Biomarkers Study for Dentatorubral-Pallidoluysian Atrophy
(DRPLA NHBS Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on understanding the rare genetic condition known as dentatorubral-pallidoluysian atrophy (DRPLA). Researchers observe how DRPLA progresses over time and seek biological markers that could help predict its course. The study includes both adults and children with a genetic diagnosis of DRPLA, as well as individuals without the condition for comparison. Those with a confirmed DRPLA diagnosis, or who know someone with the condition, may find that participating in this study contributes to the development of future treatments. As an unphased trial, this study offers a unique opportunity to contribute to foundational research that could lead to breakthroughs in understanding and treating DRPLA.
Do I have to stop taking my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
Why are researchers excited about this trial?
Researchers are excited about this trial because it aims to uncover key insights into dentatorubral-pallidoluysian atrophy (DRPLA) by studying its natural history and identifying important biomarkers. Unlike typical treatment-focused studies, this trial seeks to understand the progression and biological markers of DRPLA, which could lead to earlier diagnosis and more targeted therapies in the future. By comparing individuals with the genetic mutation to those without neurological conditions, researchers hope to discover new pathways for intervention and improve outcomes for those affected by this rare genetic disorder.
Who Is on the Research Team?
Paola Giunti
Principal Investigator
University College, London
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Participants undergo initial assessments to establish baseline measurements for biomarkers and clinical features
Annual Follow-up
Participants have annual visits for follow-up assessments to monitor disease progression and biomarker changes
Final Assessment
Participants who complete the protocol are assessed on two consecutive days to reduce patient burden
What Are the Treatments Tested in This Trial?
Interventions
- Positive genetic test for pathological expansion in ATN1
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Subjects without neurological conditions (other than primary headache disorders), without a family history of DRPLA or a previous negative genetic test for pathological expansions in the ATN1 gene.
Subjects with a positive genetic test for a pathological expansion in the ATN1 gene.
Find a Clinic Near You
Who Is Running the Clinical Trial?
University College, London
Lead Sponsor
NYU Langone Health
Collaborator
NYU Grossman School of Medicine
Collaborator
University of North Carolina, Chapel Hill
Collaborator
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