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ACTIVE NOT RECRUITINGPhase2

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

Sponsor: Laboratoires Thea

NCT ID: NCT06627179

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Intravitreal Injection of Ultevursen (drug), No intervention, will not receive any active study intervention (other)
How long the study runs
Study runs about 43 months (dates as stated)
About the drug or intervention
Intravitreal Injection of Ultevursen — drug: Up to 4 doses over a 24-month period · No intervention, will not receive any active study intervention — other: Sham-procedure (no experimental drug administered)
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
8 Years and over
Who
All
Number of participants
81
Started
2024-12-11
Last checked
2025-07

Plain English Summary

What is this study?

  • • Testing a new treatment for retinitis pigmentosa (rp)
  • • Phase2 - 81 participants
  • • 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

Who can take part?

  • • Ages 8 Years and over
  • • Diagnosed with retinitis pigmentosa (rp)

Where?

  • • Headington - Oxford Eye Hospital
  • • Edinburgh - University of Edinburgh / NHS Lothian
  • • London - Moorfields Eye Hosptial

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

More detail

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.

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

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 8 Years and over
  • Who can join: All genders

Treatment history

Treatments you must have had:

  • ✓ and willing and able to complete all study assessments, in the opinion of the Investigator

What the study is looking for

  • ✓An adult (≥18 years) willing and able to provide agreement to take part for participation prior to performing any study...
  • ✓Clearly visible and measurable SD-OCT horizontal EZ width of ≥2.2 mm in both eyes based on the assessment of the CRC.
  • ✓BCVA ≥55 letters based on ETDRS (equivalent to 20/80 based on Snellen notation, or logarithm of the minimum angle of...
  • ✓Impairment of VF as assessed by SP with a mean sensitivity greater than 4 decibels (dB) and less than 25 dB measured...
  • ✓Mean sensitivity greater than 2 dB as determined by MP in the TE at screening.

Who cannot take part

  • ✗Presence of additional non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles in subjects who have biallelic...
  • ✗At screening, the EZ horizontal or vertical width are outside the field of the SD-OCT scan based on the assessment...
  • ✗Receipt of any IVT injection prior to study entry.
See the full criteria
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.

Where Is This Study? (3 UK sites)

Oxford Eye Hospital

Headington OX3 9DU, United Kingdom

Hospital R&D contact (matched)

Shahista Hussain

ouhtma@ouh.nhs.uk---

University of Edinburgh / NHS Lothian

Edinburgh EH39HA, United Kingdom

Hospital R&D contact (matched)

Accord Office

enquiries@accord.scot0131 242 3330

Moorfields Eye Hosptial

London EC1V 2PD, United Kingdom

Hospital R&D contact (matched)

Research Portfolio Managers- Daniela Narvaez, Anika Kadchha, Abi Chandrakumar, Xin Liu

moorfields.resadmin@nhs.net0207 566 2036
Data sourced from ClinicalTrials.gov · Last verified: 2025-07