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AAV2-hRPE65v2, voretigene neparvovec-rzyl · 1 trial · 2 indications
The MLMT measures changes in functional vision, as assessed by the ability to navigate a course accurately and at a reasonable pace at different levels of environmental illumination. MLMT was assessed using both eyes at 1 or more of 7 levels of illumination, ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night). Each light level was assigned a score code ranging from 0 to 6. A higher score indicated that a subject was able to pass the MLMT at a lower light level. A score of -1 was assigned to those who could not pass MLMT at 400 lux. The MLMT of each subject was videotaped and assessed by independent graders. The MLMT score was determined by the lowest light level at which the subject was able to pass the MLMT. The MLMT score change was defined as the difference between the score at Baseline and the score at Year 1. A positive MLMT score change from Baseline to Year 1 visit indicated that the subject was able to complete the MLMT at a lower light level.
| Arm | Type | Description |
|---|---|---|
| AAV2-hRPE65v2,voretigene neparvovec-rzyl | EXPERIMENTAL | voretigene neparvovec rzyl, 1.5 E11 vector genomes, per eye, administered by subretinal injection in a volume of 0.3mL, 6-18 days apart |
| Control | NO_INTERVENTION | No intervention |
| Name | Type | Description |
|---|---|---|
| AAV2-hRPE65v2,voretigene neparvovec-rzyl | BIOLOGICAL | Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector genomes per eye) to both eyes via surgical procedures on separate days. |
Inclusion Criteria: * Willingness to adhere to protocol and long-term follow-up as evidenced by written informed consent or parental permission and subject assent (where applicable). * Diagnosis of LCA due to RPE65 mutations; molecular diagnosis is to be performed, or confirmed, by a CLIA-approved ...
AAV2-hRPE65v2, also known as voretigene neparvovec-rzyl, is an investigational gene therapy being studied for the treatment of inherited retinal dystrophy due to RPE65 mutations, including Leber congenital amaurosis. It is currently in Phase 3 clinical development.
AAV2-hRPE65v2 is a gene therapy that targets the RPE65 gene. It is designed to deliver a functional copy of the RPE65 gene to retinal cells to address inherited retinal dystrophy caused by mutations in this gene.
AAV2-hRPE65v2 is being developed by Roche Holding AG, a company traded under the ticker RHHBY. The therapy is currently in Phase 3 clinical trials for inherited retinal dystrophy due to RPE65 mutations.
AAV2-hRPE65v2 is in Phase 3 clinical development. It is an investigational therapy and has not been approved by regulatory authorities. The ongoing Phase 3 trial is active but not recruiting participants.
AAV2-hRPE65v2 is being evaluated in a Phase 3 clinical trial with the identifier NCT00999609. This study, titled 'Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis,' is enrolling 31 participants in the United States and is active but not recruiting.
Yes, AAV2-hRPE65v2 is the same as voretigene neparvovec-rzyl. These names refer to the same investigational gene therapy being developed by Roche for inherited retinal dystrophy due to RPE65 mutations.