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Hope for the blind: experimental drug targets rare genetic vision loss

NCT ID NCT06891443

Recruiting now Disease control Sponsor: Laboratoires Thea Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated Jun 13, 2026 · Updated 26 times

Summary

This study tests an experimental drug called sepofarsen in people with a rare genetic eye disease (LCA10) that causes blindness from a specific CEP290 gene mutation. About 32 participants aged 6 and older will receive the drug in one eye and a placebo in the other to see if it improves vision. The goal is to measure changes in eyesight and patient-reported vision improvement over time.

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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

  • Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)

    RECRUITING

    São Paulo, São Paulo, 04023-062, Brazil

  • INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

    RECRUITING

    Belo Horizonte, Minas Gerais, 30150270, Brazil

  • 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

The condition(s) this trial relates to.

blindness (disorder) Eye Abnormalities Eye Diseases, Hereditary eye disorder Leber congenital amaurosis Leber congenital amaurosis 10 Leber hereditary optic neuropathy Meckel Syndrome, Type 4 Neurologic Manifestations retinal disorder Sensation Disorders vision disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BLINDNESS BLINDNESS EYE DISEASES EYE DISEASES, HEREDITARY EYE DISORDERS CONGENITAL LEBER CONGENITAL AMAUROSIS LEBER CONGENITAL AMAUROSIS 10 NEUROLOGICAL MANIFESTATIONS NEUROLOGICAL MANIFESTATIONS RETINAL DISEASE SENSATION DISORDERS VISION DISORDER