A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

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.

Description

Eligibility

Age range

8+ years

Sex

All

Healthy volunteers

No

Interventions

• Drug
  Intravitreal Injection of Ultevursen

  Up to 4 doses over a 24-month period

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

  Sham-procedure (no experimental drug administered)

Locations (27)