Ignite Creation Date:
2024-05-06 @ 8:19 PM
Last Modification Date:
2024-10-26 @ 3:25 PM
Study NCT ID:
NCT06333249
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-03-27
First Post:
2024-02-16
Brief Title:
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations SKYLINE
Sponsor:
Beacon Therapeutics
Organization:
Beacon Therapeutics
Study Overview
Official Title:
A Phase 12 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 rAAV2tYF-GRK1-RPGR and a Phase 2 Randomized Controlled Masked Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-08
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 study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector rAAV2tYF-GRK1-RPGR in patients with X-linked retinitis pigmentosa caused by RPGR mutations
Detailed Description:
Approximately 12 participants who were not part of the Phase 12 HORIZON study will be enrolled into the dose expansion portion of the study These participants will be randomized in a 11 ratio to 1 of 2 treatment groups ie Group 1 low dose and Group 2 high dose Each participant will receive the assigned dose of AGTC-501 in one eye on a single occasion
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