NCT03512314

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency As Open Label Extension

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: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: AB2 Bio Ltd. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is an open-label extension study for patients previously enrolled in the AB2 Bio Ltd. ongoing Phase III clinical trial NLRC4/XIAP.2016.001 (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

Conditions

• XIAP Deficiency
• NLRC4-MAS

Interventions

Type	Name	Description
DRUG	Tadekinig alfa	Open label, 26 weeks on Tadekinig alfa treatment.

Timeline

• Start date: 2018-01-24
• Primary completion: 2024-04-08
• Completion: 2024-05-08
• First posted: 2018-04-30
• Last updated: 2025-03-17

Locations

11 sites across 3 countries: United States, Canada, Germany

Regulatory

• FDA-regulated drug study