NCT03512314

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

Completed Phase 3 Last updated 17 March 2025

What this trial tests

Phase 3 trial testing Tadekinig alfa in XIAP Deficiency in 11 participants. Completed in 8 May 2024.

Timeline

24 January 2018

Primary endpoint
8 April 2024

8 May 2024

Quick facts

Lead sponsor	AB2 Bio Ltd.
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	11
Start date	24 January 2018
Primary completion	8 April 2024
Estimated completion	8 May 2024
Sites	11 locations across Canada, United States, Germany

Drugs / interventions tested

• Tadekinig alfa — full drug profile →

Conditions studied

• XIAP Deficiency — all drugs for XIAP Deficiency →
• NLRC4-MAS — all drugs for NLRC4-MAS →

Sponsor

AB2 Bio Ltd. — full company profile →

Who can join

Eligibility, any sex, with XIAP Deficiency or NLRC4-MAS. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Can we use interleukin-6 (IL-6) blockade for coronavirus disease 2019 (COVID-19)-induced cytokine release syndrome (CRS)?
   Liu B, Li M, Zhou Z, Guan X, et al · · 2020 · cited 507× · PMID 32291137 · DOI 10.1016/j.jaut.2020.102452
2. Biological and clinical roles of IL-18 in inflammatory diseases.
   Landy E, Carol H, Ring A, Canna S. · · 2024 · cited 117× · PMID 38081945 · DOI 10.1038/s41584-023-01053-w
3. Interleukin-18 in Inflammatory Kidney Disease.
   Hirooka Y, Nozaki Y. · · 2021 · cited 69× · PMID 33732720 · DOI 10.3389/fmed.2021.639103
4. Hemophagocytic lymphohistiocytosis: current treatment advances, emerging targeted therapy and underlying mechanisms.
   Wu Y, Sun X, Kang K, Yang Y, et al · · 2024 · cited 47× · PMID 39511607 · DOI 10.1186/s13045-024-01621-x
5. Riding the storm: managing cytokine-related toxicities in CAR-T cell therapy.
   Hughes AD, Teachey DT, Diorio C. · · 2024 · cited 37× · PMID 39012374 · DOI 10.1007/s00281-024-01013-w
6. Immunopathology of and potential therapeutics for secondary hemophagocytic lymphohistiocytosis/macrophage activation syndrome: a translational perspective.
   Nguyen TTT, Kim YT, Jeong G, Jin M. · · 2024 · cited 26× · PMID 38448692 · DOI 10.1038/s12276-024-01182-6
7. Interleukin-18 Binding Protein in Immune Regulation and Autoimmune Diseases.
   Park SY, Hisham Y, Shin HM, Yeom SC, et al · · 2022 · cited 19× · PMID 35885055 · DOI 10.3390/biomedicines10071750
8. Immune-mediated inflammatory diseases with chronic excess of serum interleukin-18.
   Miyazawa H, Wada T. · · 2022 · cited 13× · PMID 35958573 · DOI 10.3389/fimmu.2022.930141