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Sepofarsen for Leber Congenital Amaurosis (BRIGHTEN Trial)
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.
- 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 belowBRIGHTEN Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.BRIGHTEN Trial Design
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Who is running the clinical trial?
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- 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.
- 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
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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.
Frequently Asked Questions
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."
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."
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."
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."
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 _______."
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."
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