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NCT05809635 · ClinicalTrials.gov registry record

Study of BEST1 Vitelliform Macular Dystrophy

A clinical trial of Retinitis Pigmentosa and Best Vitelliform Macular Dystrophy, sponsored by Columbia University.

Recruiting
Registry status
52
Enrollment target
3
Study locations

NCT05809635 is a study of Retinitis Pigmentosa and Best Vitelliform Macular Dystrophy that is actively recruiting participants, run by Columbia University. The registered enrollment target is 52 participants, below the 260-participant average among 37 other Retinitis Pigmentosa trials with a reported enrollment target (80% lower). The trial reports 3 study locations across 2 states.

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The verdict

NCT05809635, a study of Retinitis Pigmentosa and Best Vitelliform Macular Dystrophy, is actively recruiting participants, sponsored by Columbia University.

RECRUITING
Registry status
52 participants
Enrollment target
3
Study locations

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.

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

FieldValue
Enrollment Target 52 participants
Start Date 2021-03-30
Est. Completion 2026-05-31

Sponsor

Columbia University

958 total trials

What the Registry Record Tells You About NCT05809635

The ClinicalTrials.gov registry entry for NCT05809635 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 52 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 260-participant average among 37 other Retinitis Pigmentosa trials with a reported enrollment target (80% lower). The listed sponsor is Columbia University, which has 958 total studies on file at ClinicalTrials.gov.

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.

NCT05809635 reports 3 study locations spanning 2 distinct geographic areas - top geographies include Other, New York.

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.

What interventions are being tested in trial NCT05809635?

The interventions under investigation include: Natural History Study (OTHER).

Who is sponsoring clinical trial NCT05809635?

This trial is sponsored by Columbia University, which has 958 total clinical trials registered on ClinicalTrials.gov.

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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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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