X4P-001-110
Research type
Research Study
Full title
A Phase 3, randomized, double-blind, placebo-controlled, multicenter study of mavorixafor in participants with congenital and acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections
IRAS ID
1009994
Contact name
Christophe Arbet-Engels
Contact email
Sponsor organisation
X4 Pharmaceuticals Inc.
Clinicaltrials.gov Identifier
Research summary
Chronic Neutropenia (CN) is a condition, defined by a low level of neutrophils in the blood for more than three months. The most common clinical consequences of Chronic Neutropenia are infections. The number of infections could be decreased by increasing the neutrophil count in blood by receiving an already approved treatment: Human recombinant granulocyte-colony stimulating factor (G-CSF). G-CSF might not be working for every patient and many still suffer with recurring infections. Also, G-CSF is administered by injection. The study drug - Mavoxifavor is a medication that could also increase neutrophil count and thus reduce the infections in humans with CN and it is taken by mouth in form of capsules.
To take part in this study, participants must be older than 12 years, have been diagnosed with Chronic Neutropenia and having recurring infections – at least two in the last 12 months. Participants will be receiving their usual therapy for their Chronic Neutropenia together with the study treatment. Participants will be assigned in 1:1 ratio to receive either study drug or a placebo (a pill that does not contain any medication). Participants will take the tablets once a day for about a year. The participants will be required to attend study visits at the site and also to document in a diary at home information about their treatment and how they feel.REC name
London - London Bridge Research Ethics Committee
REC reference
24/LO/0328
Date of REC Opinion
28 May 2024
REC opinion
Further Information Favourable Opinion