Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
NCT ID: NCT06627179
Last Updated: 2026-02-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
81 participants
INTERVENTIONAL
2024-12-11
2027-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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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).
Intravitreal Injection of Ultevursen
Up to 4 doses over a 24-month period
Sham Procedure
Sham-procedure (no experimental drug administered)
No intervention, will not receive any active study intervention
Sham-procedure (no experimental drug administered)
Interventions
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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)
Eligibility Criteria
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Inclusion Criteria
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
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.
8 Years
ALL
No
Sponsors
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Sepul Bio
INDUSTRY
Laboratoires Thea
INDUSTRY
Responsible Party
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Locations
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The University of California, San Francisco
San Francisco, California, United States
Bascom Palmer Eye Institute/University of Miami
Miami, Florida, United States
Emory University
Atlanta, Georgia, United States
Massachusetts Eye and Ear
Boston, Massachusetts, United States
University of Michigan- Kellogg Eye Center
Ann Arbor, Michigan, United States
Casey Eye Institute, Oregon Health & Science University
Portland, Oregon, United States
University of Pennsylvania, Scheie Eye Institute
Philadelphia, Pennsylvania, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
University of Wisconsin- Madison
Madison, Wisconsin, United States
Ghent University Hospital
Ghent, , Belgium
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
São Paulo, , Brazil
Hospital for Sick Children
Toronto, Ontario, Canada
McGill University Health Centre for Innovative Medicine
Montreal, Quebec, Canada
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
AOU Università degli Studi della Campania Luigi Vanvitelli
Napoli, , 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, United Kingdom
University of Edinburgh / NHS Lothian
Edinburgh, , United Kingdom
Moorfields Eye Hosptial
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2024-515199-10-00
Identifier Type: CTIS
Identifier Source: secondary_id
SB-421a-006
Identifier Type: -
Identifier Source: org_study_id
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