← Back to Search

Virus Therapy

Gene Therapy for Leber Congenital Amaurosis

Phase 1 & 2

Waitlist Available

Research Sponsored by Spark Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Prior participation in Phase 1 study with unilateral, subretinal administration of AAV2-hRPE65v2

Sufficient viable retinal cells in contralateral, previously uninjected eye, as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy. Must have either: 1) an area of retina within the posterior pole of > 100 µm shown on OCT; 2) ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole; or 3) remaining visual field within 50 degrees of fixation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 15 years

Awards & highlights

Study Summary

This trial is to assess the safety and effectiveness of an experimental drug in people with a specific type of cancer.

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

While specific side effects aren't listed here, typical risks may include immune reactions due to the viral vector used for gene delivery, inflammation inside the eye, changes in intraocular pressure or vision disturbances post-treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Select...

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

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

Adverse events as a measure of safety and tolerability

Secondary outcome measures

Contrast sensitivity

Full-field light threshold sensitivity testing

Mobility testing

+3 more

Trial Design

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:

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

voretigene neparvovec-rzyl

2007

Completed Phase 1

~20

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor

14 Previous Clinical Trials

422 Total Patients Enrolled

2 Trials studying Leber Congenital Amaurosis

43 Patients Enrolled for Leber Congenital Amaurosis

Spark TherapeuticsLead Sponsor

13 Previous Clinical Trials

342 Total Patients Enrolled

2 Trials studying Leber Congenital Amaurosis

43 Patients Enrolled for Leber Congenital Amaurosis

Clinical DirectorStudy DirectorSpark Therapeutics, Inc.

17 Previous Clinical Trials

4,483 Total Patients Enrolled

1 Trials studying Leber Congenital Amaurosis

12 Patients Enrolled for Leber Congenital Amaurosis

Media Library

voretigene neparvovec-rzyl (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01208389 — Phase 1 & 2

Leber Congenital Amaurosis Research Study Groups: voretigene neparvovec-rzyl (AAV2-hRPE65v2)

Leber Congenital Amaurosis Clinical Trial 2023: voretigene neparvovec-rzyl Highlights & Side Effects. Trial Name: NCT01208389 — Phase 1 & 2

voretigene neparvovec-rzyl (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01208389 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could you provide a synopsis of the prior research conducted with voretigene neparvovec-rzyl?

"Presently, there are 2 live trials for voretigene neparvovec-rzyl with one in the advanced stage 3. While most of these studies occur in Philadelphia, Pennsylvania; other medical centres have also been enlisted to oversee the clinical trial process."

Answered by AI

Has a trial of this sort ever been conducted previously?

"Presently, there are two active investigations exploring the effectiveness of voretigene neparvovec-rzyl in two metropolitan areas. The initial study conducted by Spark Therapeutics began in 2010 and enrolled 12 patients to complete Phase 1 & 2 trials. Since this trial's completion a decade ago, one additional investigation has been completed."

Answered by AI

Does this medical experiment currently have open enrollment?

"According to clinicaltrials.gov, this experiment is not presently in need of participants - it was initially posted on November 1st 2010 and last updated on July 15th 2022. However, 28 other trials remain open for enrolment."

Answered by AI

How many participants are being enrolled for this research program?

"This clinical trial is currently not accepting participants. According to the data available, it was initially posted on November 1st 2010 and last updated on July 15th 2022. Other trials related to leber congenital amaurosis are recruiting now, with 26 studies looking for patients; similarly, there are two separate investigations concerning voretigene neparvovec-rzyl that require volunteers at this time."

Answered by AI

Recent research and studies

New England Journal of MedicineJournal

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Maguire, Albert M., Francesca Simonelli, Eric A. Pierce, Edward N. Pugh Jr., Federico Mingozzi, Jeannette Bennicelli, Sandro Banfi, et al.. 2008. “Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa0802315.

Molecular TherapyJournal

Gene Therapy for Leber's Congenital Amaurosis Is Safe and Effective Through 1.5 Years After Vector Administration

Simonelli, Francesca, Albert M Maguire, Francesco Testa, Eric A Pierce, Federico Mingozzi, Jeannette L Bennicelli, Settimio Rossi, et al.. 2010. “Gene Therapy for Leber's Congenital Amaurosis Is Safe and Effective Through 1.5 Years After Vector Administration”. Molecular Therapy. Elsevier BV. doi:10.1038/mt.2009.277.

The LancetJournal

Age-dependent Effects of RPE65 Gene Therapy for Leber's Congenital Amaurosis: A Phase 1 Dose-escalation Trial

Maguire, Albert M, Katherine A High, Alberto Auricchio, J Fraser Wright, Eric A Pierce, Francesco Testa, Federico Mingozzi, et al.. 2009. “Age-dependent Effects of RPE65 Gene Therapy for Leber's Congenital Amaurosis: A Phase 1 Dose-escalation Trial”. The Lancet. Elsevier BV. doi:10.1016/s0140-6736(09)61836-5.

Science Translational MedicineJournal