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
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
NCTID
NCT02781480
(View at clinicaltrials.gov)
Description
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
(Show More)
Indication
Leber Congenital Amaurosis-Type 2
Compound Name
Cevaretigene ritoparvovec (AAV2/5-RPE65P/OPTIRPRE65)
Sponsor
MeiraGTx UK II Ltd
Funder Type
Industry
Status
Completed
Enrollment Count
15
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
AAV2/5
Editor Type
Dose 1
1.0 x 10^11 vg/mL
Dose 2
3.0 x 10^11 vg/mL
Dose 3
1.0 x 10^12 vg/mL
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2016-04-28
Completion Date
2018-12
Last Update
2021-07-12
Participation Criteria
Eligible Age
>=3 Years
Standard Ages
Child, Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
2
Locations
United States,United Kingdom
Regulatory Information
Has US IND
True
Recent Updates
Resources/Links
Patents
WO2024079655A1
Preclinical Publications
PMID: 27653967
https://meiragtx.com/wp-content/uploads/2018/08/ESGCT-2017-OPTIRPE65-poster-31-1.pdf
Protocol
https://cdn.clinicaltrials.gov/large-docs/80/NCT02781480/Prot_SAP_000.pdf