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Scientists map rare blindness to speed future cures

NCT ID NCT04765345

First seen Feb 03, 2026 · Last updated Jun 21, 2026 · Updated 19 times

Summary

This study follows 44 people with a rare genetic form of Usher syndrome caused by PCDH15 mutations. Over 48 months, researchers measure how their vision changes using eye exams and imaging. The goal is to identify the best ways to track disease progression, which will help design future clinical trials for potential treatments.

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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

Locations

  • CHNO des Quinze-Vingts

    Paris, France

  • Duke University, Duke Eye Center

    Durham, North Carolina, 27710, United States

  • Haddassah Medical Center

    Jerusalem, Israel

  • Hospital for Sick Children

    Toronto, Ontario, Canada

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • Radboud University

    Nijmegen, Netherlands

  • The Johns Hopkins Wilmer Eye Institute

    Baltimore, Maryland, 21287, United States

  • University Hospital Basel

    Basel, 4031, Switzerland

  • University of California, San Francisco

    San Francisco, California, 94143-0344, United States

  • University of Tubingen

    Tübingen, Germany

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this study could provide the key measurements needed to test new treatments for this rare form of blindness.

What could go wrong

This is an observational study, not a treatment trial. It only measures disease progression and does not test any therapy, so it cannot directly help participants.

Conditions

The condition(s) this trial relates to.

autosomal recessive nonsyndromic hearing loss 23 Eye Diseases, Hereditary retinal degeneration retinitis pigmentosa

As listed by the trial registrant

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