Actively Recruiting
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Led by Laboratoires Thea · Updated on 2026-03-30
81
Participants Needed
27
Research Sites
155 weeks
Total Duration
On this page
Sponsors
L
Laboratoires Thea
Lead Sponsor
S
Sepul Bio
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults (18 years or older) able and willing to provide informed consent before any study procedures
- Minors aged 8 to less than 18 years able to provide age-appropriate assent with a parent or legal guardian providing written permission
- Both eyes show clinical signs of Retinitis Pigmentosa involving Usher syndrome type 2 or non-syndromic RP
- Molecular diagnosis of biallelic disease-causing variants in the USH2A gene with at least one variant in exon 13
- Measurable SD-OCT horizontal EZ width of 2.2 mm or more in both eyes
- Best corrected visual acuity of 55 letters or better (ETDRS) in both eyes
- Visual field mean sensitivity between 4 and 25 decibels in the treatment eye
- Mean sensitivity over 2 decibels by microperimetry in the treatment eye
- Symmetry of disease between eyes within 10 letters difference in visual acuity
You will not qualify if you...
- Presence of non-exon 13 USH2A pathogenic variants on the allele carrying the exon 13 mutation if only one exon 13 variant is present
- Presence of non-exon 13 USH2A pathogenic mutations on both alleles if biallelic exon 13 mutations are present
- Pathogenic variants in other genes causing inherited retinal degenerative diseases
- EZ width outside the field of SD-OCT scan
- Significant ocular or non-ocular diseases or abnormalities that may increase risk or interfere with study assessments
- Unstable cystoid macular edema or recent medication changes for CME within 3 months prior to enrollment
- Any intraocular surgery within 3 months before study entry or planned during the study
- Prior intravitreal injections before study entry
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 27 locations
1
The University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
Bascom Palmer Eye Institute/University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
3
Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Massachusetts Eye and Ear
Boston, Massachusetts, United States, 02114
Actively Recruiting
5
University of Michigan- Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
Actively Recruiting
6
Duke Eye Center
Durham, North Carolina, United States, 27705
Actively Recruiting
7
Casey Eye Institute, Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
8
University of Pennsylvania, Scheie Eye Institute
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
9
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
10
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
11
University of Wisconsin- Madison
Madison, Wisconsin, United States, 53705
Actively Recruiting
12
Ghent University Hospital
Ghent, Belgium, B-9000
Actively Recruiting
13
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
São Paulo, Brazil, 04021-001
Actively Recruiting
14
Hospital for Sick Children
Toronto, Ontario, Canada, M5G1E8
Actively Recruiting
15
McGill University Health Centre for Innovative Medicine
Montreal, Quebec, Canada, H4A3J1
Actively Recruiting
16
Rigshospitalet and University of Copenhagen
Glostrup Municipality, Denmark, 2600
Actively Recruiting
17
Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique
Montpellier, France, 34295
Actively Recruiting
18
Centre de maladies rares CHNO des Quinze Vingt
Paris, France, 75012
Actively Recruiting
19
Universitätsklinikum Tübingen
Tübingen, Germany, 72076
Actively Recruiting
20
ASST Santi Paolo e Carlo Hospital, University of Milan
Milan, Italy, 20142
Actively Recruiting
21
AOU Università degli Studi della Campania Luigi Vanvitelli
Naples, Italy, 80131
Not Yet Recruiting
22
Amsterdam University Medical Center - Locatie AMC
Amsterdam, Netherlands, 1105 AZ
Actively Recruiting
23
Radboud Universitair Medisch Centrum
Nijmegen, Netherlands, 6525 GA
Actively Recruiting
24
Het Oogziekenhuis Rotterdam
Rotterdam, Netherlands, 3011 BH
Actively Recruiting
25
Oxford Eye Hospital
Headington, Oxford, United Kingdom, OX3 9DU
Actively Recruiting
26
University of Edinburgh / NHS Lothian
Edinburgh, United Kingdom, EH39HA
Actively Recruiting
27
Moorfields Eye Hosptial
London, United Kingdom, EC1V 2PD
Actively Recruiting
Research Team
S
Sepul Bio Advocacy Director
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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