Badanie skuteczności mavorixaforu u pacjentów z zespołem WHIM

WHIM syndrome is a rare genetic disorder that stands for Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. This condition affects the immune system, making it difficult for the body to fight infections. The study focuses on a medication called mavorixafor, which is taken as a hard capsule. The purpose of the study is to test how well mavorixafor works in increasing the number of certain white blood cells, called neutrophils, in people with WHIM syndrome. The study also aims to check the long-term safety of the medication.

The study is divided into two parts. In the first part, participants will be randomly assigned to receive either mavorixafor or a placebo. This part is designed to compare the effects of the medication with those of the placebo. In the second part, all participants will receive mavorixafor to further evaluate its safety and how well it is tolerated over a longer period. The study will monitor changes in the levels of neutrophils and other immune cells, as well as any side effects that may occur.

Participants will be involved in the study for about a year, with regular check-ups every three months. These check-ups will include various tests to assess the effectiveness and safety of mavorixafor. The study aims to provide valuable information on whether mavorixafor can be a beneficial treatment for people with WHIM syndrome.