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CureDRPLA Global Patient Registry

Sponsor
CureDRPLA (Other)
Overall Status
Recruiting
CT.gov ID
NCT05489393
Collaborator
Ataxia UK (Other)
100
Enrollment
1
Location
128
Anticipated Duration (Months)
0.8
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    The CureDRPLA Global Patient Registry aims to collect data from Dentatorubral-pallidoluysian atrophy (DRPLA) patients worldwide to identify a well-characterized cohort for participation in retrospective and prospective research. Participants will be required to read and sign an IRB-approved informed consent document prior to accessing questionnaires within the Registry. The Registry is solely for patients diagnosed with DRPLA.

    After informed consent is given, and assent for those age 12 to the age of consent, participants will be asked to complete a series of questionnaires to include information on demographics, specifics of diagnosis, medical history, research, functional mobility, quality of life, and health economics. Participants will be asked to indicate if they are interested in being contacted by the Registry Coordinator for potential participation in future clinical trials and/or studies. Participants will have the option to withdraw from the Registry at any time, and upon withdrawal, they will no longer be contacted from the Registry for any reason. Data that they have entered will remain part of the Registry, but their identification will not be released for any reason. Active participants in the Registry will receive yearly email reminders to update their questionnaire data to reflect the current status of disease progression.

    The Registry is available in English, French, Italian, Japanese, Korean, and Portuguese to encourage DRPLA patients worldwide to register. Participation is completed online, please visit our website for more information https://curedrpla.org/en/global-patient-registry/

    The REDCap Cloud platform will serve as the Registry Data Coordinating Center and will manage data entered into a web based electronic data capture (EDC) system that is 21 CFR Part 11 validated, ISO 27001 certified, HIPPA, CDISC, and GDPR compliant.

    Study Design

    Study Type:
    Observational [Patient Registry]
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    CureDRPLA Global Patient Registry for Individuals With Dentatorubral-pallidoluysian Atrophy (DRPLA)
    Actual Study Start Date :
    Mar 1, 2021
    Anticipated Primary Completion Date :
    Nov 1, 2031
    Anticipated Study Completion Date :
    Nov 1, 2031

    Arms and Interventions

    Arm Intervention/Treatment
    Individuals with DRPLA

    This registry is for people with a diagnosis of Dentatorubral-pallidoluysian atrophy (DRPLA)

    Outcome Measures

    Primary Outcome Measures

    1. Patient- or caregiver-filled questionnaires [1 year]

      The questionnaires cover information about demographics, diagnosis, medical history, activities of daily living, functional mobility and disease burden.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Years to 100 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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).

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 CureDRPLA New York New York United States 11231

    Sponsors and Collaborators

    • CureDRPLA
    • Ataxia UK

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    CureDRPLA
    ClinicalTrials.gov Identifier:
    NCT05489393
    Other Study ID Numbers:
    • CureDRPLA Registry_Version1.0
    First Posted:
    Aug 5, 2022
    Last Update Posted:
    Aug 5, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No

    Study Results

    No Results Posted as of Aug 5, 2022