New eye injection aims to slow blindness in rare genetic disease

NCT ID NCT06627179

Disease control Sponsor: Laboratoires Thea Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 10, 2026 · Updated 4 times

Summary

This study tests an experimental drug called ultevursen for people with retinitis pigmentosa caused by a specific gene mutation (USH2A). The drug is injected into the eye and may help slow vision loss. The trial involves 81 participants, some of whom will receive a sham (fake) injection for comparison. The study lasts two years and measures changes in the retina and vision.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Ultevursen (given as an eye injection)
What this could lead to: If it works, this could slow vision loss in people with a specific genetic form of retinitis pigmentosa.
What could go wrong: This is an early Phase 2 trial with only 81 people. The drug is injected into the eye, which carries risks like infection or inflammation. It may not improve vision or could cause side effects.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 81 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

Jul 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

8 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Eye Abnormalities Eye Diseases, Hereditary eye disorder retinal disorder retinitis pigmentosa retinitis pigmentosa 39 Usher syndrome Usher syndrome type 2 vision disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Deaf blind Eye diseases, hereditary Eye disorders congenital Retinal disease Retinitis pigmentosa (RP) Usher syndrome type 2 Vision disorders

Contacts and locations

Locations

ASST Santi Paolo e Carlo Hospital, University of Milan

Milan, 20142, Italy
Amsterdam University Medical Center - Locatie AMC

Amsterdam, 1105 AZ, Netherlands
Bascom Palmer Eye Institute/University of Miami

Miami, Florida, 33136, United States
Baylor College of Medicine

Houston, Texas, 77030, United States
Casey Eye Institute, Oregon Health & Science University

Portland, Oregon, 97239, United States
Centre de maladies rares CHNO des Quinze Vingt

Paris, 75012, France
Emory University

Atlanta, Georgia, 30322, United States
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)

São Paulo, 04021-001, Brazil
Ghent University Hospital

Ghent, B-9000, Belgium
Het Oogziekenhuis Rotterdam

Rotterdam, 3011 BH, Netherlands
Hospital for Sick Children

Toronto, Ontario, M5G1E8, Canada
Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique

Montpellier, 34295, France
INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

Belo Horizonte, 30150270, Brazil
Massachusetts Eye and Ear

Boston, Massachusetts, 02114, United States
McGill University Health Centre for Innovative Medicine

Montreal, Quebec, H4A3J1, Canada
Moorfields Eye Hosptial

London, EC1V 2PD, United Kingdom
Oxford Eye Hospital

Headington, Oxford, OX3 9DU, United Kingdom
Radboud Universitair Medisch Centrum

Nijmegen, 6525 GA, Netherlands
Retina Foundation of the Southwest

Dallas, Texas, 75231, United States
Rigshospitalet and University of Copenhagen

Glostrup Municipality, 2600, Denmark
The University of California, San Francisco

San Francisco, California, 94143, United States
University of Edinburgh / NHS Lothian

Edinburgh, EH39HA, United Kingdom
University of Michigan- Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States
University of Pennsylvania, Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States
University of Wisconsin- Madison

Madison, Wisconsin, 53705, United States
Universitätsklinikum Tübingen

Tübingen, 72076, Germany

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