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Gene Vector
Gene Therapy for Leber Congenital Amaurosis (LCA Trial)
Phase 1
Waitlist Available
Led By Samuel G. Jacobson, MD, PhD
Research Sponsored by University of Pennsylvania
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Clinical diagnosis of Leber congenital amaurosis (LCA)/early-onset retinal degeneration (EORD) and of severely impaired visual and retinal function, and best corrected visual acuity of 20/40 or worse in the study eye
Specific for Cohorts 1, 2 and 4: 18 years of age and older
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 15 years
Awards & highlights
LCA Trial Summary
This trial will help determine if a gene-carrying virus is safe for humans with a certain eye disease, which could pave the way for treatments of other eye diseases in the future.
Who is the study for?
This trial is for individuals with RPE65-associated retinal disease, which can lead to severe vision loss. It's open to adults and children (8-17 years after safety confirmation in adults). Participants must have a visible photoreceptor layer on OCT scans, be able to perform visual tests, and comply with research procedures. They should not have complicating diseases or conditions that could interfere with the study.Check my eligibility
What is being tested?
The trial is testing a gene therapy called rAAV2-CBSB-hRPE65 delivered directly under the retina. The goal is to restore vision in those affected by certain genetic mutations causing retinal disease. This early-phase study will assess safety across different age groups before and after treatment.See study design
What are the potential side effects?
Potential side effects are not detailed but may include ocular discomfort or inflammation from subretinal injections, systemic immune reactions due to viral vector use, and other unforeseen complications related to gene therapy.
LCA Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have LCA/EORD with very poor vision in my study eye.
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I am 18 years old or older.
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I can perform tests for my eyesight and retina health.
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I am between 8 and 17 years old.
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I have a genetic eye condition caused by two RPE65 mutations.
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I am in good health overall.
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I have a retinal disease caused by two RPE65 mutations.
LCA Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 15 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~15 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
The primary safety endpoint in this trial is the standard ocular examination. Toxicity will also be assessed by measurement of vision, hematology and serum chemistries, assays for vector genomes, reported subject history of symptoms and adverse events.
Secondary outcome measures
Visual function will be quantified prior to and after vector administration in order to determine whether vector administration affects visual function.
LCA Trial Design
1Treatment groups
Experimental Treatment
Group I: ExperimentalExperimental Treatment1 Intervention
All clinical trial subjects received the same vector.
Find a Location
Who is running the clinical trial?
University of PennsylvaniaLead Sponsor
2,000 Previous Clinical Trials
42,880,073 Total Patients Enrolled
National Eye Institute (NEI)NIH
546 Previous Clinical Trials
1,401,913 Total Patients Enrolled
Samuel G. Jacobson, MD, PhDPrincipal InvestigatorUniversity of Pennsylvania
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your body has a high level of AAV antibodies before starting the study.I don't have eye conditions that would affect surgery or study results.I am 18 years old or older.I have LCA/EORD with very poor vision in my study eye.You have recently taken a new experimental biological treatment.You are in overall good health.You have low levels of tetanus antibody in your blood, which means your immune system may not be working properly.I am between 8 and 17 years old.I have a genetic eye condition caused by two RPE65 mutations.I am in good health overall.Your eye scan shows a visible photoreceptor layer.I am currently taking medications that suppress my immune system.I can perform tests for my eyesight and retina health.I am of childbearing age and not willing to use birth control.I do not have any major illnesses besides my current condition.I haven't taken any blood-thinning medications in the last 7 days.I have a retinal disease caused by two RPE65 mutations.
Research Study Groups:
This trial has the following groups:- Group 1: Experimental
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.
Retinal Disease Patient Testimony for trial: Trial Name: NCT00481546 — Phase 1
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is enrollment now available for this experiment?
"Clinicaltrials.gov states that this trial is not currently accepting new participants, despite being initially posted on July 1st 2007 and last edited on December 13th 2021. Nevertheless, there are 116 other trials actively seeking volunteers."
Answered by AI
Are there substantial risks associated with using rAAV2-CBSB-hRPE65?
"As this is a Phase 1 trial, with minimal data supporting efficacy and safety, our internal team at Power has assigned rAAV2-CBSB-hRPE65 a score of 1."
Answered by AI
Who else is applying?
What state do they live in?
Florida
What site did they apply to?
Shands Children's Hospital, University of Florida
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
3+
What questions have other patients asked about this trial?
When would trial start?
PatientReceived no prior treatments
Why did patients apply to this trial?
I have wet AMD in right eye and Dry AMD in both eyes. I need to stop, hopefully reverse to some extent my vision problems.
PatientReceived no prior treatments
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