RecruitingNot Applicable

CureDRPLA Global Patient Registry

United States100 participantsStarted 2021-03-01

Plain-language summary

The objective of the CureDRPLA Global Patient Registry is to establish a longitudinal database of patient-reported data on individuals affected with Dentatorubral-pallidoluysian atrophy (DRPLA) from anywhere in the world. The CureDRPLA Global Patient Registry will address patient needs by: * Expanding patient engagement by documenting quality of life outcomes. * Providing anonymized data to the DRPLA research community on patient experience with the disease and priorities for treatment. * Connecting DRPLA patients with opportunities to participate in clinical research.

Who can participate

Age range0 Years – 100 Years

SexALL

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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).

What they're measuring

Patient- or caregiver-filled questionnaires

Timeframe: 1 year

Trial details

NCT IDNCT05489393

SponsorCureDRPLA

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2031-11-01

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