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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
Be younger than 18 years old
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
- Retinal Disease
- Vision Impairment
- Retinal Degeneration
- Leber Congenital Amaurosis
- Retinal Dystrophy
- Neurologic Symptoms
- Eye Diseases
- Congenital Eye Disorders
- Blindness
- Sensory Disorders
BRIGHTEN Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
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
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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
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have received treatments in the past year that affect the immune system.You have taken or are planning to take medications that can harm your eyes within the past 3 months.Children under 8 years old with a specific genetic mutation called CEP290 p.Cys998X, confirmed by a genotyping test.Your vision in the eye being treated is better than a certain level on a vision chart but not too good.The outer nuclear layer in the macula area can be seen.
Research Study Groups:
This trial has the following groups:- Group 1: Group 2 - open label
- Group 2: Group 1 - open label
- Group 3: Group 3: open label
- Group 4: Group 4: double-masked, randomized to one of 2 dose cohorts
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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