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Stem Cell Models of Best Disease and Other Retinal Degenerative Diseases.
NCT02162953 · View on ClinicalTrials.gov ↗
Study Summary
Background: Autosomal recessive bestrophinopathy (ARB) is one of 5 blinding eye diseases caused by mutations in the gene BEST1. These diseases, collectively termed "bestrophinopathies" include ARB, Best vitelliform macular dystrophy (BVMD), adult-onset vitelliform dystrophy (AVMD), autosomal dominant vitreoretinalchoroidopathy (ADVIRC) and retinitis pigmentosa (RP) . Objective: To collect DNA/RNA and skin samples from individuals with ARB or other diseases due to mutations in the gene BEST1. These models will be used to identify and test therapeutic approaches to treating these diseases. Design: Study involves a one time donation of a skin punch biopsy and whole blood. Once the skin biopsy is obtained, skin fibroblasts will be isolated, which will be reprogrammed into iPSCs. RPE cells will be derived from the iPSCs
Conditions Studied
Study Locations (1)
Minnesota
- Mayo Clinic — Rochester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 48 participants |
| Start Date | 2014-02 |
| Est. Completion | 2022-12-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02162953
The ClinicalTrials.gov registry entry for NCT02162953 describes a study currently listed as completed. 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 48 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mayo Clinic, which has 3,246 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 5 conditions, with Retinal Disease appearing as the primary indexed condition, and to 0 interventions. 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: NCT02162953 reports 1 study location spanning 1 distinct geographic area — top geographies include Minnesota. 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 NCT02162953 about?
NCT02162953 is a clinical study titled "Stem Cell Models of Best Disease and Other Retinal Degenerative Diseases.". Background: Autosomal recessive bestrophinopathy (ARB) is one of 5 blinding eye diseases caused by mutations in the gene BEST1. These diseases, collectively termed "bestrophinopathies" include ARB, Best vitelliform macular dystrophy (BVMD), adult-onset vitelliform dystrophy (AVMD), autosomal dominan...
What is the current status of trial NCT02162953?
This trial is currently completed. The enrollment target is 48 participants. The study started on 2014-02. Estimated completion is 2022-12-31.
What conditions does trial NCT02162953 study?
This clinical trial studies the following conditions: Retinal Disease, Best Vitelliform Macular Dystrophy, Bestrophinopathy, Adult Onset Vitelliform Macular Dystrophy, Autosomal Dominant Vitreoretinalchoroidopathy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT02162953?
This trial is sponsored by Mayo Clinic, which has 3,246 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02162953 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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