Ignite Creation Date:
2024-05-06 @ 5:08 PM
Last Modification Date:
2024-10-26 @ 2:22 PM
Study NCT ID:
NCT05203939
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-10-25
First Post:
2021-11-16
Brief Title:
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Sponsor:
Ocugen
Organization:
Ocugen
Study Overview
Official Title:
A Phase 12 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 and RHO Mutations and Leber Congenital Amaurosis With Mutations in CEP290 Gene
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase 12 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutations in CEP290 gene OCU400-101 To document prospective eye pathology in the above subjects Investigators will also conduct a Natural History Study OCU400-104i
This is a multicenter study which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study
This is a multicenter study which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study
Detailed Description:
This study will be conducted in two phases enrolling up to 24 subjects Treated subjects will receive a single subretinal injection of OCU400 in the study eye
This is a multicenter open-label dose-ranging study in two subgroups of subjects with three consecutive cohorts
A total of 18 adult RP subjects from each of the following subgroups with Biallelic autosomal recessive NR2E3 mutations autosomal dominant NR2E3 mutations or Autosomal dominant RHO mutations will be selected for dose escalation
For the Phase I portion of the study the 33 design for sequential dose-escalating cohorts will be used with scheduled 3 dosing levels between 9 and 18 subjects will be used to follow the design
Up to 3 additional adult LCA patients with CEP290 mutations and at least 1 pediatric LCA subject will be enrolled in the Phase 2 portion
Sample Size Justification
The trial will enroll up to 24 patients 18 adult RP up to 3 LCA patients and at least 1 pediatric LCA patient in both Phase 1 and Phase 2 components
Natural History Study OCU400-104 A Prospective and Retrospective Natural History Study of RP and LCA
This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with the earliest timepoint on or after the date of their diagnosis of RP or LCA
This is a multicenter open-label dose-ranging study in two subgroups of subjects with three consecutive cohorts
A total of 18 adult RP subjects from each of the following subgroups with Biallelic autosomal recessive NR2E3 mutations autosomal dominant NR2E3 mutations or Autosomal dominant RHO mutations will be selected for dose escalation
For the Phase I portion of the study the 33 design for sequential dose-escalating cohorts will be used with scheduled 3 dosing levels between 9 and 18 subjects will be used to follow the design
Up to 3 additional adult LCA patients with CEP290 mutations and at least 1 pediatric LCA subject will be enrolled in the Phase 2 portion
Sample Size Justification
The trial will enroll up to 24 patients 18 adult RP up to 3 LCA patients and at least 1 pediatric LCA patient in both Phase 1 and Phase 2 components
Natural History Study OCU400-104 A Prospective and Retrospective Natural History Study of RP and LCA
This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with the earliest timepoint on or after the date of their diagnosis of RP or LCA
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None