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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
Ability to perform tests of visual and retinal function
Must not have
Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications
Use of immunosuppressive medications
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
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.
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.
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.
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.
Select...
I can perform tests for my eyesight and retina health.
Select...
I have a genetic eye condition caused by two RPE65 mutations.
Select...
I have a retinal disease caused by two RPE65 mutations.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I don't have eye conditions that would affect surgery or study results.
Select...
I am currently taking medications that suppress my immune system.
Select...
I am of childbearing age and not willing to use birth control.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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
2,084 Previous Clinical Trials
42,727,033 Total Patients Enrolled
National Eye Institute (NEI)NIH
558 Previous Clinical Trials
1,408,456 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.
Herpes simplex retinopathy Patient Testimony for trial: Trial Name: NCT00481546 — Phase 1