Demonstrating efficiency of drugs to raise white blood cell count
The aim of this study is to demonstrate the efficacy of Mavorixafor in patients with WHIM syndrome.
Research Objectives
Status
Recruitment
Patient Group
Study location
Phase
Study type
Lead investigators
- Dr Daman Langguth
Clinical Trial Coordinator
- Venita Bali
Technical title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension (4WHIM)
About this research project
WHIM syndrome is a rare genetic disease of the immune system that affects the body’s immune system ability to fight off infections. Patients diagnosed with WHIM syndrome have a deficiency in specific infection-fighting antibodies in the blood and have an extremely low level of infection fighting white blood cells called neutrophils, which move from the bone marrow into the bloodstream where they can patrol and protect the body.
Mavorixafor is a drug that is intended to release certain types of white blood cells (neutrophils and lymphocytes) from the bone marrow and to raise the number of white blood cells in blood circulation.
The more of these type of white blood cells you have in your blood the better your body can fight infection and as such, may help to reduce the number and severity of your infections.
The aim of this study is to demonstrate the efficacy of Mavorixafor in patients with WHIM syndrome.
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