EUCTR2020-005841-18-Outside-EU/EEA进行中（未招募）1 期
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. - ADAPT JR
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- argenx BV
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. 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 (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
- •2. Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent.
- •3. Diagnosed with gMG with confirmed documentation
- •4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
- •5. 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.
- •6. Positive serologic test for anti-AChR antibodies at screening (for younger participants (<15kg) historical values can be used).
- •7. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
- •a. Male participants: Male participants must agree to not donate sperm from the time the ICF was signed until the end of the trial.
- •b. Female participants:
- •Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before IMP can be administered.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 12
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Participants with MGFA class I, IVb, and V.
- •2. Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP.
- •3. Has any of the following medical conditions:
- •a) Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
- •b) Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.
- •c) History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for =3 years before the first administration of the IMP. Participants with the following cancers can be included at any time:
- •-Adequately treated 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 (TNM Classification of Malignant Tumors stage T1a or T1b)
- •d) Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk.
- •4. Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
- •5. A documented lack of clinical response to plasma exchange (PLEX).
- •6. 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.
- •7. Received a thymectomy <3 months before screening or 1 is planned to be performed during the trial period.
- •8. The following results from these diagnostic assessments will be considered exclusionary:
- •a. Positive serum test at screening for an active viral infection with any of the following conditions:
- •-Hepatitis B virus (HBV) that is indicative of an acute or chronic infection(https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
- •-Hepatitis C virus (HCV) based on HCV antibody assay
- •-Human immunodeficiency virus (HIV) associated with a CD4 count <200 cells/mm3 with an acquired immunodeficiency syndrome (AIDS)-defining condition, such as: Cytomegalovirus retinitis with loss of vision, Pneumocystis jiroveci pneumonia, chronic intestinal cryptosporidiosis, HIV-related encephalopathy, Mycobacterium tuberculosis (pulmonary or
- •extrapulmonary), or invasive cervical cancer
- •b. Positive nasopharyngeal swab PCR test for SARS-CoV-2 at screening.
- •9. Using the following prior or concomitant therapies:
- •a. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
- •b. Use of any monoclonal antibody within the 6 months before the first dose of the IMP.
- •c. Use of IVIg, immunoglobulins administered SC or intramuscularly, or PLEX within 4 weeks before screening.
- •10. Total IgG levels below the lower limit of normal (LLN) according to the reference ranges of the central laboratory for participant by sex and age at screening.
- •11. A known hypersensitivity reaction to efgartigimod or any of its excipients.
研究者
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