Gene Therapy for Leber Congenital Amaurosis

Recruiting at 1 trial location

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Spark Therapeutics, Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.

Research Team

Stephen R Russell, MD

Principal Investigator

University of Iowa

Albert M Maguire, MD

Principal Investigator

Children's Hospital of Philadelphia

Eligibility Criteria

This trial is for individuals aged three or older with Leber Congenital Amaurosis (LCA) due to RPE65 mutations, confirmed by a lab. Participants must have poor vision but enough viable retinal cells and be able to perform mobility tests. Pregnant individuals or those not using contraception, with prior gene therapy, recent investigational drug use, or conditions affecting the study's outcome cannot join.

Inclusion Criteria

Willingness to adhere to protocol and long-term follow-up as evidenced by written informed consent or parental permission and subject assent (where applicable)

You have enough healthy cells in your eyes, as checked by non-invasive tests like optical coherence tomography (OCT) and ophthalmoscopy.

I am at least three years old.

Exclusion Criteria

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

Participation in a clinical study with an investigational drug in the past six months

I don't have eye conditions or other illnesses that would affect surgery.

Treatment Details

Interventions

• AAV2-hRPE65v2,voretigene neparvovec-rzyl (Gene Therapy)

Trial Overview The trial studies the safety and effectiveness of a gene therapy called AAV2-hRPE65v2 (voretigene neparvovec-rzyl). It's given as an injection into the eye to see if it can improve sight in people with specific genetic changes causing LCA. The treatment will be administered at select hospitals.

Participant Groups

Group I: AAV2-hRPE65v2,voretigene neparvovec-rzyl Experimental Treatment 1 Intervention

voretigene neparvovec rzyl, 1.5 E11 vector genomes, per eye, administered by subretinal injection in a volume of 0.3mL, 6-18 days apart

Group II: Control Active Control 1 Intervention

No intervention

Who Is Running the Clinical Trial?

Spark Therapeutics, Inc.

Lead Sponsor

Trials

16

Recruited

410+

Spark Therapeutics

Lead Sponsor

Trials

14

Recruited

350+

Children's Hospital of Philadelphia

Collaborator

Trials

749

Recruited

11,400,000+

University of Iowa

Collaborator

Trials

486

Recruited

934,000+