at La Jolla, California and other locations
study started
completion around



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.

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


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


XIAP Deficiency, NLRC4-MAS, Lymphoproliferative Disorders, X-Linked Genetic Diseases, Tadekinig alfa


You can join if…

(both criteria must be met)

1. Patients have participated in AB2 Bio ltd. Phase III clinical trial NLRC4/XIAP.2016.001 (IND N° 127953) by one of the following mechanisms : a) Patients that have completed the first 18-week RCT phase of the preceding clinical trial but were not eligible for the RW phase due to flare symptoms. Or b) Patients that completed the first 18-week RCT phase and completed the RW phase of the preceding clinical trial. Or c) Patients who have exited either the RCT or RW phase of the preceding clinical trial due to treatment failure requiring rescue immunosuppression. Such patients must wait a minimum of 4 weeks after treatment discontinuation from the preceding clinical trial before enrolling in this OLE. If patients do not consent to enroll in the OLE after their early termination in the main study, they will be asked to continue with the planned visits of the main study 2. Women of childbearing potential with negative urine pregnancy test (UPT) at all visits

You CAN'T join if...

  1. Patients may not enter the OLE if they voluntarily withdrew from RCT or RW study or if the time period between participation exceeds 3 months
  2. Evidence or history of malignancy
  3. Evidence of invasive or life-threatening infection
  4. History of tuberculosis
  5. Life-threatening bleeding within 2 weeks of screening
  6. Vaccination with a live vaccine within the previous 3 months
  7. Evidence of severe organ compromise including but not limited to: (see details in the protocol)
  8. Pregnant or breastfeeding females
  9. Inability to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment.

    10. Inability to provide informed consent, and also assent if applicable 11. Life expectancy less than 4 weeks 12. Concomitant use of other immunosuppression except NSAIDs, glucocorticoids,

    cyclosporine, tacrolimus, IL-1 inhibitors (Anakinra, Canakinumab, or Rilonacept)


  • UCSD _ Department of Pediatrics / Rady Children's Hospital
    La Jolla California 92056 United States
  • Texas Children's Hospital _ Baylor College of Medicine
    Houston Texas 77030 United States


in progress, not accepting new patients
Start Date
Completion Date
AB2 Bio Ltd.
Phase 3 research study
Study Type
Expecting 10 study participants
Last Updated