計劃書編號MG0006
試驗執行中
2023-12-01 - 2026-11-30
Phase II/III
召募中2
ICD-10G70.00
重症肌無力未伴有急性惡化
ICD-10G70.01
重症肌無力伴有急性惡化
ICD-9358.0
重症肌無力
一項開放性、單一組別試驗，評估ROZANOLIXIZUMAB 用於中度至重度全身型重症肌無力兒童試驗參與者的活性、安全性與藥物動力學
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試驗申請者
百瑞精鼎國際股份有限公司
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試驗委託 / 贊助單位名稱
百瑞精鼎國際股份有限公司
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臨床試驗規模
多國多中心
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更新日期
2026/02/01
試驗主持人及試驗醫院
實際收案人數
0 召募中
實際收案人數
0 召募中
適應症
全身型重症肌無力
試驗目的
評估rozanolixizumab以sc 給藥，用於年齡≥ 2至< 18 歲gMG兒童參與者的安全性與耐受性
藥品名稱
注射劑
主成份
solution for injection
劑型
270
劑量
140mg/mL
評估指標
主要安全性估計目標
治療：Rozanolixizumab
目標族群：年齡≥ 2至< 18 歲出現中度至重度症狀(依試驗主持人判定)的gMG 孩童及青少年
試驗指標：主要試驗指標之定義如下
併發事件處理：
○ 投予救援療法或變更標準照護(SOC)劑量療程。於併發事件發生當下及之後收集的資料，將用於敘述性統計。將採用「治療方針」法。
○ 參與者停用試驗藥品(IMP)，但繼續參與試驗。於併發事件發生當下及之後收集的資料，將用於敘述性統計。將採用「治療方針」法。
○ 參與者中止試驗參與(退出)。截至併發事件發生當下的資料將用於敘述性統計。將採用「治療方針」法。
族群層級摘要：敘述性統計主要安全性試驗指標(收集至試驗結束[EOS]回診為止)為：
治療中產生的嚴重不良事件(TEAE)發生率
因TEAE 而造成永久停用IMP 的發生率
特別關注之不良事件發生率(AESM)
治療：Rozanolixizumab
目標族群：年齡≥ 2至< 18 歲出現中度至重度症狀(依試驗主持人判定)的gMG 孩童及青少年
試驗指標：主要試驗指標之定義如下
併發事件處理：
○ 投予救援療法或變更標準照護(SOC)劑量療程。於併發事件發生當下及之後收集的資料，將用於敘述性統計。將採用「治療方針」法。
○ 參與者停用試驗藥品(IMP)，但繼續參與試驗。於併發事件發生當下及之後收集的資料，將用於敘述性統計。將採用「治療方針」法。
○ 參與者中止試驗參與(退出)。截至併發事件發生當下的資料將用於敘述性統計。將採用「治療方針」法。
族群層級摘要：敘述性統計主要安全性試驗指標(收集至試驗結束[EOS]回診為止)為：
治療中產生的嚴重不良事件(TEAE)發生率
因TEAE 而造成永久停用IMP 的發生率
特別關注之不良事件發生率(AESM)
主要納入條件
Study participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation
Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening
Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening
Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening
Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin (IVIg))
Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening
Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening
Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening
Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin (IVIg))
主要排除條件
Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline
Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-neonatal-Fc receptor (FcRn) medications
Study participant with any active or untreated thymoma
Study participant has a history of thymectomy within 6 months prior to Screening
Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP
Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study
Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-neonatal-Fc receptor (FcRn) medications
Study participant with any active or untreated thymoma
Study participant has a history of thymectomy within 6 months prior to Screening
Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP
Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study
試驗計畫預計收納受試者人數
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台灣人數
4 人
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全球人數
12-15 人
