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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

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 below

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I have LCA/EORD with very poor vision in my study eye.

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I can perform tests for my eyesight and retina health.

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I have a genetic eye condition caused by two RPE65 mutations.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 15 years for reporting.

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.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of PennsylvaniaLead Sponsor

2,009 Previous Clinical Trials

42,894,638 Total Patients Enrolled

National Eye Institute (NEI)NIH

547 Previous Clinical Trials

1,402,142 Total Patients Enrolled

Samuel G. Jacobson, MD, PhDPrincipal InvestigatorUniversity of Pennsylvania

Media Library

rAAV2-CBSB-hRPE65 (Gene Vector) Clinical Trial Eligibility Overview. Trial Name: NCT00481546 — Phase 1

Retinal Disease Research Study Groups: Experimental

Retinal Disease Clinical Trial 2023: rAAV2-CBSB-hRPE65 Highlights & Side Effects. Trial Name: NCT00481546 — Phase 1

rAAV2-CBSB-hRPE65 (Gene Vector) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00481546 — Phase 1

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