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A Patient Registry Study for Patients Treated With Voretigene Neparvovec

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03597399
Recruitment Status : Recruiting
First Posted : July 24, 2018
Last Update Posted : March 12, 2019
Information provided by (Responsible Party):
Spark Therapeutics

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.

Condition or disease Intervention/treatment
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy 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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 40 participants
Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration: 5 Years
Official Title: A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec
Actual Study Start Date : January 10, 2019
Estimated Primary Completion Date : August 2028
Estimated Study Completion Date : August 2028

Intervention Details:
  • 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.
    Other Names:
    • AAV2-hRPE65v2
    • voretigene neparvovec
    • gene therapy vector

Primary Outcome Measures :
  1. Collection of all Adverse Events and Serious Adverse Events [ Time Frame: up to 5 years ]
    adverse events

Secondary Outcome Measures :
  1. Collection of Pregnancy Outcomes [ Time Frame: Up to 5 years ]
    Follow pregnancy outcomes in participants (and female partners of male participants) who received voretigene neparvovec

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Individuals who received voretigene neparvovec-rzyl in at least one eye.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03597399

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Contact: Clinical Study Lead 1-215-220-9300

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United States, California
Children's Hospital of Los Angeles Not yet recruiting
Los Angeles, California, United States, 90027
Contact: Aaron Nagiel, MD, PhD         
United States, Florida
Bascom Palmer Eye Institute Not yet recruiting
Miami, Florida, United States, 33136
Contact: Byrom Lam, MD         
United States, Iowa
University of Iowa Hospitals & Clinics Not yet recruiting
Iowa City, Iowa, United States, 52242
Contact: Stephen Russell, MD         
United States, Massachusetts
Massachusetts Eye and Ear Institute Not yet recruiting
Boston, Massachusetts, United States, 02114
Contact: Jason Comander, MD, PhD         
United States, Michigan
Kellogg Eye Center Recruiting
Ann Arbor, Michigan, United States, 48105
Contact: Cagri Besirli, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Contact: Patricia Cobb         
United States, Oregon
Casey Eye Institute Not yet recruiting
Portland, Oregon, United States, 97239
Contact: Paul Yang, MD, PhD         
United States, Pennsylvania
Scheie Eye Institute Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Tomas Aleman, MD         
United States, Texas
Cullen Eye Institute Not yet recruiting
Houston, Texas, United States, 77030
Contact: Tahira Scholle         
Sponsors and Collaborators
Spark Therapeutics
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Study Director: Clinical Ophthalmic Lead Spark Therapeutics

Additional Information:

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Responsible Party: Spark Therapeutics Identifier: NCT03597399     History of Changes
Other Study ID Numbers: SPKRPE-PASS
First Posted: July 24, 2018    Key Record Dates
Last Update Posted: March 12, 2019
Last Verified: December 2018

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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

Keywords provided by Spark Therapeutics:
Leber Congenital Amaurosis (LCA)
Inherited Retinal Dystrophy
Retinitis Pigmentosa (RP)

Additional relevant MeSH terms:
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Retinal Dystrophies
Retinal Degeneration
Retinal Diseases
Eye Diseases