2024-516015-24-00招募中4 期
Prospective experimental study to evaluate the efficacy and tolerability of efgartigimod as an early treatment in patients with generalised myasthenia gravis.
Bellvitge University Hospital, Fundacio Institut D'Investigacio Biomedica De Bellvitge IDIBELL1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2024年12月5日最近更新:
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Proportion of patients with MGg and anti-AChR antibody responders in MG-ADL after a full course of efgartigimod in combination with the usual prednisone initiation regimen.
研究概览
简要总结
To evaluate the efficacy of efgartigimod as early treatment in patients with MGg and anti-AChR antibodies, using the activities of daily living in myasthenia gravis (MG-ADL) scale.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Adult patients ≥ 18 years of age, of both sexes.
- •Confirmed diagnosis of myasthenia gravis by a neurologist with experience in the disease [clinical symptoms suggestive of MG and positive anti-AChR antibodies and/or an electrophysiological study suggestive of neuromuscular junction disorder]
- •Myasthenia Gravis Foundation of America (MGFA) Class II, III, or IV at the time of the screening visit.
- •Clinical presentation with 5 or more points (more than 50% of the points for non-ocular symptoms) on the MG-ADL scale.
- •Not having received prior immunosuppressive treatment for MG with the exception of corticosteroids that will be started during the study(naïve patients)
- •Women of childbearing potential must have a negative serum pregnancy test at screening, be required to use contraception during the study and for 90 days after the last dose of drug, and agree not to donate eggs during the same period.
- •Men, agree to use adequate contraception and not donate sperm until the end of the study (and/or until 90 days after the last drug infusion).
- •Signed Informed Consent Form.
排除标准
- •MGFA Class I or V.
- •Active hepatitis B, or positivity for hepatitis C (unless treated and cured) and/or HIV at screening.
- •Known autoimmune or non-autoimmune disease that, in the opinion of the treating physician, would interfere with an accurate evaluation of the clinical symptoms of MG or place the patient at undue risk.
- •History of malignancy, unless considered cured with adequate treatment and no evidence of recurrence for ≥ 3 years. (Patients in whom adequate treatment of basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histological finding of prostate cancer -stage can be included at any time). TNM T1a or T1b-).
- •Have received a live or attenuated vaccine during the month prior to the screening visit.
- •Be pregnant and/or breast-feeding or intend to become pregnant during the study or within 90 days of the final dose of efgartigimod.
- •Inability to understand informed consent
- •Presence of symptoms that may endanger the patient's life if immediate rescue treatment is not instituted (intravenous immunoglobulins -I VIG- or plasma exchange/plasmapheresis), according to the treating physician's criteria.
- •Having received any immunosuppressive treatment for MG previously.
- •Treatment with IVIG or plasmapheresis within 4 weeks prior to the screening visit.
- •Treatment with rituximab or eculizumab within 6 months prior to the screening visit.
- •Treatment with any monoclonal antibody at the time of the screening visit.
- •History of thymectomy prior to the screening visit or having a thymectomy scheduled during the planned weeks of the study.
- •Have any surgical intervention scheduled during the planned weeks of the study.
- •Active or chronic uncontrolled and clinically significant bacterial, viral or fungal infection at the time of the screening visit.
结局指标
主要结局
Proportion of patients with MGg and anti-AChR antibody responders in MG-ADL after a full course of efgartigimod in combination with the usual prednisone initiation regimen.
Proportion of patients with MGg and anti-AChR antibody responders in MG-ADL after a full course of efgartigimod in combination with the usual prednisone initiation regimen.
次要结局
- Total score (per patient) and mean total score on the MG-ADL and mean change in the total score on the MG-ADL at weeks 4, 6, 8, 16 and 28, compared to the baseline score.
- Total score (per patient) and mean total QMG score and mean change in total QMG at weeks 4, 6, 8, 16 and 28, compared to baseline score.
- Proportion of responding patients in the QMG.
- Total score (per patient) and mean total MGC score and mean change in total MGC at weeks 4, 6, 8, 16 and 28, compared to baseline score.
- Proportion of responding patients in the MGC.
- Median time to onset of efficacy of efgartigimod, determined by when an improvement of 2 or more points on the MG-ADL scale is achieved.
- Median time of onset of efficacy of efgartigimod, determined by the time at which an improvement of 3 or more points on the QMG scale is achieved.
- Median time of onset of efficacy of efgartigimod, determined by the time at which an improvement of 3 or more points on the MGC scale is achieved.
- Proportion of early responders on the MG-ADL scale.
- Total score (per patient) and mean total score and mean change in total scores in the MG-QOL15r and in the EQ-5D-5L at weeks 4, 6, 8, 16 and 28, compared to the baseline score.
- Dose of steroids (prednisone) that patients are taking at 4, 6, 8, 16, and 28 weeks after starting treatment (day of first infusion of efgartigimod cycle/baseline visit).
- Global levels of IgG, and of the different IgG subclasses, at the screening visit and at weeks 2, 4, 8, 16 and 28 after starting treatment (day of the first infusion of the efgartigimod cycle/baseline visit ).
- Levels of anti-AChR antibody titers at the screening visit and at weeks 2, 4, 6, 8, 16 and 28 after starting treatment (day of the first infusion of the efgartigimod cycle/baseline visit).
- Number and percentage of patients receiving more than one cycle of efgartigimod.
- Number and percentage of patients receiving rescue treatment with IVIG and/or plasmapheresis.
- Number and percentage of patients requiring hospital admission (with description of the causes with number and percentage of them).
- Number, percentage, description and severity of adverse events.
研究者
Carlos Casasnovas
Scientific
Bellvitge University Hospital
研究点 (1)
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