Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency

PHASE3CompletedINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

July 21, 2017

Primary Completion Date

October 31, 2023

Study Completion Date

November 2, 2023

Conditions
NLRC4-MASXIAP Deficiency
Interventions
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.

Trial Locations (9)

19104

Children Hospital of Philadelphia, Philadelphia

30322

Children's Healthcare of Atlanta at Egleston, Atlanta

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

77030

Texas Children's Hospital _ Baylor College of Medicine, Houston

79106

Universitätsklinikum Freiburg, Centrum für Chronische Immundefizienz (CCI) - Paediatric Unit, Freiburg im Breisgau

92056

UCSD _ Department of Pediatrics / Rady Children's Hospital, La Jolla

02115

Boston Children's Hospital, Boston

ON M5G 1X8

The Hospital for Sick Children, Toronto

QC H3T 1C5

CHU Sainte-Justine, Montreal

Sponsors

Lead Sponsor

All Listed Sponsors
lead

AB2 Bio Ltd.

INDUSTRY