Gene therapy safety check: luxturna registry wraps up

NCT ID NCT03597399

Completed Knowledge-focused Sponsor: Spark Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 2 times

Summary

This completed study tracked 87 patients who received Luxturna gene therapy for a rare inherited eye condition that causes blindness. Researchers monitored side effects and pregnancy outcomes for up to 5 years after treatment. The goal was to gather real-world safety data, not to test if the therapy works.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: voretigene neparvovec (Luxturna) gene therapy
What this could lead to: If successful, this registry confirms the long-term safety of Luxturna, supporting its continued use for this rare blinding condition.
What could go wrong: This is an observational registry, not a controlled trial, so it cannot prove effectiveness. Results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Leber congenital amaurosis retinitis pigmentosa RPE65-related recessive retinopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY

Contacts and locations

Locations

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States
Casey Eye Institute

Portland, Oregon, 97239, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States
Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States
Cullen Eye Institute

Houston, Texas, 77030, United States
Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States
Massachusetts Eye and Ear Institute

Boston, Massachusetts, 02114, United States
Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States
University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States