Ignite Creation Date:
2024-05-06 @ 7:44 AM
Last Modification Date:
2024-10-26 @ 11:52 AM
Study NCT ID:
NCT02599922
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2022-07-22
First Post:
2015-11-05
Brief Title:
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia A Clarity Clinical Trial
Sponsor:
Applied Genetic Technologies Corp
Organization:
Beacon Therapeutics
Study Overview
Official Title:
A Multiple-Site Phase 12 Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 in Patients With Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2022-07
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 will be a non-randomized open-label Phase 12 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene The primary study endpoint will be safety and the secondary study endpoint will be efficacy
Detailed Description:
This will be a non-randomized open-label Phase 12 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene The primary study endpoint will be safety and the secondary study endpoint will be efficacy
Subjects will be enrolled sequentially in seven dosing groups Subjects in Groups 1 2 3 4 5 and 6 will be at least 18 years of age and will receive varying dose levels of study agent Subjects in Group 4a will be 6 to 17 years of age and will receive the same dose as Group 4 Subjects in Groups 5a and 7 will be between 4 and 8 years of age Subjects in Group 5a will receive the same dose as Group 5 and subjects in Group 7 will receive the maximum tolerated dose identified in Groups 1 2 3 4 4a 5 5a and 6
Safety will be monitored by evaluation of ocular and non-ocular adverse events and hematology and clinical chemistry parameters Efficacy parameters will include visual acuity light discomfort testing color vision static visual field ERG adaptive optics retinal imaging functional MRI fMRI color brightness test and OCT
Subjects will be enrolled sequentially in seven dosing groups Subjects in Groups 1 2 3 4 5 and 6 will be at least 18 years of age and will receive varying dose levels of study agent Subjects in Group 4a will be 6 to 17 years of age and will receive the same dose as Group 4 Subjects in Groups 5a and 7 will be between 4 and 8 years of age Subjects in Group 5a will receive the same dose as Group 5 and subjects in Group 7 will receive the maximum tolerated dose identified in Groups 1 2 3 4 4a 5 5a and 6
Safety will be monitored by evaluation of ocular and non-ocular adverse events and hematology and clinical chemistry parameters Efficacy parameters will include visual acuity light discomfort testing color vision static visual field ERG adaptive optics retinal imaging functional MRI fMRI color brightness test and OCT
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R24EY022023 | NIH | None | httpsreporternihgovquickSearchR24EY022023 |