Ignite Creation Date:
2025-12-24 @ 5:09 PM
Ignite Modification Date:
2025-12-28 @ 9:10 AM
Study NCT ID:
NCT06273150
Status:
RECRUITING
Last Update Posted:
2024-05-10
First Post:
2024-02-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study
Sponsor:
University College, London
Organization:
Study Overview
Official Title:
Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study
Status:
RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
DRPLA NHBS
Brief 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.
* 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.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: