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

X4 Pharmaceuticals191 个研究点分布在 15 个国家目标入组 176 人2024年6月6日

适应症Neutropenia

干预措施Mavorixafor Placebo

概览

阶段: 3 期
干预措施: Mavorixafor
疾病 / 适应症: Neutropenia
发起方: X4 Pharmaceuticals
入组人数: 176
试验地点: 191
主要终点: Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period
状态: 招募中
最后更新: 19天前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验相关资讯

概览

简要总结

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

详细描述

All participants will continue their pre-study background therapy, defined as the participant's current treatment regimen. Options include, but are not limited to, granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or "watchful waiting".

注册库

clinicaltrials.gov

开始日期

2024年6月6日

结束日期

2027年11月1日

最后更新

19天前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

X4 Pharmaceuticals

责任方

Sponsor

入排标准

入选标准

•Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy.
•Congenital Neutropenia, including but not limited to these classifications:
•Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor (CSF3R), C-X-C chemokine receptor 2 (CXCR2), Wiskott-Aldrich syndrome (WAS)
•Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease
•Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
•Shwachman-Diamond syndrome
•Acquired Primary Neutropenia
•Chronic idiopathic neutropenia
•Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with study Medical Monitor.
•Have an ANC \<1000 cells/µL during screening (single ANC value from hematology) and confirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) at baseline visit, with no clinical evidence of systemic infection.

排除标准

•A diagnosis of secondary neutropenia including those due to:
•Hypersplenism
•Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease
•Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
•Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.
•A diagnosis of any of the following:
•Aplastic anemia
•Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome
•Certain CNs, including but not limited to these classifications are excluded:
•Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)

研究组 & 干预措施

Mavorixafor

Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.

干预措施: Mavorixafor

Placebo

Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.

干预措施: Placebo

结局指标

主要结局

Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period

时间窗: Up to 52 Weeks

Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response

时间窗: Up to 52 weeks

Positive ANC response: Increase of ANC \>500 cells/microliter (µL) from baseline.

次要结局

Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) Adjudicated by a BIAC During the Treatment Period(Up to 52 Weeks)
Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections(Up to 52 Weeks)
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period(Up to 52 Weeks)
Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period(Up to 52 Weeks)
Change From Baseline in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score(Baseline, Week 52)