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

• ClinicalTrials.gov Identifier: NCT03597399
• Recruitment Status: Active, not recruiting
• First Posted: July 24, 2018
• Last Update Posted: February 18, 2021
• Sponsor: Spark Therapeutics
• Information provided by (Responsible Party): Spark Therapeutics

Study Description

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.

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.

Study Design

• Study Type: Observational [Patient Registry]
• Actual Enrollment: 86 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 in US
• Actual Study Start Date: January 10, 2019
• Estimated Primary Completion Date: June 2025
• Estimated Study Completion Date: June 2025

Groups and Cohorts

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

Outcome Measures

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

Eligibility Criteria

• 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.

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.

Contacts and Locations

Locations

United States, California

Children's Hospital of Los Angeles

Los Angeles, California, United States, 90027

United States, Florida

Bascom Palmer Eye Institute

Miami, Florida, United States, 33136

United States, Iowa

University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States, 52242

United States, Massachusetts

Massachusetts Eye and Ear Institute

Boston, Massachusetts, United States, 02114

United States, Michigan

Kellogg Eye Center

Ann Arbor, Michigan, United States, 48105

United States, Ohio

Cincinnati Eye Institute

Cincinnati, Ohio, United States, 45242

United States, Oregon

Casey Eye Institute

Portland, Oregon, United States, 97239

United States, Pennsylvania

Scheie Eye Institute

Philadelphia, Pennsylvania, United States, 19104

United States, Texas

Cullen Eye Institute

Houston, Texas, United States, 77030

Sponsors and Collaborators

Spark Therapeutics

Investigators

• Study Director: Clinical Ophthalmic Lead; Spark Therapeutics

More Information

Additional Information:

• Responsible Party: Spark Therapeutics
• ClinicalTrials.gov Identifier: NCT03597399
• Other Study ID Numbers: SPKRPE-PASS
• First Posted: July 24, 2018
• Last Update Posted: February 18, 2021
• Last Verified: February 2021

• 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; RPE65; Retinitis Pigmentosa (RP)

Additional relevant MeSH terms:

Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Eye Diseases