RecruitingPhase 2NCT06627179

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Sponsor

Laboratoires Thea

Enrollment

81 participants

Start Date

Dec 11, 2024

Study Type

INTERVENTIONAL

Conditions

deaf-blind Retinitis pigmentosa (RP)Retinal Disease Vision Disorders Eye Diseases Hereditary Eye Disorders Congenital Usher Syndrome Type 2

Summary

The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.

Eligibility

Min Age: 8 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Intravitreal Injection of Ultevursen and No intervention, will not receive any active study intervention for people with eye diseases hereditary, eye disorders congenital, and other related conditions. The study is currently recruiting participants at 28 locations. People eligible for this study include aged 8 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGIntravitreal Injection of Ultevursen

Up to 4 doses over a 24-month period

OTHERNo intervention, will not receive any active study intervention

Sham-procedure (no experimental drug administered)

Locations(28)

The University of California, San Francisco

San Francisco, California, United States

Bascom Palmer Eye Institute/University of Miami

Miami, Florida, United States

Emory University

Atlanta, Georgia, United States

Massachusetts Eye and Ear

Boston, Massachusetts, United States

University of Michigan- Kellogg Eye Center

Ann Arbor, Michigan, United States

Duke Eye Center

Durham, North Carolina, United States

Casey Eye Institute, Oregon Health & Science University

Portland, Oregon, United States

University of Pennsylvania, Scheie Eye Institute

Philadelphia, Pennsylvania, United States

Retina Foundation of the Southwest

Dallas, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

University of Wisconsin- Madison

Madison, Wisconsin, United States

Ghent University Hospital

Ghent, Belgium

INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

Belo Horizonte, Brazil

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

São Paulo, Brazil

Hospital for Sick Children

Toronto, Ontario, Canada

McGill University Health Centre for Innovative Medicine

Montreal, Quebec, Canada

Rigshospitalet and University of Copenhagen

Glostrup Municipality, Denmark

Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique

Montpellier, France

Centre de maladies rares CHNO des Quinze Vingt

Paris, France

Universitätsklinikum Tübingen

Tübingen, Germany

ASST Santi Paolo e Carlo Hospital, University of Milan

Milan, Italy

AOU Università degli Studi della Campania Luigi Vanvitelli

Naples, Italy

Amsterdam University Medical Center - Locatie AMC

Amsterdam, Netherlands

Radboud Universitair Medisch Centrum

Nijmegen, Netherlands

Het Oogziekenhuis Rotterdam

Rotterdam, Netherlands

Oxford Eye Hospital

Headington, Oxford, United Kingdom

University of Edinburgh / NHS Lothian

Edinburgh, United Kingdom

Moorfields Eye Hosptial

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06627179

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