CTIS2023-508482-32-00进行中（未招募）1 期
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. - X4P-001-110
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 150
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 65+（—）
- 性别
- All
入选标准
- •Participants must be at least 12 years of age, at the time of signing the informed consent/assent, as per the local regulations and guidelines., Bone marrow biopsy or aspirate during screening (or prior documentation of bone marrow biopsy or aspirate within past 9 months) does not demonstrate evidence of hematological malignancy., Body weight of = 15 kg (inclusive)., Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. NOTE: The reliability of sexual abstinence for male and/or female enrollment eligibility needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post ovulation methods) and withdrawal are not acceptable methods of contraception. Male Participants: •A male participant must agree to use highly effective contraception as detailed in Appendix 3 of this protocol during the treatment period and for at least 4 weeks after the last dose for participants early terminating the study or until EOS visit for participants completing the Week 52/Day 365 visit and refrain from donating sperm during this period. Female Participants: •A female participant is eligible to participate if she is not pregnant (see Appendix 3), not breastfeeding, and at least one of the following conditions applies: oNot a woman of childbearing potential (WOCBP) as defined in Appendix 3. OR oA WOCBP who agrees to follow the contraceptive guidance, as mentioned in Appendix 3 during the treatment period and for at least 4 weeks after the last dose of study treatment for participants early terminating the study or until EOS visit for participants completing the Week 52/Day 365 visit., Participant, parent, and/or appropriate legal guardian is capable of giving signed ICF and/or assent as described in Protocol Appendix 1, Section 10.1.3 which includes compliance with the requirements and restrictions listed in the ICF and in this protocol., 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: a.Isolated with a permanent (non-cyclic) presentation, e.g., ELANE, CSF3R, CXCR2, WAS b.Associated with extra-hematological manifestations, e.g., Barth syndrome, Cohen syndrome, G6PC3, Kostmann disease c.Associated with metabolic disorders, e.g., glycogen storage disease 1b (GSD1b) d.Shwachman-Diamond syndrome •Acquired Primary Neutropenia a.Chronic idiopathic neutropenia b.Primary autoimmune neutropenia. Other CN disorders that may be eligible for enrollment can be clarified and approved upon discussion with Study Medical Monitor and Sponsor., Have a confirmed trough ANC < 1500 cells/µL during the screening visit (single ANC measurement) and at baseline visit (mean ANC over 6 hours) held at least 2 weeks prior to D1 dosing, with no clinical evidence of infection. Note: In the event of a systemic infection between the screening and baseline visits that may, in the opinion of the Investigator, have an effect on ANC, the baseline visit may be postponed until the trough ANC has been confirmed to be < 1500 cells/µL., Prior history of recurrent and/or serious infections during the
排除标准
- •A diagnosis of secondary neutropenia including those due to: a.Hypersplenism b.Infection c.Malignancy d.Autoimmune disease, e.g., systemic lupus erythematosus, rheumatoid arthritis, irritable bowel disease, graft-versus-host disease, thyroid disease e.Nutritional deficiency, e.g., vitamin B12, folic acid, copper, caloric malnutrition f.Drug-induced cause, e.g., chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies., Receiving or requiring any medication/therapy that is prohibited (see Protocol Appendix 5)., Received more than 1 dose of mavorixafor in the past., Received a CXCR4 antagonist (other than mavorixafor) in the past 6 months., Patients taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening., Positive hepatitis C virus (HCV) antibodies with confirmation by HCV ribonucleic acid polymerase chain reaction reflex testing., Positive hepatitis B surface antigen (HbsAg) or hepatitis B core antibody (HbcAb). If the participant tests HbsAg negative, HbcAb positive, and hepatitis B surface antibody positive upon reflex testing, the participant would be considered eligible., Laboratory test results meeting = 1 of the following criteria at the screening visit: •Hemoglobin < 9.0 g/dL •Platelets < 30,000/µL •Estimated glomerular filtration rate = 60 mL/min/1.73 m2, as estimated by the Chronic Kidney Disease Epidemiology Collaboration equation •Serum aspartate transaminase > 2.5 × upper limit of normal (ULN) •Serum alanine transaminase > 2.5 × ULN •Total bilirubin > 1.5 × ULN (unless due to Gilbert’s syndrome, in which case total bilirubin = 3.0 × ULN and direct bilirubin > 1.5 × ULN), Prolonged corrected QT interval > 450 ms using Fridericia’s formula at the screening visit., Participant is currently taking or has taken an investigational drug < 30 days prior to the screening visit., Participant is pregnant or breastfeeding., A diagnosis of any of the following: •Aplastic anemia •WHIM syndrome •Certain Congenital Neutropenias, including but not limited to these classifications are excluded: a.Isolated with a cyclic presentation, e.g., ELANE b.Associated with immune dysregulation, e.g., autoimmune lymphoproliferative syndrome, Familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome c.Associated with bone marrow failure, e.g., Fanconi Anemia, Diamond-Blackfan anemia, Telomere biology disorders •Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia)., Unable and/or unwilling to swallow capsules., Known systemic hypersensitivity to the mavorixafor drug substance, its inactive ingredients, or the placebo., A known history of positive serology or viral load for HIV or a known history of acquired immunodeficiency syndrome., Known active COVID 19 infection or a positive test within the local accepted clinical and governmental guidelines for a communicable window. Note: Participants with prior COVID 19 exposure are permitted to enroll if they have a negative test and conform with local guidelines., Major surgery = 6 weeks before the baseline visit requiring general anesthesia or which, in the opinion of the Investigator, may compromise the safety of the participant., A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant’s successful completion of the clinical study, or could, in the opinion of the Investigator or the Sponsor, interfere with the ob
研究者
相似试验
进行中（未招募）
1 期
A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn's disease in adults and adolescents.Active Crohn's diseaseMedDRA version: 20.0 Level: LLT Classification code 10021315 Term: Ileitis terminal System Organ Class: 100000004856EUCTR2015-001924-40-SKCelgene Corporation798
进行中（未招募）
1 期
A clinical trial to assess 2 different doses of BCX7353 compared to placebo as an oral treatment for the prevention of attacks in people with HAEMedDRA version: 20.0Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersHereditary angioedemaEUCTR2017-003966-29-GBBioCryst Pharmaceuticals Inc.121
进行中（未招募）
1 期
A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn’s diseaseActive Crohn's diseaseMedDRA version: 20.0Level: LLTClassification code 10021315Term: Ileitis terminalSystem Organ Class: 100000004856EUCTR2015-001925-18-DKCelgene Corporation1,064
进行中（未招募）
1 期
A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn's disease in adults and adolescents.Active Crohn's diseaseMedDRA version: 20.0Level: LLTClassification code 10021315Term: Ileitis terminalSystem Organ Class: 100000004856EUCTR2015-001924-40-LVCelgene Corporation798
进行中（未招募）
1 期
A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn's disease in adults and adolescents.EUCTR2015-001924-40-DECelgene Corporation798