More About DRPLA
Living with dentatorubral-pallidoluysian atrophy can be challenging. This is why we at CureDRPLA are committed to gaining information on this brain disorder and what can be done to help those who suffer. Contact us today if you have questions or data to share with us.