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Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

NCT05176717 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2024-08-07

Study results available
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Summary

The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss.

Conditions

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

Interventions

DRUG

QR-421a

RNA antisense oligonucleotide for intravitreal injection

OTHER

Sham-procedure

Sham-procedure (no experimental drug administered)

Sponsors & Collaborators

  • Sepul Bio

    collaborator INDUSTRY

  • Laboratoires Thea

    lead INDUSTRY

Principal Investigators

  • Sepul Bio Chief Medical Officer · Sepul Bio

  • Sepul Bio Clinical Operations Director · Sepul Bio

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-15
Primary Completion
2022-08-02
Completion
2022-08-02
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05176717 on ClinicalTrials.gov