A Phase 2 interventional study of Intravitreal Injection of Ultevursen and No intervention, will not receive any active study intervention in Retinitis Pigmentosa (RP), Usher Syndrome Type 2 and Deaf Blind, sponsored by Laboratoires Thea. Active, not recruiting at 26 sites in 10 countries. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2026-07-09.
Sponsored by Laboratoires Thea · Phase 2, Interventional, and Treatment
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.
A total of eighty-one (81) RP subjects will be enrolled in this study, randomized in a 2:1 ratio to either ultevursen or sham procedure, respectively. Subjects randomized to the active treatment group will receive therapy with ultevursen administered via intravitreal (IVT) injection to the treatment eye (TE) on Day 1 and at Months 6, 12, and 18. Subjects randomized to sham will undergo a sham procedure in the TE at the corresponding timepoints.
33 studies on the registry are indexed under Usher Syndromes; 13 are open to participants now.
This study's planned enrollment of 81 is above the median of 39 across 18 interventional studies indexed under Usher Syndromes.
Browse Usher Syndromes studies →Laboratoires Thea is the lead sponsor of 64 studies on the registry; 6 are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 6 (75%) have results posted.
Compared within Usher Syndromes, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Retinitis Pigmentosa. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
OR A minor (8 to \<18 years) able to provide age-appropriate assent for study participation with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures. An adult willing to comply with the protocol, follow study instructions, attend study visits as required and willing and able to complete all study assessments, in the opinion of the Investigator.
OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions and attend study visits with the subject as required, in the opinion of the Investigator.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects will receive an intravitreal injection (IVT) of ultevursen with concentrations of 3.6 mg/mL for initial dose and 1.2 mg/mL for maintenance doses every 6 months thereafter through Month 18 (up to 4 doses).
Drug: Intravitreal Injection of Ultevursen
Sham-procedure (no experimental drug administered)
Other: No intervention, will not receive any active study intervention
Up to 4 doses over a 24-month period
Sham-procedure (no experimental drug administered)
To evaluate efficacy after 24 months of treatment
Annualized percent change from baseline in ellipsoid zone (EZ) width as measured by spectral-domain optical coherence tomography (SD-OCT) up to Month 24.
Time frame: 24 Months
Annualized change from baseline in static perimetry (SP) mean sensitivity
Time frame: Month 24
Annualized change from baseline in microperimetry (MP) mean sensitivity
Time frame: Month 24
Change from baseline in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
Time frame: Month 24
Change from baseline in best-corrected visual acuity (BCVA) using the ETDRS chart
Time frame: Month 24
Percent change from baseline in EZ area by SD-OCT
Time frame: Month 24
Percent change from baseline in EZ width by SD-OCT
Time frame: Month 24
Change from baseline in the Michigan Retinal Degeneration Questionnaire (MRDQ) for subjects ≥13 years of age
Michigan Retinal Degeneration Questionnaire (MRDQ) scores in central vision, color vision, contrast sensitivity, scotopic function, photopic peripheral vision, mesopic peripheral vision, and photosensitivity Unabbreviated scale title: Michigan Retinal Degeneration Questionnaire Minimum and Maximum values: -3 and +3 Higher scores indicate a worse outcome.
Time frame: Month 24
Change from baseline in the Michigan Vision-Related Anxiety Questionnaire (MVAQ) for subjects ≥13 years of age
Michigan Vision-Related Anxiety Questionnaire (MVAQ) scores in rod-function anxiety and cone-function anxiety. Unabbreviated scale title: Michigan Vision-Related Anxiety Questionnaire Minimum and Maximum values: -3 and +3 Higher scores indicate a worse outcome.
Time frame: Month 24
Frequency and severity of ocular and non-ocular adverse events (AEs)
Time frame: Up to month 24
Systemic Exposure
Systemic serum concentration of ultevursen
Time frame: Up to month 24
Plan to share: No
Started Dec 2024 (actual). Primary completion is estimated for Jul 2028, 43 months after the start.
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This study is active, not recruiting, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.
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