2024-513854-31-00招募中3 期
ARGX-113-2006: Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Argenx
- 入组人数
- 12
- 试验地点
- 13
- 主要终点
- Efgartigimod concentrations as input for compartmental, model-driven analysis to determine age and size dependency of clearance and volume of distribution
研究概览
简要总结
To confirm an age-adjusted optimum dose of efgartigimod IV and provide (model-predicted) evidence for a treatment response
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable, willingness and ability to comply with the trial protocol procedures.
- •Male or female participants between 2 to < 18 years of age at the time of providing informed consent/assent.
- •Diagnosed with gMG with confirmed documentation
- •Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
- •Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or AChE inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening.
- •Positive serologic test for anti-AChR antibodies at screening (for younger participants (<15kg) historical values can be used).
排除标准
- •Participants with MGFA class I, IVb, and V.
- •Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
- •A documented lack of clinical response to plasma exchange (PLEX).
- •Received a live or live-attenuated vaccine fewer than 28 days before screening. (Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.)
- •Received a thymectomy <3 months before screening or 1 is planned to be performed during the trial period.
- •A known hypersensitivity reaction to efgartigimod or any of its excipients.
- •Current participation in another interventional clinical trial.
结局指标
主要结局
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine age and size dependency of clearance and volume of distribution
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine age and size dependency of clearance and volume of distribution
PD parameters: total IgG levels and anti-acetylcholine receptors antibodies (AChR-Ab) as input for PK/PD modeling analysis
PD parameters: total IgG levels and anti-acetylcholine receptors antibodies (AChR-Ab) as input for PK/PD modeling analysis
次要结局
- Incidence and severity of AEs, incidence of serious AEs (SAEs), incidence of AEs of special interest (AESIs), and changes in laboratory test results, physical examination results, vital sign measurements, and electrocardiogram (ECG) (part B only for ECGs)
- Efgartigimod serum concentrations
- Levels of total IgG and AChR-Ab: absolute values, change from baseline and percent (%) change from baseline
- Incidence and prevalence of antidrug antibodies (ADAs) against efgartigimod
- MG-ADL total score: absolute value and change from baseline total MG-ADL score
- Total QMG score: absolute value and change from baseline
- Total score EQ-5D-Y: absolute value and change from baseline
- Quality of Life in Neurological Disorders (Neuro-QoL) Pediatric Fatigue Score: values and change from baseline
- Change in protective antibody titers to vaccines received before or received during the trial
研究者
Chief Scientific Officer
Scientific
Argenx
研究点 (13)
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