NCT03597399

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 87 (actual)
• Sponsor: Spark Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Months
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

Interventions

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

Timeline

• Start date: 2019-01-10
• Primary completion: 2025-06-30
• Completion: 2025-06-30
• First posted: 2018-07-24
• Last updated: 2025-09-22

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study