X4P-001-103 A Study of Mavorixafor in Patients with WHIM Syndrome

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension

• IRAS ID

  272576

• Contact name

  Sorena Kiani-Alikhan

• Contact email

  skiani@nhs.net

• Sponsor organisation

  X4 Pharmaceuticals Incorporated

• Eudract number

  2019-001153-10

• Clinicaltrials.gov Identifier

  NCT03995108

• Clinicaltrials.gov Identifier

  129092, FDA IND Number

• Duration of Study in the UK

  2 years, 0 months, 18 days

• Research summary

  This study is being done to learn about an investigational drug called mavorixafor. The condition being studied is warts, hypogammaglobulinaemia, infections, and myelokathexis (WHIM) Syndrome, a disease that affects the body’s ability to fight off infections. Mavorixafor is a drug that is intended to release certain types of white blood cells (neutrophils and lymphocytes) from the bone marrow. The more of these cells existent in the blood, the better the body can fight infection.
  The purpose of the study is to test mavorixafor when it is given to patients diagnosed with WHIM Syndrome. The goals of the study are to determine whether mavorixafor is safe, how it is tolerated, and what effect it may have on the body and WHIM Syndrome.

• REC name

  London - Central Research Ethics Committee

• REC reference

  20/LO/1075

• Date of REC Opinion

  21 Dec 2020

• REC opinion

  Further Information Favourable Opinion