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Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

NCT03780257 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2022-04-20

No results posted yet for this study

Summary

The purpose of this study is to evaluate the 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.

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 (dose cohort 1&2 only)

Sham-procedure (no experimental drug administered)

Sponsors & Collaborators

  • ProQR Therapeutics

    lead INDUSTRY

Principal Investigators

  • ProQR Medical Monitor · ProQR Therapeutics

  • ProQR Clinical Trial Manager · ProQR Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-03-06
Primary Completion
2021-10-14
Completion
2021-10-14
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France

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 NCT03780257 on ClinicalTrials.gov