Status:
ACTIVE_NOT_RECRUITING
Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Eligibility:
All Genders
4-100 years
Phase:
PHASE3
Brief 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 ...
Detailed 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-associate...
Eligibility Criteria
Inclusion
- Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis designated laboratory in Japan.
- Age four years or older.
- Visual acuity worse than 20/60 (both eyes) and/or visual field less than 20 degrees in any meridian as measured by a III4e isopter or equivalent (both eyes).
- Sufficient viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/ or ophthalmoscopy. Must have either:
- An area of retina within the posterior pole of \> 100 µm thickness shown on OCT, or
- ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole, or
- Remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent
Exclusion
- Any prior participation in a study in which a gene therapy vector was administered.
- Participation in a clinical study with an investigational drug in the past 6 months from screening visit.
- Known hypersensitivity to any of the study treatments including excipients or to medications planned for use in the peri-operative period.
- Unable to reliably perform the FST assessment.
- Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme in the past 6 months from screening visit.
- Prior intraocular surgery within 6 months from screening visit.
- Prior use of any medicines that, in the opinion of the investigator, may have caused retinal damage (e.g., sildenafil or related compounds, hydroxychloroquine, chloroquine, thioridazine, any other retino-toxic compounds)
- Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery or interfere with the interpretation of study. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.
Key Trial Info
Start Date :
November 24 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 22 2026
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT04516369
Start Date
November 24 2020
End Date
June 22 2026
Last Update
April 25 2025
Active Locations (1)
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1
Novartis Investigative Site
Meguro-ku, Tokyo, Japan, 152-8902