Ignite Creation Date:
2024-05-06 @ 12:53 PM
Last Modification Date:
2024-10-26 @ 1:05 PM
Study NCT ID:
NCT03872479
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2022-12-05
First Post:
2018-10-12
Brief Title:
Single Ascending Dose Study in Participants With LCA10
Sponsor:
Editas Medicine Inc
Organization:
Editas Medicine Inc
Study Overview
Official Title:
Open-Label Single Ascending Dose Study to Evaluate the Safety Tolerability and Efficacy of EDIT-101 in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 LCA10 With Centrosomal Protein 290 CEP290-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c29911655AG in Intron 26 IVS26 of the CEP290 Gene LCA10-IVS26
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2022-12
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 tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c29911655AG in intron 26 of the CEP290 gene LCA10-IVS26
Detailed Description:
This is an open-label single ascending dose study of EDIT-101 in adult and pediatric ie ages 3 to 17 participants with LCA10-IVS26 Up to 34 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of EDIT-101 in this study EDIT-101 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26
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