Ignite Creation Date:
2024-05-06 @ 9:55 AM
Last Modification Date:
2024-10-26 @ 12:22 PM
Study NCT ID:
NCT03113760
Status:
COMPLETED
Last Update Posted:
2024-02-02
First Post:
2017-02-28
Brief Title:
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
Sponsor:
AB2 Bio Ltd
Organization:
AB2 Bio Ltd
Study Overview
Official Title:
Multicenter Double-blind Placebo-controlled Randomized Withdrawal Trial With Tadekinig Alfa r-hIL-18BP in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions NLRC4 Mutation and XIAP Deficiency
Status:
COMPLETED
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic interleukin-18 IL 18 driven autoinflammation due to Nucleotide-binding oligomerization domain leucine-rich repeat and caspase recruiting domain CARD domain containing 4 NLRC4 - Macrophage activation syndrome MAS mutation NLRC4-MAS mutation or X-linked inhibitor of apoptosis XIAP deficiency Because of the likelihood for pathogenic IL-18 in certain monogenic diseases patients known to harbor deleterious mutations in NLRC4-MAS or XIAP and who have a history of ongoing inflammation will be enrolled if they have ferritin 500 ngmL or persistent C reactive protein CRP elevation 2 times the upper limit of normal ULN and the patients should have a Modified Autoinflammatory Disease Activity Index mAIDAI 4
Detailed Description:
The study is designed with single-arm open-label phase SAOL of Tadekinig alfa treatment duration for 18-week followed by an up to 16-week Randomized Withdrawal RW period for efficacy and safety evaluation with no interruption between the two phases of treatment The screening period will occur before the SAOL phase and before the first dose of Investigational Medicinal Product IMP
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