Ignite Creation Date:
2024-05-06 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 12:45 PM
Study NCT ID:
NCT03512314
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-11-08
First Post:
2018-04-09
Brief Title:
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension
Sponsor:
AB2 Bio Ltd
Organization:
AB2 Bio Ltd
Study Overview
Official Title:
Open-label Extension Study With Tadekinig Alfa r-hIL-18BP to Monitor Safety and Tolerability in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions NLRC4 Mutation and XIAP Deficiency
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-07
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 an open-label extension study for patients previously enrolled in the AB2 Bio Ltd ongoing Phase III clinical trial NLRC4XIAP2016001 IND N 127953 This OLE study will evaluate the long-term safety and tolerability of Tadekinig alfa in patients suffering from pediatric monogenic autoinflammatory diseases harboring deleterious mutations of NLRC4 and XIAP
Detailed Description:
Pediatric auto-inflammatory conditions related to spontaneous activating mutations of the NLRC4 and with recurrent MAS-like flares with constitutive IL-18 hypersecretion may require long-term blockade of the IL-18 pathway
Patients with X-linked inhibitor of apoptosis XIAP deficiency and suffering from Hemophagocytic-Lymphohistiocytosis HLH a MAS-like syndrome also show high levels of serum IL-18 and may benefit from IL-18 blockade treatment until a curative hematopoietic stem cell transplantation can be performed The safety of IL-18 blockade during long-term periods is of major interest for the treatment of these patients
Patients with X-linked inhibitor of apoptosis XIAP deficiency and suffering from Hemophagocytic-Lymphohistiocytosis HLH a MAS-like syndrome also show high levels of serum IL-18 and may benefit from IL-18 blockade treatment until a curative hematopoietic stem cell transplantation can be performed The safety of IL-18 blockade during long-term periods is of major interest for the treatment of these patients
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