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Gene Therapy
Subretinal Injection for Leber Congenital Amaurosis
Phase 1 & 2
Waitlist Available
Research Sponsored by Atsena Therapeutics Inc.
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 baseline to week 52 and baseline to week 260
Awards & highlights
Study Summary
This trial is testing a new drug for Leber Congenital Amaurosis, a rare disease that causes blindness. The drug is being injected into the retina in order to see if it is safe and effective.
Who is the study for?
This trial is for adults and children with Leber Congenital Amaurosis (LCA) due to specific GUCY2D gene mutations. Adults must have very limited vision, while kids can have slightly better sight. Participants need some retinal structure visible on scans and agree to contraception and not donating biological materials post-treatment.Check my eligibility
What is being tested?
The study tests different doses of SAR439483, injected under the retina, for safety and effectiveness in LCA patients with GUCY2D mutations. It includes a single injection followed by monitoring. Other medications like steroids may be used to manage side effects or prepare the eye.See study design
What are the potential side effects?
Potential side effects might include discomfort from the injection, inflammation inside the eye, increased eye pressure, cataracts, or other changes affecting vision. Systemic side effects could occur due to steroid use such as mood swings, weight gain, or increased blood sugar.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to week 52 and baseline to week 260
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 52 and baseline to week 260
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of participants with AEs from baseline up to the end of the safety follow-up period
Number of participants with adverse events (AEs) from baseline up to the end of the observation period
Secondary outcome measures
Change in best -corrected visual acuity (BCVA)
Change in sensitivity
Trial Design
1Treatment groups
Experimental Treatment
Group I: ATSN-101Experimental Treatment6 Interventions
ATSN-101 single dose according to an ascending dose design (dose escalation phase) or ATSN-101 single dose (dose expansion phase)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisone
2014
Completed Phase 4
~2370
Find a Location
Who is running the clinical trial?
Atsena Therapeutics Inc.Lead Sponsor
1 Previous Clinical Trials
18 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am not using, nor plan to use, any anti-viral therapy that could affect the trial treatment.I have Leber congenital amaurosis due to GUCY2D gene mutations.My eyes are healthy enough for surgery and won't affect the study's outcome.I haven't had any eye procedures in the last 6 months that would affect my surgery.My eyes are healthy enough for surgery and follow-up without affecting the study.I am currently taking medications that suppress my immune system.I am not allergic to the study drug or related medications.I am at least 18 years old for Cohorts 1-4, or between 6 and 17 years old for Cohort 5.My tests show mutations in the GUCY2D gene from a certified lab.I have not used blood thinners in the last two weeks.I have no allergies or health issues that prevent surgery or anesthesia use.I am not pregnant, lactating, or breastfeeding.I have not had a serious illness or infection in the last 28 days.I agree to follow the trial's birth control requirements.I do not have any health conditions that weaken my immune system or would interfere with the study's treatment.I have not had gene therapy in the last 15 years.
Research Study Groups:
This trial has the following groups:- Group 1: ATSN-101
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.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there currently any open spots for enrollment in this research endeavor?
"Sadly, clinicaltrials.gov displays that this medical trial is not presently recruiting any participants, despite its original post date of September 12th 2019 and last edit on June 6th 2022. However, there are 434 other trials enrolling patients right now."
Answered by AI
What is the scale of cohort recruitment for this research endeavor?
"At this moment, recruitment for the study has been suspended. It was initially posted on September 12th 2019 and last updated June 6th 2022. Fortunately, there are currently 26 other clinical trials that have open enrolment for leber congenital amaurosis and 408 studies searching for participants to be treated with Prednisone."
Answered by AI
Has Prednisone been subject to any previous clinical investigations?
"At present, there are 408 ongoing trials involving Prednisone and 117 of those fall under Phase 3. With 17068 locations running studies, several sites are in Duarte, California."
Answered by AI
What medical conditions can be alleviated by Prednisone?
"Prednisone can be utilised to alleviate synovitis as well as afflictions such as ophthalmia, sympathetic, bacterial conjunctivitis, and thyroiditis."
Answered by AI
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