Clinical Trials Logo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04516369
Other study ID # CLTW888A11301
Secondary ID 2019-003781-41
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date November 24, 2020
Est. completion date May 31, 2026

Study information

Verified date April 2024
Source Novartis
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.


Description:

This is an open-label, single-arm study to evaluate the safety and efficacy of bilateral subretinal administration of voretigene neparvovec in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include full-field light sensitivity threshold testing, visual fields, visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.


Read more »

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
voretigene neparvovec
Voretigene neparvovec is an adeno-associated viral type 2 (AAV2) gene therapy vector driving expression of normal human retinal pigment epithelium 65 kDa protein (hRPE65) gene.

Locations

Country Name City State
Japan Novartis Investigative Site Meguro-ku Tokyo

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Japan, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change from Baseline in full-field light sensitivity threshold Full-field light sensitivity threshold (FST) is evaluated using white light, as averaged over both eyes. Baseline, Day 30, 90, 180, 270, and Year 1 after second eye injection
Secondary Change from Baseline in visual field Visual Field is assessed using the sum total degrees for VF, averaged over both eyes, as measued using Goldmann kinetic perimetry testing with a III4e target. Baseline, Day 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection
Secondary Change from Baseline in macular threshold Macular threshold is assessed as averaged over both eyes, as measured using Humphrey static visual field testing. Baseline, Day 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection
Secondary Change from Baseline in visual acuity Visual acuity is assessed as averaged over both eyes. Baseline, Day 1, and 3 after first eye injection; Day 1, 3, 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection
Secondary Change from Baseline in FST for long-term period FST is assessed using white light, as averaged over both eyes. Baseline, Year 2, 3, 4 and 5 after second eye injection
Secondary Proportion of subject with the presence of vector shedding of voretigene neparvovec during the study period Assessed as the presence of vector in peripheral blood or collected tear. Baseline, Day 0, 1 and 3 after first eye injection; Day 0, 1, 3, 14, 30, 90, 180, 270, and Year 1 after second eye injection
Secondary Proportion of subject with the presence of immunogenicity of voretigene neparvovec during the study period Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product . Baseline, Day 30, 90, 180, 270, and Year 1 after second eye injection
See also
  Status Clinical Trial Phase
Recruiting NCT05858983 - Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy Phase 1/Phase 2