Breakthrough eye injection trial offers hope for children born blind

NCT ID NCT06891443

Summary

This study is testing whether regular eye injections of an experimental drug called sepofarsen can improve vision in people with a specific genetic form of Leber congenital amaurosis (LCA), which causes severe vision loss from childhood. In this carefully designed trial, each participant will receive the drug in one eye and a placebo injection in the other eye, allowing doctors to directly compare results. The goal is to see if the treatment is safe and can help control this blinding disease.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Centre de maladies rares CHNO des Quinze Vingt

    RECRUITING

    Paris, 75012, France

  • Justus-Liebig Universität - Department of Ophthalmology

    RECRUITING

    Giessen, 35392, Germany

  • Klinikum der Ludwig-Maximilian Universität München

    RECRUITING

    München, 81377, Germany

  • Moorfields Eye Hospital NHS Foundation Trust

    RECRUITING

    London, EC1V 2PD, United Kingdom

  • Radboud Universitair Medisch Centrum

    RECRUITING

    Nijmegen, 6525 GA, Netherlands

  • The Hospital for Sick Children - SickKids

    RECRUITING

    Toronto, Ontario, M5G 2L3, Canada

  • UCSF Wayne and Gladys Valley Center for Vision

    RECRUITING

    San Francisco, California, 94158, United States

  • Universitair Ziekenhuis Gent (UZ)

    RECRUITING

    Ghent, 9000, Belgium

  • University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G 2C8, Canada

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Miami - Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33156, United States

  • University of Minnesota Medical School

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • University of Tuebingen - Inst. for Ophthalmic Research

    RECRUITING

    Tübingen, 72076, Germany

Conditions

Explore the condition pages connected to this study.