Experimental eye drug aims to slow blindness in rare genetic disease

NCT ID NCT06627179

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

First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 37 times

Summary

This study tests an experimental drug called ultevursen for people with retinitis pigmentosa caused by a specific gene mutation (USH2A exon 13). The drug is injected into the eye and may help preserve vision. The trial will enroll 81 participants, including children as young as 8, and compare the drug to a sham procedure over two years.

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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

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Study contacts

Contact

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

Locations

AOU Università degli Studi della Campania Luigi Vanvitelli
NOT_YET_RECRUITING

Naples, 80131, Italy
ASST Santi Paolo e Carlo Hospital, University of Milan
RECRUITING

Milan, 20142, Italy
Amsterdam University Medical Center - Locatie AMC
RECRUITING

Amsterdam, 1105 AZ, Netherlands
Bascom Palmer Eye Institute/University of Miami
RECRUITING

Miami, Florida, 33136, United States
Baylor College of Medicine
RECRUITING

Houston, Texas, 77030, United States
Casey Eye Institute, Oregon Health & Science University
RECRUITING

Portland, Oregon, 97239, United States
Centre de maladies rares CHNO des Quinze Vingt
RECRUITING

Paris, 75012, France
Duke Eye Center
RECRUITING

Durham, North Carolina, 27705, United States
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
RECRUITING

São Paulo, 04021-001, Brazil
Ghent University Hospital
RECRUITING

Ghent, B-9000, Belgium
Het Oogziekenhuis Rotterdam
RECRUITING

Rotterdam, 3011 BH, Netherlands
Hospital for Sick Children
RECRUITING

Toronto, Ontario, M5G1E8, Canada
Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique
RECRUITING

Montpellier, 34295, France
INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte
RECRUITING

Belo Horizonte, 30150270, Brazil
Massachusetts Eye and Ear
RECRUITING

Boston, Massachusetts, 02114, United States
McGill University Health Centre for Innovative Medicine
RECRUITING

Montreal, Quebec, H4A3J1, Canada
Moorfields Eye Hosptial
RECRUITING

London, EC1V 2PD, United Kingdom
Oxford Eye Hospital
RECRUITING

Headington, Oxford, OX3 9DU, United Kingdom
Radboud Universitair Medisch Centrum
RECRUITING

Nijmegen, 6525 GA, Netherlands
Retina Foundation of the Southwest
RECRUITING

Dallas, Texas, 75231, United States
Rigshospitalet and University of Copenhagen
RECRUITING

Glostrup Municipality, 2600, Denmark
The University of California, San Francisco
RECRUITING

San Francisco, California, 94143, United States
University of Edinburgh / NHS Lothian
RECRUITING

Edinburgh, EH39HA, United Kingdom
University of Michigan- Kellogg Eye Center
RECRUITING

Ann Arbor, Michigan, 48105, United States
University of Pennsylvania, Scheie Eye Institute
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
University of Wisconsin- Madison
RECRUITING

Madison, Wisconsin, 53705, United States
Universitätsklinikum Tübingen
RECRUITING

Tübingen, 72076, Germany

What this could mean

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

Active substance

ultevrursen (an experimental drug injected into the eye)

What this could lead to

If successful, this could slow vision loss in people with a specific genetic form of retinitis pigmentosa, potentially preserving sight longer.

What could go wrong

This is an early Phase 2 trial with only 81 people. The drug may not work, and eye injections carry risks like infection or inflammation.

Conditions

The condition(s) this trial relates to.

Eye Abnormalities Eye Diseases, Hereditary retinal disorder retinitis pigmentosa Usher syndrome vision disorder

As listed by the trial registrant

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

DEAF BLIND DEAF BLIND EYE DISEASES, HEREDITARY EYE DISORDERS CONGENITAL RETINAL DISEASE RETINAL DISEASE RETINITIS PIGMENTOSA (RP) USHER SYNDROME TYPE 2 USHER SYNDROME TYPE 2 VISION DISORDERS