NCT06987539招募中2 期
A Phase 2 Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children From 2 Years to Less Than 18 Years of Age With Generalized Myasthenia Gravis (gMG)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Amgen
- 入组人数
- 15
- 试验地点
- 10
- 主要终点
- Maximum Observed Concentration (Cmax) of Inebilizumab
研究概览
简要总结
The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines before any study-specific activities/procedures being initiated.
- •Age ≥ 2 to < 18 years of age on the day of enrollment.
- •Diagnosis of gMG defined as:
- •Positive serologic test for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody (Ab) titers as confirmed at screening (1 retest allowed), and
- •At least 1 of the following:
- •History of abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; or
- •History of positive anticholinesterase test (eg, edrophonium chloride test); or
- •Participant demonstrated improvement in gMG signs on oral cholinesterase inhibitors, as assessed by the treating physician; or
- •Clinical syndrome consistent with a diagnosis of gMG, and not otherwise explained by another condition.
- •Myasthenia Gravis Foundation of America Clinical Classification Class II, III, or IV at the time of screening.
- •Participants must be on:
- •Corticosteroids only, with no dose increase within 4 weeks prior to screening, or
- •One allowed non-steroidal immunosuppressive therapies (IST), (azathioprine, mycophenolate mofetil, or mycophenolic acid) with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening, or
- •Combination of (1) corticosteroids with no dose increase within 4 weeks prior to screening and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening.
- •The maximum allowed dose of prednisone at the time of enrollment will be 40 mg/day or 80 mg every other day, or equivalent corticosteroid dose.
- •Tacrolimus is allowed in Japan only, with continued use for ≥ 6 months prior to screening and no dose increase within 4 months prior to screening.
- •Participants may enter the study on a stable dose of acetylcholinesterase inhibitors (pyridostigmine dose). The acetylcholinesterase inhibitor dose must have been stable for at least 2 weeks prior to enrollment.
- •Vital signs and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the investigator.
排除标准
- •Employees of the Sponsor, contract research organization (CRO), site staff, and their family members.
- •Thymectomy within 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the treatment period.
- •Unresected thymoma- Participants with benign thymoma resected > 12 months prior to screening may enroll.
- •History of recurrent significant infections.
- •Known immunodeficiency disorder, including current infection or positive test for human immunodeficiency virus (HIV).
- •Positive test for chronic hepatitis B infection at screening.
- •History of untreated hepatitis C infection, or positive antibody test for hepatitis C virus (HCV).
- •Active tuberculosis (TB); latent TB with no documented history of adequate treatment per local standard of care; or a positive QuantiFERON®-TB test at screening, unless treatment for TB was completed per local guidelines.
- •History of progressive multifocal leukoencephalopathy.
- •Participants diagnosed with congenital myasthenic syndromes.
- •Receipt of any biologic B-cell-depleting therapy (eg, rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab) or any experimental B-cell-depleting agent in the 6 months prior to screening.
- •Receipt of any other monoclonal antibody (mAb) or large molecule biologic, including but not limited to FcRn inhibitors, anti-TNF mAbs, anti-janus kinase (JAK) Stat mAbs, and complement inhibitors within 6 months prior to screening.
- •Receipt of the following medications or treatments at any time prior to enrollment: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy, natalizumab.
- •Participants who are pregnant or breastfeeding or planning to get pregnant.
- •Receipt of intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) within 4 weeks prior to enrollment.
研究组 & 干预措施
Inebilizumab
Experimental
Inebilizumab will be administered intravenously (IV).
干预措施: Inebilizumab (Drug)
结局指标
主要结局
Maximum Observed Concentration (Cmax) of Inebilizumab
时间窗: Up to Week 52
Area Under the Concentration-time Curve (AUC) of Inebilizumab
时间窗: Up to Week 52
Half-life (t1/2) of Inebilizumab
时间窗: Up to Week 52
Clearance (CL) of Inebilizumab
时间窗: Up to Week 52
Volume of Distribution at Steady State (Vss) of Inebilizumab
时间窗: Up to Week 52
Change from Baseline in Cluster of Differentiation 20 (CD20)+ B-cell Counts
时间窗: Baseline and Week 78
Number of Participants Experiencing Treatment-emergent Adverse Events
时间窗: Up to Week 78
Number of Participants Experiencing Clinically Significant Changes in Laboratory Parameters
时间窗: Up to Week 78
Number of Participants Experiencing Clinically Significant Changes in Vital Signs
时间窗: Up to Week 78
次要结局
- Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score(Baseline and Week 78)
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) Score(Baseline and Week 78)
- Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score(Baseline and Week 78)
- Change from Baseline in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) Score(Baseline and Week 78)
- Change from Baseline in Neurological Quality of Life (Neuro-QoL) Paediatric Fatigue Score(Baseline and Week 78)
- Number of Participants with Anti-drug Antibodies (ADAs) Present(Up to Week 78)
研究者
研究点 (10)
Loading locations...
相似试验
进行中（未招募）
2 期
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyNCT06450639Hoffmann-La Roche30
Unknown
2 期
An Open Label Study of Multiple Doses of Cannabidiol in the Prevention of Acute Graft-Versus-Host Disease (GVHD)Prevention aGVHDNCT03840512Kalytera Therapeutics Israel, Ltd.36
已完成
2 期
A Study of Bomedemstat (IMG-7289/MK-3543) in Participants With Polycythemia Vera (IMG-7289-CTP-203/MK-3543-004)Polycythemia VeraNCT05558696Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)20
招募中
2 期
AZD0901 in Participants With Advanced Solid Tumours Expressing Claudin18.2Gastric CancerNCT06219941AstraZeneca226
撤回
1 期
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung DiseaseLung DiseasePulmonary DiseaseAlpha-1 Antitrypsin Deficiency-associated Lung DiseaseAATDAlpha-1 Antitrypsin DeficiencyNCT06622668Intellia Therapeutics30