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

Completed · Phase 3

Conditions studied: XIAP Deficiency, NLRC4-MAS

In brief

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.

Key facts

Study ID

NCT03512314

Run by

AB2 Bio Ltd.

People needed

11

Starts

2018-01-24

Expected to finish

2024-05-08

Last updated by the study team

2025-03-17

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• 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
• Evidence or history of malignancy
• Evidence of invasive or life-threatening infection
• History of tuberculosis
• Life-threatening bleeding within 2 weeks of screening
• Vaccination with a live vaccine within the previous 3 months
• Evidence of severe organ compromise including but not limited to: (see details in the protocol)
• Pregnant or breastfeeding females
• Inability to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment.
• Inability to provide informed consent, and also assent if applicable
• Life expectancy less than 4 weeks
• Concomitant use of other immunosuppression except NSAIDs, glucocorticoids, cyclosporine, tacrolimus, IL-1 inhibitors (Anakinra, Canakinumab, or Rilonacept)

Where it is running

• UCSD _ Department of Pediatrics / Rady Children's Hospital — La Jolla, California, United States
• Shands Children's Hospital — Gainsville, Florida, United States
• Children's Healthcare of Atlanta at Egleston — Atlanta, Georgia, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Texas Children's Hospital _ Baylor College of Medicine — Houston, Texas, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada
• CHU Sainte-Justine — Montreal, Canada
• Universitätsklinikum Freiburg, Centrum für Chronische Immundefizienz (CCI) - Paediatric Unit — Freiburg im Breisgau, Baden-Wurttemberg, Germany

Full record on ClinicalTrials.gov

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