Ignite Creation Date:
2025-12-24 @ 9:43 PM
Ignite Modification Date:
2025-12-25 @ 7:24 PM
Study NCT ID:
NCT07054632
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-10
First Post:
2025-06-27
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Sponsor:
Innostellar Biotherapeutics Co.,Ltd
Organization:
Study Overview
Official Title:
Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-07
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:
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
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?: