Contact SCGE
Your email
Message
Send
SCGE Consortium Home
|
About SCGE TCDC
|
Contact Us
|
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
NCTID
NCT06196827
(View at clinicaltrials.gov)
Description
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
(Show More)
Indication
Leber Congenital Amaurosis-Type 2
Compound Name
LX101
Sponsor
Innostellar Biotherapeutics Co.,Ltd
Funder Type
Industry
Status
Active not recruiting
Enrollment Count
9
Therapy Information
Target Gene/Variant
RPE65
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
Dose 1
Undisclosed low dose (0.3mL/eye)
Dose 2
Undisclosed high dose (0.3mL/eye)
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2023-12-25
Completion Date
2027-12
Last Update
2024-01-09
Participation Criteria
Eligible Age
>=6 Years
Standard Ages
Child, Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
2
Locations
China
Regulatory Information
Has US IND
False
Recent Updates
Resources/Links
Patents
US11970519B2