全身型重症肌無力

對於rozanolixizumab 用於全身性重症肌無力(gMG)兒童族群的臨床開發計畫，本項開放性延伸試驗(OLE)MG0008 將作為其中的一部分，以補足進行中的第2/3 期試驗MG0006。 MG0008 試驗將在經試驗主持人判斷出現gMG 症狀惡化而需要給藥時，評估rozanolixizumab 在最多額外3 個6 週治療週期內的長期安全性、耐受性和活性(假設治療期[TP]為6 週，以及觀察期[OP]為8 週)。本試驗將針對後續rozanolixizumab 治療週期的安全性和活性反應以及持續至下一個治療週期的時間，收集其相關資訊。本試驗將支持rozanolixizumab 用於gMG 兒童試驗參與者的目標用途(即，經試驗主持人判斷出現gMG 症狀惡化而進行重複的6 週治療週期)。 主要目的 • 評估rozanolixizumab用於在MG0006篩選回診時年齡≥ 2歲的 gMG 兒童參與者，其經額外6 週治療週期的長期安全性和耐受性 次要目標 • 評估rozanolixizumab的活性 其他目標 • 評估rozanolixizumab的藥物動力學(PK) • 評估rozanolixizumab的免疫原性 • 評估對gMG 藥物的需求 • 評估rozanolixizumab對IgM 與IgA 濃度的影響 • 評估rozanolixizumab在額外6 週治療週期後的長期安全性和耐受性 • 評估rozanolixizumab的活性

注射劑

Rozanolixizumab

270

140mg/ml

Study participants are eligible to be included in the study only if all of the following criteria apply:

Type of participant and disease characteristics
1. Study participant must meet one of the following:
− Study participant completed MG0006 according to the protocol (ie, TP1 and OP1) and further treatment with rozanolixizumab will be in the interest of the study participant in the Investigator’s opinion.
− Study participant completed TP1 according to the protocol and has a worsening of gMG symptoms in OP1 of MG0006 and should receive a new treatment cycle of rozanolixizumab at the discretion of the Investigator.

Weight
2a. Study participant must be a minimum of 10kg in body weight and, based on the Investigator's discretion, the participant must be suitable for a sc infusion/injection.

Sex
3. For female study participants:
− A female participant who is sexually active is eligible to participate if she is not pregnant (see Appendix 4 [Section 10.4]), not breastfeeding, and at least one of the following conditions applies:
◦ Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 (Section 10.4)
OR
A WOCBP who agrees to follow the contraceptive guidance or local regulations for the duration of the study and for at least 90 days after the last dose of study treatment. The study participant must have a negative serum pregnancy test at Screening, which is confirmed to be negative by urine testing prior to the first dose of IMP at Baseline.
− A female participant is eligible to participate if she is not a WOCBP and/or not sexually active.

Note: Contraceptive and barrier use as well as pregnancy testing is required as appropriatefor the age and sexual activity of pediatric participants and as required by local regulation. If the childbearing potential changes after the start of the study (eg, a premenarchal female participant experiences menarche) or the risk of pregnancy changes
(eg, a female participant who is not heterosexually active becomes active), the participant must discuss this with the Investigator, who should determine if a female participant must begin a highly effective method of contraception or a male participant must use a condom. If reproductive status is questionable, additional evaluation should be
considered

Informed consent
4. Study participant is capable of giving/having parent/legal representative(s) provide signed informed consent/study participant assent (where appropriate) as described in Appendix 1 (Section 10.1), which includes compliance with the requirements and restrictions listed in the ICF and/or assent and in this protocol.For study participants without a Gap Period the criteria pertaining to laboratory measurements, the last value from MG0006 (ie, EOS Visit of MG0006) will be used for evaluation of study participant eligibility (see the Schedule of Activities [Section 1.3]).

Study participants are excluded from the study if any of the following criteria apply:
1. Study participant met any mandatory withdrawal or mandatory permanent IMP discontinuation criteria in MG0006 or permanently discontinued IMP

Medical conditions
2. Study participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant’s ability to participate in this study.
3. Study participant has a current history of alcohol or drug use disorder, as defined in Diagnostic and Statistical Manual of Mental Disorders (DSM-5), within the previous 12 months.
4. Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis.
5. Study participant has a known hypersensitivity to any components of the IMP or other FcRn drugs.
6. Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI).
7. Study participant with known concurrent viral hepatitis or known positivity for Hepatitis B surface antigen or Hepatitis C virus (HCV) antibody or known human immunodeficiency virus (HIV) infection

Prior/Concomitant therapy
8. Study participant has received a live vaccination within 4 weeks prior to IMP administration or intends to have a live vaccination during the TPs of the study.

Diagnostic assessments
9. Study participant has an absolute neutrophil count ＜1500cells/mm3 .
10. Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant’s ability to participate in this study.
11. Study participant has a current or recent history of clinically significant severe and/or progressive renal disease.
12a. Study participant has renal impairment defined as eGFR ＜45 mL/min/1.73 m2 as calculated using the under 25 creatinine-based Chronic Kidney Disease in Children equation (Pierce et al, 2021).
13. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) are ＞3x upper limit of normal (ULN) defined as per relevant age range.
14a. Study participant has elevations only in total bilirubin that are ＞1.5xULN (isolated bilirubin ＞1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin is ＜35%) defined as per relevant age range.

For randomized study participants with a Baseline result ＞ULN for ALT, AST, ALP, or total bilirubin, a Baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in the electronic case report form (eCRF).
If a study participant has ＞ULN for ALT, AST, or ALP that does not meet the exclusion limit at Screening, repeat the tests, if possible, prior to dosing to ensure there is no further ongoing clinically relevant increase. In case of a clinically relevant increase, inclusion of the study participant must be discussed with the Medical Monitor.


Other exclusions
A female study participant, who plans to get pregnant during the participation in the study.
Study participant has a history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS) (for study participants aged ≥12 years) or upon medical history (for study participant ＜12 years).