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Study of BEST1 Vitelliform Macular Dystrophy
NCT05809635 · View on ClinicalTrials.gov ↗
Study Summary
The purpose of this study is to establish the natural history of of participants with BESTROPHIN 1 Vitelliform Macular Dystrophy. The blinding disorder Best Vitelliform Macular Dystrophy (VMD) is caused by any one of more than 250 different mutations in the BEST1 gene. As new treatments are developed, a clear understanding of the natural history of disease progression of BEST1 VMD is necessary. The goals of this natural history study are to: 1. Report the natural history of retinal degeneration in participants with a clinical diagnosis of VMD with molecular confirmation of a pathogenic BEST1 mutation(s). 2. Identify sensitive structural and functional outcome measures to use for future multicenter clinical trials for the treatment of BESTROPHIN 1 VMD. 3. Compare progression of the identified structural and functional measures between the two eyes to judge the suitability of the second untreated eye as a control for a future clinical trial involving unilateral treatment 4. Identify well-defined patient populations for future clinical trials of investigative treatments for BEST1 VMD.
Conditions Studied
Interventions
- OTHER Natural History Study
Study Locations (3)
Other
- Institut de la Vision/Centre de maladies rares du Centre Hospitalier National Ophtalmologique des Quinze-Vingts — Paris
- Eberhard Karls University Tubingen — Tübingen
New York
- Columbia University Irving Medical Center — New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 52 participants |
| Start Date | 2021-03-30 |
| Est. Completion | 2026-05-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05809635
The ClinicalTrials.gov registry entry for NCT05809635 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 52 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Columbia University, which has 875 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Retinitis Pigmentosa appearing as the primary indexed condition, and to 1 intervention — of which Natural History Study is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05809635 reports 3 study locations spanning 2 distinct geographic areas — top geographies include Other, New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05809635 about?
NCT05809635 is a clinical study titled "Study of BEST1 Vitelliform Macular Dystrophy". The purpose of this study is to establish the natural history of of participants with BESTROPHIN 1 Vitelliform Macular Dystrophy. The blinding disorder Best Vitelliform Macular Dystrophy (VMD) is caused by any one of more than 250 different mutations in the BEST1 gene. As new treatments are develo...
What is the current status of trial NCT05809635?
This trial is currently recruiting. The enrollment target is 52 participants. The study started on 2021-03-30. Estimated completion is 2026-05-31.
What conditions does trial NCT05809635 study?
This clinical trial studies the following conditions: Retinitis Pigmentosa, Best Vitelliform Macular Dystrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05809635?
The interventions under investigation include: Natural History Study (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05809635?
This trial is sponsored by Columbia University, which has 875 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05809635 being conducted?
This trial has 3 study locations across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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