Gene Therapy for Leber Congenital Amaurosis

Recruiting in Palo Alto (17 mi)

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Spark Therapeutics, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?The study is a follow-on to a Phase 1 dose-escalation and safety study.

Eligibility Criteria

This trial is for individuals with Leber Congenital Amaurosis who have participated in a prior Phase 1 study and received treatment in one eye. They must have some visual acuity, viable retinal cells in the other eye, and be willing to follow long-term protocols. Pregnant individuals or those not using effective contraception are excluded, as well as anyone with conditions that could affect the study's outcome.

Inclusion Criteria

I have previously been in a study where I received a specific gene therapy in one eye.

You can see light or have better vision.

My other eye has enough healthy retina cells, confirmed by eye scans.

+1 more

Exclusion Criteria

I do not have eye conditions or systemic diseases that could affect my eyes or interfere with the surgery.

My gender, race, or ethnicity does not affect my eligibility.

Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study

+6 more

Participant Groups

The trial is testing voretigene neparvovec-rzyl, a gene therapy previously administered to one eye of participants. Now it will be given to their other eye which hasn't been treated yet. The focus is on safety following this second administration.

1Treatment groups

Experimental Treatment

Group I: voretigene neparvovec-rzyl (AAV2-hRPE65v2)Experimental Treatment1 Intervention

Administration of study agent (AAV2-hRPE65v2) to the previously, uninjected contralateral eye: