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Gene therapy safety check: luxturna registry wraps up

NCT ID NCT03597399

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Participants

87 people

The number who actually took part.

Started

Jan 2019

Finished

Jun 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Individuals who received voretigene neparvovec-rzyl in at least one eye.

Ages

12 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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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