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

Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03597399
• Other study ID #: SPKRPE-PASS
• Status: Active, not recruiting
• Start date: January 10, 2019
• Est. completion date: June 2025

Study information

• Verified date: January 2024
• Source: Spark Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 87
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months and older

Eligibility

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.

Related Conditions & MeSH terms

• Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
• Retinal Dystrophies

Intervention

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.

Locations

Country	Name	City	State
United States	Kellogg Eye Center	Ann Arbor	Michigan
United States	Massachusetts Eye and Ear Institute	Boston	Massachusetts
United States	Cincinnati Eye Institute	Cincinnati	Ohio
United States	Cullen Eye Institute	Houston	Texas
United States	University of Iowa Hospitals & Clinics	Iowa City	Iowa
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Bascom Palmer Eye Institute	Miami	Florida
United States	Children's Hospital of Philadelphia (CHOP)	Philadelphia	Pennsylvania
United States	Scheie Eye Institute	Philadelphia	Pennsylvania
United States	Casey Eye Institute	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Spark Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Collection of all Adverse Events and Serious Adverse Events	adverse events	up to 5 years
Secondary	Collection of Pregnancy Outcomes	Follow pregnancy outcomes in participants (and female partners of male participants) who received voretigene neparvovec	Up to 5 years