Ignite Creation Date:
2024-05-06 @ 5:13 PM
Last Modification Date:
2024-10-26 @ 2:24 PM
Study NCT ID:
NCT05230459
Status:
RECRUITING
Last Update Posted:
2024-04-03
First Post:
2022-01-27
Brief Title:
A Study to Evaluate the Safety of AB-1003 Previously LION-101 in Subjects With Genetic Confirmation of LGMD2IR9 Part1
Sponsor:
Asklepios Biopharmaceutical Inc
Organization:
Asklepios Biopharmaceutical Inc
Study Overview
Official Title:
A Two-part Multicenter Study a Randomized Double-blind Placebo-controlled Dose-escalation Safety Phase Part 1 Followed by Double-blind Placebo-controlled Adaptive Phase Part 2 Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2IR9 Mutations in the Gene Encoding Fukutin Related Protein FKRP
Status:
RECRUITING
Status Verified Date:
2024-04
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 of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2IR9 LGMD2IR9 Participants will be treated in sequential dose-level cohorts Part 1
Detailed Description:
None
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