A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Active, not recruiting
X4 Pharmaceuticals
Phase 2
2016-12-01
This is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The
primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose
selection of mavorixafor in participants with WHIM syndrome. Participants are allowed to
continue treatment in an Extension Phase, if regionally applicable, until it becomes
commercially available or until the study is terminated by the Sponsor.
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Active, not recruiting
X4 Pharmaceuticals
Phase 3
2019-10-17
This study has a double-blind, placebo-controlled Randomized Period and an Open-Label
extension Period. The primary objective of the Randomized Period is to demonstrate the
efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels
of circulating neutrophils compared with placebo, and relative to a clinically meaningful
threshold. The primary objective of the Open-Label Period is to evaluate the safety and
tolerability of mavorixafor in participants with WHIM syndrome. Participants are allowed to
continue treatment in the Open-Label extension Period, if regionally applicable, until
mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.
A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Neutropenia Disorders
Recruiting
X4 Pharmaceuticals
Phase 1
2020-10-16
This Phase 1b study will determine the safety and tolerability of mavorixafor in participants
with severe chronic idiopathic neutropenia (CIN) and selected congenital neutropenia
disorders. The anticipated enrollment is up to 25 participants.
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