A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Plain-language summary

This is a patient registry study to follow patients treated with the gene therapy voretigene neparvovec-rzyl in the US. Eligible participants received this gene therapy in at least one eye and provided informed consent. It was conducted as a study without a formal phase and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

Spark Therapeutics, Inc.

Intervention

AAV2-hRPE65v2,voretigene neparvovec-rzyl

Start date

1/10/2019

Estimated completion

6/30/2025

Countries

United States

Related genes

Locations

• Los Angeles, United States
• Miami, United States
• Iowa City, United States
• Boston, United States
• Ann Arbor, United States
• Cincinnati, United States
• Portland, United States
• Philadelphia, United States
• Philadelphia, United States
• Houston, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. Received voretigene neparvovec-rzyl in at least one eye.
2. Signed informed consent/assent (when applicable). These are obtained as required under institutional policies and applicable laws and regulations unless a consent waiver is obtained from the Institutional Review Board (IRB)/Independent Ethics Committee (IEC).

Exclusion Criteria:

1\. Previously participated in, or are currently participating in, a Spark Therapeutics clinical trial and received voretigene neparvovec-rzyl in both eyes.