Natural History Study for Best Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to better understand Best Vitelliform Macular Dystrophy (VMD), an eye disorder caused by mutations in the BEST1 gene that can lead to vision loss. Researchers seek to track the natural progression of the disease to aid in designing future treatments. The study will identify reliable methods to measure changes in the eyes and compare them between both eyes to determine if one can serve as a control in future trials. It is suitable for individuals diagnosed with VMD linked to the BEST1 gene who can attend four study visits over three years. As an unphased study, this trial allows participants to contribute to foundational research that could shape future treatment strategies for VMD.
Do I need to stop my current medications for this 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 focuses on understanding the natural history of Best Vitelliform Macular Dystrophy (VMD), a rare genetic eye disorder. Unlike treatments that aim to manage symptoms, this study seeks to uncover how the condition progresses over time, particularly in those with specific genetic variants in the BEST1 gene. By gaining a deeper understanding of the disease's progression, scientists hope to pave the way for more targeted and effective treatments in the future.
Who Is on the Research Team?
Stephen H Tsang, MD, PhD
Principal Investigator
Columbia University
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
Natural History Observation
Participants are observed to report the natural history of retinal degeneration and identify outcome measures for future trials
Follow-up
Participants are monitored for safety and effectiveness after the observation period
What Are the Treatments Tested in This Trial?
Interventions
- Natural History Study
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants with a clinical picture of Retinitis pigmentosa with dominant and recessive variants in the BEST1 gene
Find a Clinic Near You
Who Is Running the Clinical Trial?
Columbia University
Lead Sponsor
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Collaborator
National Eye Institute (NEI)
Collaborator
Centre Hospitalier National d'Ophtalmologie
Collaborator
Universität Tübingen
Collaborator
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