Clinical trial NCT03597399: A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Official title: A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Keywords: Leber Congenital Amaurosis (LCA)
Inherited Retinal Dystrophy
RPE65
Retinitis Pigmentosa (RP)
Lead sponsor: Spark Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-07-24
Enrollment: 87
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Individuals who received voretigene neparvovec-rzyl in at least one eye.

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Collection of all Adverse Events and Serious Adverse Events
timeFrame: up to 5 years
Secondary outcomes:
measure: Collection of Pregnancy Outcomes
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Miami
country: United States
facility: Bascom Palmer Eye Institute
state: Florida
city: Iowa City
country: United States
facility: University of Iowa Hospitals & Clinics
state: Iowa
city: Boston
country: United States
facility: Massachusetts Eye and Ear Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: Kellogg Eye Center
state: Michigan
city: Cincinnati
country: United States
facility: Cincinnati Eye Institute
state: Ohio
city: Portland
country: United States
facility: Casey Eye Institute
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia (CHOP)
state: Pennsylvania
city: Philadelphia
country: United States
facility: Scheie Eye Institute
state: Pennsylvania
city: Houston
country: United States
facility: Cullen Eye Institute
state: Texas
Study officials:
affiliation: Spark Therapeutics, Inc.
name: Clinical Ophthalmic Lead
role: STUDY_DIRECTOR
References: