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Antisense Oligonucleotide

Sepofarsen for Leber Congenital Amaurosis (BRIGHTEN Trial)

Phase 2 & 3
Waitlist Available
Research Sponsored by ProQR Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female child, <8 years of age at Screening with a clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation, based on genotyping analysis at Screening. A historic genotyping report from a certified laboratory are acceptable with Sponsor approval.
Detectable outer nuclear layer (ONL) in the area of the macula.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

BRIGHTEN Trial Summary

This trial is testing a new treatment for a rare genetic eye disease called Leber Congenital Amaurosis. The treatment is given as an injection in the eye, and the trial will track safety and tolerability over 2 years.

Eligible Conditions
  • Vision Impairment
  • Retinal Disease
  • Retinal Degeneration
  • Leber Congenital Amaurosis
  • Retinal Dystrophy
  • Neurologic Symptoms
  • Eye Diseases
  • Congenital Eye Disorders
  • Blindness
  • Sensory Disorders

BRIGHTEN Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
Children under 8 years old with a specific genetic mutation called CEP290 p.Cys998X, confirmed by a genotyping test.
Select...
The outer nuclear layer in the macula area can be seen.

BRIGHTEN Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence and severity of non-ocular adverse events (AEs)
Incidence and severity of ocular adverse events (AEs)
Secondary outcome measures
Change from baseline to Month 12 in Best-corrected visual acuity (BCVA)
Change from baseline to Month 12 in retinal sensitivity measured by Full-field stimulus testing (FST)

BRIGHTEN Trial Design

4Treatment groups
Experimental Treatment
Group I: Group 4: double-masked, randomized to one of 2 dose cohortsExperimental Treatment1 Intervention
Group II: Group 3: open labelExperimental Treatment1 Intervention
Group III: Group 2 - open labelExperimental Treatment1 Intervention
Group IV: Group 1 - open labelExperimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

ProQR TherapeuticsLead Sponsor
11 Previous Clinical Trials
282 Total Patients Enrolled
ProQR Medical MonitorStudy DirectorProQR Therapeutics
7 Previous Clinical Trials
162 Total Patients Enrolled

Media Library

Sepofarsen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04855045 — Phase 2 & 3
Vision Impairment Research Study Groups: Group 2 - open label, Group 1 - open label, Group 3: open label, Group 4: double-masked, randomized to one of 2 dose cohorts
Vision Impairment Clinical Trial 2023: Sepofarsen Highlights & Side Effects. Trial Name: NCT04855045 — Phase 2 & 3
Sepofarsen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04855045 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How does one go about joining this trial?

"This study is testing a new medication for amaurosis in children aged seven and younger. So far, around 15 young patients have been accepted into the trial."

Answered by AI

Could you please provide information about other times sepofarsen has been tested in a clinical setting?

"3 clinical trials are currently underway to study the efficacy of sepofarsen. Of these ongoing investigations, 2 have progressed to Phase 3. Although most research on sepofarsen is based in Strasbourg and Iowa, 32 different locations across the globe are running studies related to this treatment."

Answered by AI

Can new patients join this clinical trial?

"That is correct, the listing on clinicaltrials.gov indicates that this study is still open to new participants. The trial was posted on March 23rd, 2021 and updated March 24th, 2022. There is space for 15 more patients at a single location."

Answered by AI

Is this research novel in any way?

"Since 2019, sepofarsen has been the subject of 3 clinical trials taking place in 11 cities and 9 countries. The earliest study was conducted in 2019 by ProQR Therapeutics. Following that initial Phase 1 study involving 36 patients, sepofarsen received approval for Phases 2 & 3."

Answered by AI

Are young adults eligible for this clinical study?

"Children aged 0 to 7 are the only patients eligible for this particular trial. Out of 305 trials that exist for people under 18 and 862 trials meant for those 65 and older, this is the only one _______."

Answered by AI

How many research subjects are a part of this investigation?

"That is correct. The website clinicaltrials.gov has the latest information on this matter. According to their records, this study was first posted on March 23rd, 2021 and was edited most recently on March 24th, 2022. They are looking for 15 participants total from 1 location."

Answered by AI
~4 spots leftby Mar 2025