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

Part of paid clinical trials in San Francisco, California.

Sponsor
Laboratoires Thea
Study ID
NCT06627179
Phase
PHASE2
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
8 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • 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)

Study Details

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.

Key Dates

Start date
Dec 11, 2024
Status verified
Jul 2025
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
81 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

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).
  • Sham Comparator: Sham Procedure
    Sham-procedure (no experimental drug administered)

Primary Outcome Measure

To evaluate efficacy after 24 months of treatment [ Time Frame: 24 Months ]

Central Contacts

Locations (11)

FacilityCityStateZIPSite coordinators
The University of California, San FranciscoSan FranciscoCalifornia94143-
Bascom Palmer Eye Institute/University of MiamiMiamiFlorida33136-
Emory UniversityAtlantaGeorgia30322-
Massachusetts Eye and EarBostonMassachusetts02114-
University of Michigan- Kellogg Eye CenterAnn ArborMichigan48105-
Duke Eye CenterDurhamNorth Carolina27705-
Casey Eye Institute, Oregon Health & Science UniversityPortlandOregon97239-
University of Pennsylvania, Scheie Eye InstitutePhiladelphiaPennsylvania19104-
Retina Foundation of the SouthwestDallasTexas75231-
Baylor College of MedicineHoustonTexas77030-
University of Wisconsin- MadisonMadisonWisconsin53705-

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By research site
The University of California, San Francisco· San Francisco, CABascom Palmer Eye Institute/University of Miami· Miami, FLEmory University· Atlanta, GAMassachusetts Eye and Ear· Boston, MAUniversity of Michigan- Kellogg Eye Center· Ann Arbor, MIDuke Eye Center· Durham, NC

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