Recruiting

Observational Study

Sponsor:

CureDRPLA

Code:

NCT05489393

Conditions

DRPLA

Eligibility Criteria

Sex: All

Age: 0 - 70+

Healthy Volunteers: Not accepted

Study Details

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.

Conditions

DRPLA

Study ID

NCT05489393

Start date

Mar 1, 2021

Status verified date

Mar, 2026

Completion date

Nov 1, 2031

Anticipated

Primary completion date

Nov 1, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 70+

Healthy Volunteers: Not accepted

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).

Study Design

Enrollment

100 participants

Anticipated

Interventions and Outcome Measures

Arms

Individuals with DRPLA

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

Primary outcome measure

  • Patient- or caregiver-filled questionnaires [ Time Frame: 1 year ]

Central Contacts and Locations

Locations

CureDRPLA

Recruiting

New York, New York, United States, 11231

Contacts

Silvia Prades, PhD

718-624-6994silvia.prades@curedrpla.org

Principal Investigator:

Silvia Prades, PhD

More Information

Sponsor

CureDRPLA

Last update posted

Mar 17, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by CureDRPLA on 2026-03-17.