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Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study

NCT06273150 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 225

Last updated 2024-05-10

No results posted yet for this study

Summary

DRPLA Natural History and Biomarkers Study (DRPLA NHBS) is a prospective observational study that will lay the foundation for clinical trials in DRPLA. The aims of this project are:

* To characterize the natural history of DRPLA in both juvenile- and adult-onset patients and study different modalities of biomarkers in this condition.
* To identify genetic factors and biomarkers that could predict disease progression.
* To provide a platform to support the design and conduct of clinical trials.

This study has three arms:

1. Adult Participants: this arm of the study will require participants to be 16 years old or over to participate.
2. Pediatric Participants: this arm of the study will require participants to be under 16 years old to participate.
3. Remote Participants: patients that cannot or do not wish to travel to one of the study sites can participate in this arm of the study, irrespective of their age.

Participants will have an annual visit for three years (baseline visit and two follow-up visits, three visits in total). Subjects who complete the whole protocol will be assessed on two consecutive days to reduce patient burden.

This project will allow for a better understanding of DRPLA and its course, and therefore allow for future clinical trials on this condition to be more precisely and effectively conducted.

Conditions

  • Dentatorubral-Pallidoluysian Atrophy

Interventions

OTHER

Positive genetic test for pathological expansion in ATN1

Positive genetic test for pathological expansion in ATN1

Sponsors & Collaborators

  • University of North Carolina, Chapel Hill

    collaborator OTHER

  • NYU Langone Health

    collaborator OTHER

  • University College, London

    lead OTHER

Principal Investigators

  • Paola Giunti · University College, London

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2022-05-01
Primary Completion
2026-04-30
Completion
2026-04-30

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06273150 on ClinicalTrials.gov