A Patient Registry Study for Patients Treated With Voretigene Neparvovec



Status:Recruiting
Healthy:No
Age Range:Any
Updated:3/14/2019
Start Date:January 10, 2019
End Date:August 2028
Contact:Clinical Study Lead
Email:clinicaltrials@sparktx.com
Phone:1-215-220-9300

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

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.

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.

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.
We found this trial at
9
sites
900 Northwest 17th Street
Miami, Florida 33136
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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6565 Fannin Street
Houston, Texas 77030
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Houston, TX
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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Ann Arbor, Michigan 48105
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Ann Arbor, MI
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Boston, Massachusetts 02114
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Boston, MA
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200 Hawkins Dr,
Iowa City, Iowa 52242
866-452-8507
University of Iowa Hospitals and Clinics University of Iowa Hospitals and Clinics—recognized as one of...
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Iowa City, IA
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Philadelphia, Pennsylvania 19104
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Philadelphia, PA
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Portland, Oregon 97239
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Portland, OR
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