Ignite Creation Date:
2025-12-24 @ 11:04 PM
Ignite Modification Date:
2026-02-24 @ 5:46 AM
Study NCT ID:
NCT02064569
Status:
COMPLETED
Last Update Posted:
2020-07-01
First Post:
2014-02-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
Sponsor:
GenSight Biologics
Organization:
Study Overview
Official Title:
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene
Status:
COMPLETED
Status Verified Date:
2020-06
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 this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: