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An open-label, single-arm study evaluating the activity, safety, and pharmacokinetics of rozanolixizumab in pediatric study participants with moderate to severe generalized myasthenia gravis

A study of rozanolixizumab in pediatric study participants with moderate to severe generalized myasthenia Gravis

Ikeda Kaori

UCB Japan Co., Ltd.

8-17-1 Nishi-shinjuku, Shinjuku-ku, Tokyo, 160-0023

CTR-JRCT.UCBJapan@ucb.com

Global Clinical Science & Operation

UCB Japan Co., Ltd.

8-17-1 Nishi-shinjuku, Shinjuku-ku, Tokyo, 160-0023

+81-3-6864-7587

CTR_SCC_UCBJapan@UCB.com

Recruiting

July. 31, 2025

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

- 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 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

Both

Generalized Myasthenia Gravis

Drug: rozanolixizumab
Study participants will pre-defined doses of receive rozanolixizumab for 6 weeks.

- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit [Time Frame: From Baseline up to the EOS Visit (up to 18 weeks)]
- Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit [Time Frame: From Baseline up to the EOS Visit (up to 18 weeks)]
- Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks) [Time Frame: From Baseline up to the EOS Visit (up to 18 weeks)]

- Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6 [Time Frame: From Baseline to the end of Week 6]
- Absolute change in total IgG from Baseline at the end of Week 6 [Time Frame: From Baseline to the end of Week 6]
- Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6 [Time Frame: From Baseline to the end of Week 6]
- Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6 [Time Frame: From Baseline to the end of Week 6]
- Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6 [Time Frame: From Baseline to the end of Week 6]
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 [Time Frame: From Baseline to the end of Week 6]
- Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1) [Time Frame: During TP1 and OP1 (up to 14 weeks)]
- Evaluation of local tolerability at each scheduled assessment during TP1 [Time Frame: At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks)]
- Plasma concentration of rozanolixizumab at the 6-week treatment cycle [Time Frame: At the 6-week treatment cycle]
- Incidence of antidrug antibodies (ADAs) at the end of Week 6 [Time Frame: At the end of Week 6]

+81-3-5494-7297

Yes

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Italy/Poland/Taiwan/Turkey