ClinicalTrials.gg
Active, not recruitingNCT06627179LUNAUpdated Jul 9, 2026

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

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.

Primary conditionUsher SyndromesRegistry: Retinitis PigmentosaReclassifiedWhy we changed it
Registry
Retinitis Pigmentosa — the first condition term the registry derived for this study.
ClinicalTrials.gg
Usher Syndromes
Why
  1. The study's listed conditions name Usher Syndromes, a narrower term within Retinitis Pigmentosa.
Confidence
High: the new condition is named in the study's own listed conditions. Of 351 hand-checked studies reclassified this way, 68% were exactly right and 84% in the right disease family; the registry's first term was right for 5% (measured 2026-10-21). How we classify →
Wrong?
Tell us the study's main condition →

Sponsored by Laboratoires Thea · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
81
Allocation
Randomized
Ages
8 Years and older
Sex
All
01

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.

Read the detailed description

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.

02

Conditions studied

  • Retinitis Pigmentosa (RP)
  • Usher Syndrome Type 2
  • Deaf Blind
  • Retinal Disease
  • Eye Diseases, Hereditary
  • Eye Disorders Congenital
  • Vision Disorders

Keywords

  • Retinitis Pigmentosa
  • Usher Syndrome Type 2
  • Deaf Blind
  • USH2A
  • RP
  • Exon 13
  • RNA therapies
  • antisense oligonucleotide
  • exon skipping
  • LUNA
  • IVT
  • NSRP
03

In context

Usher Syndromes Reclassified

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 →

Lead sponsor

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 →

04

Who can participate

Ages eligible
8 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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.

Inclusion criteria

  1. An adult (≥18 years) willing and able to provide 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

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.
Terms in these criteria, in plain language(1)

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.

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.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
81 participants (estimated)

Study arms

  • Experimental
    Ultevursen 180/60 μg

    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 comparator
    Sham Procedure

    Sham-procedure (no experimental drug administered)

    Other: No intervention, will not receive any active study intervention

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)

06

What researchers measure

Primary outcomes

  1. 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

Secondary outcomes

  1. Annualized change from baseline in static perimetry (SP) mean sensitivity

    Time frame: Month 24

  2. Annualized change from baseline in microperimetry (MP) mean sensitivity

    Time frame: Month 24

  3. Change from baseline in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart

    Time frame: Month 24

  4. Change from baseline in best-corrected visual acuity (BCVA) using the ETDRS chart

    Time frame: Month 24

  5. Percent change from baseline in EZ area by SD-OCT

    Time frame: Month 24

  6. Percent change from baseline in EZ width by SD-OCT

    Time frame: Month 24

  7. 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

  8. 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

  9. Frequency and severity of ocular and non-ocular adverse events (AEs)

    Time frame: Up to month 24

  10. Systemic Exposure

    Systemic serum concentration of ultevursen

    Time frame: Up to month 24

07

Study locations

26 sites
08

References and documents

Publications

  • Stephenson KAJ, Dhanji SR, Kolawole OU, Gregory-Evans CY, Gregory-Evans K. Ethnic disparities in inherited retinal degenerations. Can J Ophthalmol. 2025 Dec;60(6):e869-e878. doi: 10.1016/j.jcjo.2025.04.007. Epub 2025 Jun 6. PubMed 40339601 ↗

Individual participant data

Plan to share: No

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Dec 2024 (actual). Primary completion is estimated for Jul 2028, 43 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 25, 2024
First posted
Oct 4, 2024
StartActual
Dec 11, 2024
Primary completionEst.
Jul 2028
CompletionEst.
Jul 2028
Last update
Jul 9, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jul 9, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06627179
Lead sponsor
Laboratoires Thea
Collaborators
Sepul Bio
Responsible party
Sponsor
First posted
Oct 4, 2024
Start date
Dec 11, 2024
Primary completion
Jul 2028 (estimated)
Completion
Jul 2028 (estimated)
Last update
Jul 9, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

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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