Clinical Trials Directory

Trials / Terminated

TerminatedNCT05158296

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

A Double-Masked, Randomized, Controlled, Multiple-Dose 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 (Sirius)

Status
Terminated
Phase
Phase 2 / Phase 3
Study type
Interventional
Enrollment
7 (actual)
Sponsor
Laboratoires Thea · Industry
Sex
All
Age
12 Years
Healthy volunteers
Not accepted

Summary

The purpose of this study is to evaluate the efficacy 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.

Detailed description

The purpose of this study is to evaluate the efficacy 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. The below dose levels of ultevursen will be evaluated with the loading dose administered at Day 1 and maintenance dose administered at Month 3 and every 6 months thereafter: 1. Loading dose of 60 µg, maintenance dose of 60 µg 2. Loading dose of 180 µg, maintenance dose of 60 µg Dose levels will include subjects randomized to sham-procedure or treatment with ultevursen.

Conditions

Interventions

TypeNameDescription
DRUGUltevursenRNA antisense oligonucleotide for intravitreal injection
OTHERSham-procedureSham-procedure (no experimental drug administered)

Timeline

Start date
2021-12-08
Primary completion
2022-10-12
Completion
2022-10-12
First posted
2021-12-15
Last updated
2024-07-12
Results posted
2024-07-12

Locations

15 sites across 4 countries: United States, Germany, Netherlands, United Kingdom

Regulatory

Source: ClinicalTrials.gov record NCT05158296. Inclusion in this directory is not an endorsement.