Experimental eye drug hopes to restore sight in rare blindness
NCT ID NCT06891443
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely improves vision. Participants must have a specific CEP290 gene mutation and be at least 6 years old.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- sepofarsen (an RNA-based drug injected into the eye)
- What this could lead to
- If it works, this could improve vision in people with a rare genetic form of blindness.
- What could go wrong
- This is a small, early-stage trial with only 32 people. The drug may not improve vision or could cause side effects like eye irritation or infection.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Centre de maladies rares CHNO des Quinze Vingt
RECRUITINGParis, 75012, France
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Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
RECRUITINGSão Paulo, São Paulo, 04023-062, Brazil
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Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital)
RECRUITINGBarcelona, 08950, Spain
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INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte
RECRUITINGBelo Horizonte, Minas Gerais, 30150270, Brazil
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Justus-Liebig Universität - Department of Ophthalmology
RECRUITINGGiessen, 35392, Germany
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Klinikum der Ludwig-Maximilian Universität München
RECRUITINGMünchen, 81377, Germany
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Moorfields Eye Hospital NHS Foundation Trust
RECRUITINGLondon, EC1V 2PD, United Kingdom
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Radboud Universitair Medisch Centrum
RECRUITINGNijmegen, 6525 GA, Netherlands
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The Hospital for Sick Children - SickKids
RECRUITINGToronto, Ontario, M5G 2L3, Canada
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UCSF Wayne and Gladys Valley Center for Vision
RECRUITINGSan Francisco, California, 94158, United States
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Universitair Ziekenhuis Gent (UZ)
RECRUITINGGhent, 9000, Belgium
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University of Alberta
RECRUITINGEdmonton, Alberta, T6G 2C8, Canada
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
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University of Miami - Bascom Palmer Eye Institute
RECRUITINGMiami, Florida, 33156, United States
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University of Minnesota Medical School
RECRUITINGMinneapolis, Minnesota, 55455, United States
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University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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University of Tuebingen - Inst. for Ophthalmic Research
RECRUITINGTübingen, 72076, Germany
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