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

NCT03597399 | Completed FDA Drug

• 1 other identifier: SPKRPE-PASS
• Study Type: observational
• Target: 87
• Locations: 1 country
• Sites: 10

Brief Summary

The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products. The enrollment period will last for two years from the first treatment following product approval (through 31March2020) and include a minimum of 40 patients.

Conditions

Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

Keywords

Leber Congenital Amaurosis (LCA); Inherited Retinal Dystrophy; RPE65; Retinitis Pigmentosa (RP)

Outcome Measures

Primary Outcomes (1)

• Collection of all Adverse Events and Serious Adverse Events

  adverse events

  up to 5 years

Secondary Outcomes (1)

• Collection of Pregnancy Outcomes

  Up to 5 years

Interventions

AAV2-hRPE65v2,voretigene neparvovec-rzyl BIOLOGICAL

Subretinal administration of gene therapy vector AAV2-hRPE65v2 (voretigene neparvovec-rzyl) to both eyes via surgical procedures on separate days.

Also known as: AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector

Eligibility Criteria

• Age: 12 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• Received voretigene neparvovec-rzyl in at least one eye.
• 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).

You may not qualify if:

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

Sponsors & Collaborators

• Spark Therapeutics, Inc.: lead

Study Sites (10)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States

Location

Massachusetts Eye and Ear Institute

Boston, Massachusetts, 02114, United States

Location

Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

Location

Casey Eye Institute

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

Location

Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

Cullen Eye Institute

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Leber Congenital Amaurosis; Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Ophthalmic Lead

  Spark Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 12, 2018
• First Posted: July 24, 2018
• Study Start: January 10, 2019
• Primary Completion: June 30, 2025
• Study Completion: June 30, 2025
• Last Updated: September 22, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)