NCT03113760

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: AB2 Bio Ltd. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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 ng/mL 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)

Conditions

• NLRC4-MAS
• XIAP Deficiency

Interventions

Type	Name	Description
DRUG	Tadekinig alfa	Tadekinig alfa is a soluble glycoprotein of 164 amino acids produced from Chinese Hamster Ovary cell line. Tadekinig alfa is supplied as a colorless to slightly yellow, sterile solution for injection in glass vials containing sodium chloride, and 0.02M sodium phosphate buffer as excipients. It is available in a concentration of 20mg/0.5mL.
OTHER	0.9% sodium chloride	To ensure that the treatment remains blinded for the entire study period, the placebo solutions will be supplied in identical vials and similar labelling as the active drug and will be indistinguishable in terms of their texture, color, and smell.

Timeline

• Start date: 2017-07-21
• Primary completion: 2023-10-31
• Completion: 2023-11-02
• First posted: 2017-04-14
• Last updated: 2025-07-02
• Results posted: 2025-07-02

Locations

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

Regulatory

• FDA-regulated drug study