Global registry for patients with DRPLA
CureDRPLA Global Patient Registry for Individuals With Dentatorubral-pallidoluysian Atrophy (DRPLA)
This study is creating a global database to gather information from people with DRPLA to help improve understanding of the condition and connect patients with future research opportunities.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Ages | 0 Years to 100 Years |
| Sex | All |
| Sponsor | CureDRPLA Academic / other |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT05489393 on ClinicalTrials.gov |
What this trial studies
The CureDRPLA Global Patient Registry aims to create a comprehensive database of patient-reported data from individuals diagnosed with Dentatorubral-pallidoluysian atrophy (DRPLA) worldwide. This observational registry will collect information on demographics, medical history, quality of life, and functional mobility through questionnaires completed by participants. The initiative seeks to enhance patient engagement, provide valuable anonymized data to the research community, and connect patients with opportunities for future clinical trials. Participants can withdraw at any time, ensuring their data remains confidential while contributing to ongoing research efforts.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals of all ages with a self-reported diagnosis of DRPLA.
Not a fit: Patients with ataxia conditions other than DRPLA will not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could significantly improve understanding of DRPLA and enhance treatment options for patients.
How similar studies have performed: Other patient registries have successfully contributed to research and treatment advancements in rare diseases, indicating a positive precedent for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Individuals of all ages with a self-reported diagnosis of DRPLA. * Understand and sign the informed consent form (IFC). Participants who lack the capacity to consent (e.g. cognitively impaired individuals) will require consent from the legal authorized representative, and the assent of the subject will be obtained to the extent compatible with their capacity. Participants at age 12 to age of consent will require assent along with the consent of their parent or legal guardian. Exclusion Criteria: * Ataxia conditions other than DRPLA. * Failure to sign the IFC (and assent form, as needed).
Where this trial is running
New York, New York
- CureDRPLA — New York, New York, United States (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.