2023-506159-12-00招募中3 期
ARGX-113-2207: An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Argenx
- 入组人数
- 11
- 试验地点
- 11
- 主要终点
- Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of CL and Vd 2. PD parameters: total IgG levels and AChR-Ab as input for PK/PD modeling analysis
研究概览
简要总结
To confirm an appropriate dose of efgartigimod PH20 SC in pediatric participants with gMG
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Follow-Up period
- 盲法
- None
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
- •The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written informed consent/assent and complying with protocol requirements. • The participant is aged 2 to <18 years at the time of informed consent/assent. • The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for anti-acetylcholine receptor antibodies • The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable concomitant MG therapy. If receiving corticosteroids and/or immunosuppressants, must be on a stable dose for ≥1 month before screening. • The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug.
排除标准
- •Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant during their participation in the study. • Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication. • Has a documented lack of clinical response to plasma exchange (PLEX). • Received a live or live-attenuated vaccine within <4 weeks before screening. • Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study. • Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised Myasthenia Gravis or puts the participant at undue risk. • History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer • Clinically significant active infection that is not sufficiently resolved in the investigator’s opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV • Has a positive PCR test for SARS-CoV-2 at screening. • Has/had a clinically significant disease, had recent major surgery (within 3 months of screening) or intends to have major surgery during the study, or has/had any other medical condition that, in the investigator’s opinion, would confound the results of the study or put the participant at undue risk. • Has received a different study drug in another clinical study within <12 weeks before screening. • Is currently participating in another interventional clinical study. • Has previously participated in an efgartigimod clinical study and received at least one dose of study drug. • Has a known hypersensitivity to study drug or any of its excipients • Has a history of or current episode of alcohol, drug, or medication abuse as assessed by the investigator. • Use of some medications before screening (more information is found in the protocol) The complete list of exclusion criteria can be found in the protocol.
结局指标
主要结局
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of CL and Vd 2. PD parameters: total IgG levels and AChR-Ab as input for PK/PD modeling analysis
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of CL and Vd 2. PD parameters: total IgG levels and AChR-Ab as input for PK/PD modeling analysis
次要结局
- Incidence and severity of adverse events (AEs), including serious AEs and AEs of special interest • Changes in vital signs, electrocardiogram (ECGs), and clinical laboratory tests • Efgartigimod serum concentrations • Total IgG and AChR-Ab levels: absolute values, changes from baseline values, and percent (%) reductions from baseline values"
- Prevalence and incidence of antidrug antibodies (ADA) against efgartigimod and antibodies against rHuPH20 • Myasthenia Gravis Activities of Daily Living (MG-ADL) total score: absolute value and change from baseline appropriate for pediatric use • Quantitative Myasthenia Gravis (QMG): absolute value and change from baseline "
- EQ-5D-Y: absolute value and change from baseline • Quality of Life in Neurological Disorders Questionnaire (Neuro-QoL) Pediatric Fatigue Score: change from baseline • Clinical Global Impression of Improvement (CGI-I): change from baseline • Changes in protective antibody titers to vaccines
研究者
Chief Scientific Officer
Scientific
Argenx
研究点 (11)
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