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Antisense Oligonucleotide

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)

Phase 2 & 3
Waitlist Available
Research Sponsored by Laboratoires Thea
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 27 months

Summary

This trial is testing a new eye injection drug called QR-421a for patients with a specific genetic form of Retinitis Pigmentosa. The drug aims to correct the genetic mutation causing their vision loss, potentially slowing or stopping the disease.

Eligible Conditions
  • Retinitis Pigmentosa
  • None
  • Eye Conditions
  • Usher Syndrome
  • Congenital Eye Disorders
  • Retinal Disease
  • Visual Impairment
  • Deafblind
  • Eye Diseases
  • Vision Impairment

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~27 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 27 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change From Baseline in Mean Sensitivity
Secondary study objectives
Change From Baseline in Best Corrected Visual Acuity (BCVA)
Change From Baseline in Full-field Stimulus Threshold (FST)
Change From Baseline in Low Luminance Visual Acuity (LLVA)
+8 more
Other study objectives
Change From Baseline in Mobility Course Score

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: QR-421a 60/60 µgExperimental Treatment1 Intervention
60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
Group II: QR-421a 180/60 µgExperimental Treatment1 Intervention
180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter
Group III: Sham-procedurePlacebo Group1 Intervention
Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
QR-421a
2019
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Laboratoires TheaLead Sponsor
62 Previous Clinical Trials
6,899 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
109 Patients Enrolled for Retinitis Pigmentosa
Sepul BioUNKNOWN
5 Previous Clinical Trials
129 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
109 Patients Enrolled for Retinitis Pigmentosa
ProQR TherapeuticsLead Sponsor
11 Previous Clinical Trials
218 Total Patients Enrolled
4 Trials studying Retinitis Pigmentosa
59 Patients Enrolled for Retinitis Pigmentosa
ProQR Clinical Trial ManagerStudy DirectorProQR Therapeutics
3 Previous Clinical Trials
38 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
38 Patients Enrolled for Retinitis Pigmentosa
Sepul Bio Chief Medical OfficerStudy DirectorSepul Bio
2 Previous Clinical Trials
20 Total Patients Enrolled
Sepul Bio Clinical Operations DirectorStudy DirectorSepul Bio
1 Previous Clinical Trials
7 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
7 Patients Enrolled for Retinitis Pigmentosa
ProQR Medical MonitorStudy DirectorProQR Therapeutics
7 Previous Clinical Trials
98 Total Patients Enrolled
3 Trials studying Retinitis Pigmentosa
38 Patients Enrolled for Retinitis Pigmentosa
~1 spots leftby Jun 2025
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