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

A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
• Intervention / Treatment: Biological: AAV2-hRPE65v2,voretigene neparvovec-rzyl

Detailed Description

Voretigene neparvovec-rzyl is a gene therapy intended for use in individuals with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells. Mutations in the RPE65 gene are associated with several clinical manifestations including nyctalopia, decreased visual field and decreased visual acuity. Voretigene neparvovec-rzyl uses a non-pathogenic recombinant adeno-associated virus vector serotype 2 (AAV2) to deliver cDNA encoding RPE65 protein to target cells in the retina. Voretigene neparvovec-rzyl is administered to each eye via subretinal injection. The administration of voretigene neparvovec-rzyl is recommended to be performed to each eye on separate days within a close interval. Prescribing information recommends an immunomodulatory regimen concomitant with administration, with the actual regimen dependent upon the dosing center.

This post authorization safety study will focus on further characterizing the long-term safety profile of voretigene neparvovec-rzyl in patients with RPE65 mutation-associated retinal dystrophy using an observational, longitudinal design.

• Study Type: Observational
• Enrollment (Actual): 87

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital of Los Angeles
  • Florida
    • Miami, Florida, United States, 33136
      • Bascom Palmer Eye Institute
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals & Clinics
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts Eye and Ear Institute
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Kellogg Eye Center
  • Ohio
    • Cincinnati, Ohio, United States, 45242
      • Cincinnati Eye Institute
  • Oregon
    • Portland, Oregon, United States, 97239
      • Casey Eye Institute
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Scheie Eye Institute
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia (CHOP)
  • Texas
    • Houston, Texas, United States, 77030
      • Cullen Eye Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Collection of all Adverse Events and Serious Adverse Events	adverse events	up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Collection of Pregnancy Outcomes	Follow pregnancy outcomes in participants (and female partners of male participants) who received voretigene neparvovec	Up to 5 years

Collaborators and Investigators

• Sponsor: Spark Therapeutics
• Investigators: Study Director: Clinical Ophthalmic Lead, Spark Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): January 10, 2019
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: July 12, 2018
• First Submitted That Met QC Criteria: July 12, 2018
• First Posted (Actual): July 24, 2018

Study Record Updates

• Last Update Posted (Actual): January 23, 2024
• Last Update Submitted That Met QC Criteria: January 22, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: RPE65; Inherited Retinal Dystrophy; Leber Congenital Amaurosis (LCA); Retinitis Pigmentosa (RP)
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Retinal Dystrophies
• Other Study ID Numbers: SPKRPE-PASS

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes