Raffaele Badolato, Laia Alsina, Antoine Azar, Yves Bertrand, Audrey A Bolyard, David C Dale, Àngela Deyà-Martinez, Kathryn E Dickerson, Navid Ezra, Henrik Hasle, Hyoung Jin Kang, Sorena Kiani-Alikhan, Taco W Kuijpers, Alexander Kulagin, Daman Langguth, Carina Levin, Olaf Neth, Peter Olbrich, Jane Peake, Yulia Rodina, Caroline E Rutten, Anna Shcherbina, Teresa K Tarrant, Matthias G Vossen, Christian A Wysocki, Andrea Belschner, Gary J Bridger, Kelly Chen, Susan Dubuc, Yanping Hu, Honghua Jiang, Sunny Li, Rick MacLeod, Murray Stewart, Arthur G Taveras, Tina Yan, Jean Donadieu
We investigated efficacy and safety of mavorixafor, an oral CXCR4 antagonist for participants with Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome, a rare immunodeficiency caused by CXCR4 gain-of-function variants. This randomized (1:1), double-blind, placebo-controlled, phase 3 trial enrolled participants aged ≥12 years with WHIM syndrome and absolute neutrophil count (ANC) ≤400/μL. Participants received once-daily mavorixafor or placebo for 52 weeks. Primary endpoint was time (hours) above ANC threshold ≥500/μL (TATANC; over 24 hours)...
April 21, 2024: Blood