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Clinical Trials in the UK / NCT06627179
Active, not recruiting Phase 2

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

NCT06627179 · tracked via the Priya Life Science UK tracker
Sponsor
Laboratoires Thea
Phase
Phase 2
Started
2024-12-11
Last updated
2026-07-09

Condition(s) studied

Retinitis Pigmentosa (RP)Usher Syndrome Type 2Deaf BlindRetinal DiseaseEye Diseases, HereditaryEye Disorders CongenitalVision Disorders

Investigational drug(s) / intervention(s)

Intravitreal Injection of UltevursenNo intervention, will not receive any active study intervention

Intravitreal Injection of Ultevursen: Up to 4 doses over a 24-month period

No intervention, will not receive any active study intervention: Sham-procedure (no experimental drug administered)

Study 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

Sex
ALL
Min age
8 Years
Max age
Healthy volunteers
No
Inclusion Criteria: 1. An adult (≥18 years) willing and able to provide informed consent for participation prior to performing any study related procedures 2. 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. 3. Both eyes exhibit clinical presentation consistent with RP involving Usher syndrome type 2 or NSRP based on ophthalmic, audiologic, or vestibular examinations. At screening, the Investigator will make the clinical diagnosis of "Usher syndrome type 2a," defined as RP with congenital hearing loss, or "non-syndromic RP," defined as RP without congenital hearing loss. 4. A molecular diagnosis of biallelic disease causing variants (pathogenic or likely pathogenic) in the USH2A gene where at least one of the variants is located on exon 13. A historic genotyping report from a certified laboratory is acceptable with Sponsor approval. 5. Clearly visible and measurable SD-OCT horizontal EZ width of ≥2.2 mm in both eyes based on the assessment of the CRC. 6. BCVA ≥55 letters based on ETDRS (equivalent to 20/80 based on Snellen notation, or logarithm of the minimum angle of resolution \[logMAR\] +0.6) in both eyes. 7. Impairment of VF as assessed by SP with a mean sensitivity greater than 4 decibels (dB) and less than 25 dB measured by a V target size in the TE at screening. 8. Mean sensitivity greater than 2 dB as determined by MP in the TE at screening. 9. Symmetry of baseline disease in both eyes, defined as the mean BCVA (based on ETDRS) of one eye within ≤10 letters of the mean BCVA of the other eye at screening. Exclusion Criteria: 1. Presence of additional non-exon 13 USH2A pathogenic or likely pathogenic variant on the USH2A allele carrying the exon 13 mutation in subjects who have one exon 13 disease causing variant and one non-exon 13 disease causing variant. 2. Presence of additional non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles in subjects who have biallelic exon 13 mutations. 3. Presence of pathogenic or likely pathogenic variants in genes (other than the USH2A gene) which are known to be associated with other inherited retinal degenerative diseases or syndromes. Specifically, the presence of homozygous or compound heterozygous known disease-causing mutations in other genes involved in recessive retinal dystrophies, or the confirmed presence of a known single disease-causing variant in genes involved in dominant, X-linked, or mitochondrial retinal dystrophy genes is exclusionary. 4. At screening, the EZ horizontal or vertical width are outside the field of the SD-OCT scan based on the assessment of the CRC. 5. Presence of any significant ocular or non-ocular disease/disorder (including medication and laboratory test abnormalities) which, in the opinion of the Investigator may either put the subject at risk because of participation in the study, may impact the subject's ability to participate in the study, or may interfere with assessment of efficacy and safety in the study. 6. Presence of unstable concurrent cystoid macular edema (CME), or subject started on (or changed dose of) any medication for CME in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment). However, stable CME that disrupts the EZ width measurement, as determined by CRC, is an exclusion. 7. Any intraocular surgery within 3 months of study entry or any planned intraocular or peri-ocular surgery during the study. Subjects may be eligible after 3 months post-surgery as long as they have fully recovered, in the opinion of the Investigator. 8. Receipt of any IVT injection prior to study entry.

Primary outcome measure(s)

Trial sites (26)

FacilityCityRegionStatus
The University of California, San Francisco San Francisco California
Bascom Palmer Eye Institute/University of Miami Miami Florida
Emory University Atlanta Georgia
Massachusetts Eye and Ear Boston Massachusetts
University of Michigan- Kellogg Eye Center Ann Arbor Michigan
Casey Eye Institute, Oregon Health & Science University Portland Oregon
University of Pennsylvania, Scheie Eye Institute Philadelphia Pennsylvania
Retina Foundation of the Southwest Dallas Texas
Baylor College of Medicine Houston Texas
University of Wisconsin- Madison Madison Wisconsin
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
McGill University Health Centre for Innovative Medicine Montreal Quebec
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
Amsterdam University Medical Center - Locatie AMC Amsterdam Netherlands
Radboud Universitair Medisch Centrum Nijmegen Netherlands
Het Oogziekenhuis Rotterdam Rotterdam Netherlands
Oxford Eye Hospital Headington Oxford
University of Edinburgh / NHS Lothian Edinburgh United Kingdom
Moorfields Eye Hosptial London United Kingdom

More Laboratoires Thea trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06627179 on ClinicalTrials.gov ↗ ← All trials in the UK